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the potential benefits of our business plan and strategy to develop Zimura® (avacincaptad pegol) in age-related retinal diseases and autosomal recessive Stargardt disease, to develop our gene therapy product candidate for rhodopsin-mediated autosomal dominant retinitis pigmentosa and to potentially further expand our product pipeline, including through collaborative gene therapy research programs;

•	our ability to in-license or acquire additional products, product candidates or technologies to treat ophthalmic diseases and the timing, costs, conduct and outcome of preclinical development or clinical trials we undertake for these newly acquired assets;

•	our expectations related to our use of available cash;

•	our estimates regarding expenses, future revenues, capital requirements and needs for, and ability to obtain, additional financing;

•	the timing, costs, conduct and outcome of our ongoing and planned clinical trials, including statements regarding the timing of the initiation of and completion of enrollment in such trials, and the costs to obtain and timing of receipt of initial results from, and the completion of, such trials;

•	the timing of and our ability to obtain marketing approval of our product candidates, and the ability of our product candidates to meet existing or future regulatory standards;

•	the potential advantages of our product candidates;

•	the rate and degree of potential market acceptance and clinical utility of our product candidates, if approved;

•	our estimates regarding the potential market opportunity for our product candidates;

•	the potential receipt of revenues from future sales of our product candidates, if approved;

•	our sales, marketing and distribution capabilities and strategy;

•	our ability to establish and maintain arrangements for the manufacture of our product candidates;

•	our intellectual property position;

•	the impact of existing and new governmental laws and regulations; and

•	our competitive position.

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•	external research and development expenses incurred under arrangements with third parties, such as academic research collaborators, contract research organizations ("CROs") and other vendors and contract manufacturing organizations ("CMOs") for the production of drug substance and drug product; and

•	employee-related expenses for employees dedicated to research and development activities, including salaries, benefits and share-based compensation expense.

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•	Level 1—inputs to the valuation methodology are quoted prices (unadjusted) for an identical asset or liability in an active market. The Company's Level 1 assets consist of investments in money market funds and U.S. Treasury securities.

•	Level 2—inputs to the valuation methodology include quoted prices for a similar asset or liability in an active market or model-derived valuations in which all significant inputs are observable for substantially the full term of the asset or liability. The Company's Level 2 assets may consist of investments in investment-grade corporate debt securities.

•	Level 3—inputs to the valuation methodology are unobservable and significant to the fair value measurement of the asset or liability. The Company does not hold any assets that are measured using Level 3 inputs.

*	Investments in money market funds and investment-grade corporate debt securities with maturities less than 90 days are reflected in cash and cash equivalents in the accompanying Balance Sheets.

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OPH2003 (geographic atrophy (GA) secondary to dry AMD): an ongoing, randomized, double–masked, sham controlled, multi-center Phase 2b clinical trial evaluating the safety and efficacy of Zimura monotherapy in patients with geographic atrophy, or GA, secondary to dry AMD. GA, the end stage of dry AMD, is a disease characterized by retinal cell death and degeneration of retinal tissue. We expect to complete patient recruitment for this clinical trial during the third quarter of 2018.

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OPH2007 (wet AMD): an ongoing, randomized, dose-ranging, open-label, multi-center Phase 2a clinical trial of Zimura in combination with the anti-vascular endothelial growth factor, or anti-VEGF, agent Lucentis® (ranibizumab) for the treatment of wet AMD in patients who have not previously been treated with anti-VEGF agents, referred to as treatment-naïve patients. Wet AMD is characterized by the presence and growth of abnormal new blood vessels under and through the retina. We completed patient recruitment for this trial in April 2018 with a total enrollment of 64 patients.

•	OPH2005 (autosomal recessive Stargardt disease (STGD1)): an ongoing, randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial evaluating the safety and efficacy of Zimura monotherapy for the treatment of autosomal recessive Stargardt disease, referred to as STGD1.

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OPH2006 (IPCV): an ongoing, randomized, dose-ranging, open-label Phase 2a clinical trial of Zimura in combination with the anti-VEGF agent Eylea® (aflibercept) for the treatment of idiopathic polypoidal choroidal vasculopathy, or IPCV, in patients who have not responded to Eylea monotherapy. IPCV is an age-related retinal disease involving the choroidal vasculature characterized by the presence of polypoidal lesions, which leads to vision loss. We are at a very early stage of site initiation and patient recruitment for this trial and may re-evaluate our plans for this trial following receipt of top-line data from our OPH2007 trial of Zimura in combination with Lucentis in wet AMD in late 2018.

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•	compelling science;

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•	an identified unmet medical need based on the current standard of care;

•	a meaningful commercial opportunity based on existing treatment options and treatment options known to be in development; and

•	areas where we believe we can apply our competitive advantages.

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•	external research and development expenses incurred under arrangements with third parties, such as academic research collaborators, contract research organizations, or CROs, and other vendors and contract manufacturing organizations, or CMOs, for the production of API and drug product; and

•	employee-related expenses for employees dedicated to research and development activities, including salaries, benefits and share-based compensation expense.

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•	the scope, rate of progress and expense of our research and development activities, including manufacturing activities;

•	the potential benefits of our product candidates over other therapies;

•	clinical trial results;

•	the terms and timing of regulatory approvals;

•	our ability to market, commercialize and achieve market acceptance for any of our product candidates; and

•	our ability to successfully file, prosecute, defend and enforce patent claims and other intellectual property rights, together with associated expenses.

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•	continue the clinical development of Zimura as currently planned or potentially in other indications if we believe there is a sufficient scientific rationale to pursue such development;

•	continue the preclinical and clinical development of our RHO-adRP gene therapy product candidate;

•	in-license or acquire the rights to, and pursue the development of, other products, product candidates or technologies;

•	pursue our collaborative gene therapy research programs;

•	maintain, expand and protect our intellectual property portfolio;

•	hire additional clinical, manufacturing, quality control, quality assurance and scientific personnel, especially as we increase our internal gene therapy capabilities or if we are successful in acquiring or in-licensing rights to additional products, product candidates or technologies or progressing the clinical development of any of our product candidates;

•	seek marketing approval for any product candidates that successfully complete clinical trials;

•	expand our outsourced manufacturing activities or establish commercial operations or sales, marketing and distribution capabilities, if we receive, or expect to receive, marketing approval for any of our product candidates; and

•	expand our general and administrative functions to support future growth of the company.

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•	the scope, costs and results of our ongoing Zimura clinical programs, as well as any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Zimura in any indication;

•	the scope, costs and results of our efforts to develop our RHO-adRP gene therapy product candidate, including activities to establish manufacturing capabilities and preclinical testing to enable us to file an IND;

•	the extent to which we in-license or acquire rights to, and undertake research or development of products, product candidates or technologies, including any product candidate or other technologies we may evaluate as part of our collaborative gene therapy research programs;

•	the amount of any upfront, milestone payments and other financial obligations associated with the in-license or acquisition of other product candidates;

•	the scope, progress, results and costs of preclinical development and/or clinical trials for any other product candidates that we may develop;

•	the costs and timing of process development, manufacturing scale-up and validation activities and ongoing stability studies associated with our product candidates;

•	our ability to establish collaborations on favorable terms, if at all;

•	the costs, timing and outcome of regulatory reviews of our product candidates;

•	the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending intellectual property-related claims;

•	the timing, scope and cost of commercialization activities for any of our product candidates if we receive, or expect to receive, marketing approval for a product candidate; and

•	subject to receipt of marketing approval, net revenue received from commercial sales of any of our product candidates, after milestone payments and royalty payments that we would be obligated to make.

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(1)	The table above includes our contracted obligations under our sponsored research agreements. We have engaged academic research collaborators to conduct research that has potential to create or enhance technologies to assist our development and commercialization of new products or processes.

(2)	The table above includes our continuing rent obligations through June 2020. In June 2018, we and One Penn Plaza LLC entered into an amendment to the lease for office space at One Penn Plaza in New York, New York extending the term of our lease, which was scheduled to expire in December 2018, through the end of June 2020.

(3)

Severance and Other Employee Benefits represents our commitments under the Board of Directors' approved plan to implement a reduction in personnel that involved approximately 80% of our workforce based on the number of employees at the time the plan was approved. The reduction in personnel was substantially completed during 2017 with a limited number of departing employees scheduled to receive severance payments during 2018.

(4)

This table does not include (a) any royalty payments, sublicense fees or milestone payments which may become payable to third parties under license agreements as the timing and likelihood of such payments are not known with certainty, (b) anticipated expenditures under supply agreements for periods for which we are not yet bound under binding purchase orders, (c) contracts that are entered into in the ordinary course of business that are not material in the aggregate in any period presented above and (d) our royalty purchase liability of $125.0 million as of June 30, 2018, due to the fact that royalty payment obligations are not expected given our lack of plans for the future development of Fovista or any other anti-PDGF product that would fall under our royalty obligation.

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Under a license agreement with Archemix, for each anti-C5 aptamer product that we may develop under the agreement, including Zimura, we are obligated to make payments to Archemix of up to an aggregate of $57.5 million if we achieve specified development, clinical and regulatory milestones, with $30.5 million of such payments relating to a first indication, $24.5 million of such payments relating to second and third indications and $2.5 million of such payments relating to sustained delivery applications. Under the C5 agreement, we are also obligated to make additional payments to Archemix of up to an aggregate of $22.5 million if we achieve specified commercial milestones based on net product sales of all anti-C5 products licensed under the agreement. We are also obligated to pay Archemix a double-digit percentage of specified non-royalty payments we may receive from any sublicensee of our rights under the C5 agreement. We are not obligated to pay Archemix a running royalty based on net product sales in connection with the C5 agreement.

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Under the RHO-adRP License Agreement with UFRF and Penn we are obligated to make payments to UFRF, on behalf of both licensors, of up to an aggregate of $23.5 million if we achieve specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product and up to an aggregate of an additional $70.0 million if the we achieve specified commercial sales milestones with respect to a licensed product. We are obligated to pay UFRF, on behalf of both licensors, royalties at a low single-digit percentage of net sales of licensed products. We are also obligated to pay UFRF, on behalf of both licensors, a double-digit percentage of specified non-royalty payments we may receive from any sublicensee of the licensed patent rights to a third party. Further, if we receive a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and we subsequently use such priority review voucher in connection with a different product candidate, we will be obligated to pay UFRF, on behalf of both licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product. In addition, if we sell such a priority review voucher to a third party, we will be obligated to pay UFRF, on behalf of both licensors, a low double-digit percentage of any consideration received from such third party in connection with such sale.

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•	exposure to known and unknown liabilities, including possible intellectual property infringement claims, violations of laws, tax liabilities and commercial disputes;

•	incurrence of substantial debt, dilutive issuances of securities or depletion of cash to pay for acquisitions;

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•	higher than expected acquisition and integration costs;

•	difficulty in combining the operations and personnel of any acquired businesses with our operations and personnel;

•	inability to maintain uniform standards, controls, procedures and policies;

•	restructuring charges related to eliminating redundancies or disposing of assets as part of any such combination;

•	large write-offs and difficulties in assessing the relative percentages of in-process research and development expense that can be immediately written off as compare to the amount that must be amortized over the appropriate life of the asset;

•	increased amortization expenses or, in the event that we write-down the value of acquired assets, impairment losses;

•	impairment of relationships with key suppliers or customers of any acquired businesses due to changes in management and ownership;

•	inability to retain personnel, key customers, distributors, vendors and other business partners integral to an in-licensed or acquired product candidate or technology;

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potential failure of the due diligence processes to identify significant problems, liabilities or other shortcomings or challenges of an acquired or licensed product candidate or technology, including, without limitation, problems, liabilities or other shortcomings or challenges with respect to intellectual property, product quality, revenue recognition or other accounting practices, partner disputes or issues and other legal and financial contingencies and known and unknown liabilities; and

•	entry into therapeutic modalities, indications or markets in which we have no or limited direct prior development or commercial experience and where competitors in such markets have stronger market positions.

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•	continue the clinical development of Zimura as currently planned or potentially in other indications if we believe there is a sufficient scientific rationale to pursue such development;

•	continue the preclinical and clinical development of our RHO-adRP gene therapy product candidate;

•	in-license or acquire the rights to, and pursue the development of, other products, product candidates or technologies;

•	pursue our collaborative gene therapy research programs;

•	maintain, expand and protect our intellectual property portfolio;

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•	hire additional clinical, manufacturing, quality control, quality assurance and scientific personnel, especially as we increase our internal gene therapy capabilities or if we are successful in acquiring or in-licensing rights to additional products, product candidates or technologies or progressing the clinical development of any of our product candidates;

•	seek marketing approval for any product candidates that successfully complete clinical trials;

•	expand our outsourced manufacturing activities or establish commercial operations or sales, marketing and distribution capabilities, if we receive, or expect to receive, marketing approval for any of our product candidates; and

•	expand our general and administrative functions to support future growth of the company.

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•	the scope, costs and results of our ongoing Zimura clinical programs, as well as any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Zimura in any indication;

•	the scope, costs and results of our efforts to develop our RHO-adRP gene therapy product candidate, including activities to establish manufacturing capabilities and preclinical testing to enable us to file an IND;

•	the extent to which we in-license or acquire rights to, and undertake research or development of products, product candidates or technologies, including any product candidate or other technologies we may evaluate as part of our collaborative gene therapy research programs;

•	the amount of any upfront, milestone payments and other financial obligations associated with the in-license or acquisition of other product candidates;

•	the scope, progress, results and costs of preclinical development and/or clinical trials for any other product candidates that we may develop;

•	the costs and timing of process development, manufacturing scale-up and validation activities and ongoing stability studies associated with our product candidates;

•	our ability to establish collaborations on favorable terms, if at all;

•	the costs, timing and outcome of regulatory reviews of our product candidates;

•	the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending intellectual property-related claims;

•	the timing, scope and cost of commercialization activities for any of our product candidates if we receive, or expect to receive, marketing approval for a product candidate; and

•	subject to receipt of marketing approval, net revenue received from commercial sales of any of our product candidates, after milestone payments and royalty payments that we would be obligated to make.

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•	designing, conducting and successfully completing preclinical research and development activities, including preclinical efficacy and IND-enabling studies, for our product candidates or product candidates we are interested in in-licensing or acquiring, including product candidates we may evaluate as part of our collaborative gene therapy research programs;

•	designing, conducting and completing clinical trials for our product candidates;

•	obtaining favorable results from required clinical trials, including for each ophthalmic product candidate, favorable results from two adequate and well controlled pivotal clinical trials in the relevant indication;

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•	applying for and receiving marketing approvals from applicable regulatory authorities for the use of our product candidates;

•	making arrangements with third-party manufacturers, receiving regulatory approval of our manufacturing processes and our third-party manufacturers’ facilities from applicable regulatory authorities and ensuring adequate supply of drug product;

•	establishing sales, marketing and distribution capabilities, either internally or through collaborations or other arrangements, to effectively market and sell our product candidates;

•	achieving acceptance of the product candidate, if and when approved, by patients, the medical community and third-party payors;

•	if our product candidates are approved, obtaining from governmental and third-party payors adequate coverage and reimbursement for our product candidates and, to the extent applicable, associated injection procedures conducted by treating physicians;

•	effectively competing with other therapies, including the existing standard of care, and other forms of drug delivery;

•	maintaining a continued acceptable safety profile of the product candidate during development and following approval;

•	obtaining and maintaining patent and trade secret protection and regulatory exclusivity, including under the Orphan Drug Act and the Hatch-Waxman Act, if we choose to seek such protections for any of our product candidates;

•	protecting and enforcing our rights in our intellectual property portfolio; and

•	complying with all applicable regulatory requirements, including FDA Good Clinical Practices, or GCP, Good Manufacturing Practices, or GMP, and standards, rules and regulations governing promotional and other marketing activities.

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•	we may not be able to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation of clinical studies for any preclinical product candidates that we in-license or acquire;

•	regulators or institutional review boards may not agree with our study design, including our selection of endpoints, or may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site;

•	we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective clinical research organizations or clinical trial sites, especially in cases where we are working with clinical research organizations or clinical trial sites we have not worked with previously;

•	our contract research organizations, clinical trial sites, contract manufacturers and packagers and analytic testing service providers may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;

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•	we, through our clinical trial sites, may not be able to locate and enroll a sufficient number of eligible patients to participate in our clinical trials as required by the FDA or similar regulatory authorities outside the United States, especially in our clinical trials for orphan or other rare diseases;

•	we may decide, or regulators or institutional review boards may require us, to suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements, including GCPs, or a finding that the participants are being exposed to unacceptable health risks;

•	as there are no therapies approved for either GA, Stargardt disease or RHO-adRP in either the United States or the European Union, the regulatory pathway for product candidates in these indications, including the selection of the primary efficacy endpoint for a pivotal clinical trial, is highly uncertain;

•	there may be changes in regulatory requirements and guidance or we may have changes in trial design that require amending or submitting new clinical protocols;

•	there may be changes in the standard of care on which a clinical development plan was based, which may require new or additional trials;

•	we may decide, or regulators may require us, to conduct additional clinical trials beyond those we currently contemplate or to abandon product development programs;

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the number of patients required for clinical trials of our product candidates to demonstrate statistically significant results may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate or participants may drop out of these clinical trials at a higher rate than we anticipate. These risks may be heightened for clinical trials in orphan diseases, for which the natural history of the disease is less understood, making it more difficult to predict the drug effect required to adequately demonstrate efficacy, and because there are fewer affected patients available to participate in clinical trials;

•	the cost of clinical trials of our product candidates may be greater than we anticipate;

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the supply or quality of our product candidates or product candidates we are investigating or other materials necessary to conduct clinical trials of our product candidates, such as the anti-VEGF drugs we need to use in combination with Zimura in our wet AMD and IPCV trials, may be insufficient or inadequate or we may face delays in the manufacture and supply of our product candidates as a result of a decision to transfer manufacturing between contract manufacturers or on account of interruptions in our supply chain, including in relation to the packaging and distribution or import / export of clinical materials; and

•	we may face delays in the manufacture and supply of any product candidates we are investigating in our collaborative gene therapy research programs as a result of our inability to establish new manufacturing capabilities or processes.

•	be delayed in obtaining marketing approval for our product candidates;

•	not obtain marketing approval at all;

•	obtain approval for indications or patient populations that are not as broad as intended or desired;

•	obtain approval with labeling that includes significant use limitations, distribution restrictions or safety warnings, including boxed warnings;

•	be subject to additional post-marketing testing requirements; or

•	have the product removed from the market after obtaining marketing approval.

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•	efficacy and potential advantages compared to alternative treatments, including the existing standard of care;

•	any restrictions in the label on the use of our products in combination with other medications;

•	any restrictions in the label on the use of our products by a subgroup of patients;

•	restrictions in the label on the use of our combination therapy product candidates, such as Zimura for the treatment of wet AMD or IPCV, limiting their use in combination with particular standard of care drugs, such as a particular anti-VEGF drug;

•	restrictions in the label imposing a waiting period in between intravitreal injections;

•	our and any commercialization partner’s ability to offer our products at competitive prices, particularly in light of the cost of any of our combination therapy product candidates in addition to the cost of the underlying standard of care drug;

•	availability of third-party coverage and adequate reimbursement, particularly by Medicare given the target market for AMD indications for persons over age 50;

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•	increasing reimbursement pressures on treating physicians due to the formation of accountable care organizations and the shift away from traditional fee-for-service reimbursement models to reimbursement based on quality of care and patient outcomes;

•	willingness of the target patient population to try new therapies and of physicians to prescribe these therapies, particularly in light of the existing available standard of care or to the extent our product candidates require invasive procedures for administration, such as subretinal surgery;

•	prevalence and severity of any side effects or perceived safety concerns, especially for new therapeutic modalities such as gene therapy; and

•	whether competing products or other alternatives are more convenient or easier to administer, including whether co-formulated alternatives, alternatives that can be co-administered in a single syringe or alternatives that offer a less invasive method of administration than intravitreal injection come to market.

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There are a number of products in preclinical research and clinical development by third parties to treat dry AMD, including several that are in development for GA secondary to dry AMD. In general, these product candidates can be categorized based on their proposed mechanisms of action. The mechanisms of action for these product candidates include inflammation suppression, such as complement system inhibitors and corticosteroids, visual cycle modulators, antioxidants and neuroprotectants, cell and gene therapies and vascular perfusion enhancers. Based on publicly available information, we are aware that Apellis Pharmaceuticals, Inc., Novartis AG and MorphoSys AG, Hemera Biosciences, Inc., Achillion Pharmaceuticals, Inc. and Catalyst Biosciences, Inc. each have complement inhibitors in development, the most advanced of which we believe is Apellis's pegylated, synthetic peptide targeting complement factor C3. Apellis announced positive Phase 2 results for its product candidate and has announced plans to initiate a Phase 3 program during 2018. If Apellis's Phase 3 program for its complement factor C3 product candidate is successful, it is likely that Apellis may obtain marketing approval for its product candidate in advance of when we could reasonably expect marketing approval for Zimura in GA, if at all. Moreover, based on publicly available information, we are aware that several other companies have announced development programs for the treatment of dry AMD targeting different mechanisms of action outside of the complement system.

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There are a number of products in preclinical research and clinical development by third parties to treat wet AMD. Based on publicly available information, we are aware that multiple mechanisms of action are in clinical or preclinical development for wet AMD, including angiopoietin-2 inhibitors, tyrosine kinase inhibitors, integrin inhibitors, novel VEGF inhibitors and complement inhibitors, as well as a few remaining PDGF inhibitors. Within the complement system, we are aware that Apellis is planning a Phase 1b/2 clinical trial with their C3 inhibitor in combination with anti-VEGF therapy. Allergan recently announced positive top-line results for two Phase 3 trials of its and Molecular Partners AG's anti-VEGF abicipar pegol for treatment-naïve patients with wet AMD, and an additional open-label trial is underway with results expected during the first half of 2019. Based on publicly available data, Novartis also remains on track to file for marketing approval of its anti-VEGF brolucizumab by the end of 2018.

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Moreover, based on publicly available information, we are aware that several other companies and research organizations are pursuing treatments targeting other molecular targets, potential gene therapy treatments and stem cell transplant treatments for the treatment of wet AMD. In addition, other companies are undertaking efforts to develop technologies to allow for topical dosing of therapeutic agents such as anti-VEGF agents or integrin inhibitors through eye-drops or to allow for a less frequent intravitreal dosing schedule than currently used for standard of care anti-VEGF agents.

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The commercial opportunity for Zimura in wet AMD in particular also could be reduced or eliminated if our competitors develop and commercialize products that reduce or eliminate the use of anti-VEGF drugs for the treatment of patients with wet AMD, since we are only developing Zimura for wet AMD as a combination therapy with anti-VEGF agents. Moreover, we expect that if Zimura is approved for combination therapy for the treatment of wet AMD, the cost of combination treatment would be higher than the cost of treatment of wet AMD with Lucentis, Eylea or particularly Avastin monotherapy. Insurers and other third-party payors may encourage the use of anti-VEGF drugs as monotherapy and discourage the use of Zimura in combination with these drugs. This could limit sales of Zimura for this indication.

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There are a number of products in preclinical research and clinical development by third parties to treat Stargardt disease. Based on publicly available information, we are aware that Sanofi, Acucela Inc., Alkeus Pharmaceuticals, Inc., Vision Medicines, Inc., Lin BioScience, Inc., Nightstar Therapeutics plc and ProQR Therapeutics N.V. each have development programs in Stargardt disease. In addition, several academic organizations have early programs in Stargardt disease. Four of these programs, Acucela, Alkeus, Vision Medicines and Lin BioScience, are exploring the use of oral therapeutics, while Sanofi, with technology provided by Oxford BioMedica plc, and Nightstar are each using a gene therapy approach and ProQR is using an RNA interference approach. Acucela’s, Alkeus’s and Sanofi’s product candidates are each in Phase 2 development.

•	There are a number of products in preclinical research by third parties to treat RHO-adRP. Based on publicly available information, we are aware that Telethon Foundation and Columbia University each have early stage gene therapy development programs in RHO-adRP. In addition, Spark Therapeutics previously pursued the development of a gene therapy product candidate for RHO-adRP.

•	our inability to recruit and retain adequate numbers of effective sales and marketing personnel;

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•	the inability of sales personnel to obtain access to adequate numbers of physicians who may prescribe our products;

•	the lack of complementary products to be offered by our sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and

•	unforeseen costs and expenses associated with creating an independent sales and marketing organization.

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•	decreased demand for any product candidates or products that we may develop or in-license;

•	injury to our reputation and significant negative media attention;

•	withdrawal of clinical trial participants;

•	significant costs to defend the related litigation;

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•	substantial monetary awards to trial participants or patients;

•	loss of revenue;

•	reduced time and attention of our management to pursue our business strategy; and

•	the inability to commercialize any products that we may develop or in-license.

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•	collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations and may not perform their obligations as expected;

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collaborators may deemphasize or not pursue development and commercialization of our product candidates or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus, changes in product candidate priorities or available funding or changes in priorities as a result of a merger, acquisition or other corporate restructuring or transaction;

•	collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;

•	collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;

•	we could grant exclusive rights to our collaborators, which would prevent us from collaborating with others;

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disagreements or disputes with collaborators, including disagreements or disputes over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of products or product candidates, might lead to additional responsibilities for us with respect to product candidates or might result in litigation or arbitration, any of which would divert management attention and resources, be time-consuming and be expensive;

•	collaborators with marketing and distribution rights to one or more products may not commit sufficient resources to the marketing and distribution of such product or products;

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collaborators may not properly maintain or defend our intellectual property rights, may infringe the intellectual property rights of third parties, may misappropriate our trade secrets or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to litigation and potential liability; and

•	collaborations may be terminated for the convenience of the collaborator, our breach of the terms of the collaboration or other reasons and, if terminated, we may need to raise additional capital to pursue further development or commercialization of the applicable product candidates.

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•	our product candidates may compete with other product candidates and products for access to a limited number of suitable manufacturing facilities that operate under current good manufacturing practices, or cGMP, regulations;

•	reliance on the third party for regulatory compliance, quality assurance and quality control;

•	the possible breach of the manufacturing agreement by the third party;

•	the possible misappropriation of our proprietary information, including our trade secrets and know-how; and

•	the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us.

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•	the second applicant can establish in its application that its medicinal product, although similar to the orphan medicinal product already authorized, is safer, more effective or otherwise clinically superior;

•	the holder of the marketing authorization for the original orphan medicinal product consents to a second orphan medicinal product application; or

•	the holder of the marketing authorization for the original orphan medicinal product cannot supply sufficient quantities of orphan medicinal product.

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•	restrictions on such products, manufacturers or manufacturing processes;

•	restrictions on the labeling or marketing of a product;

•	restrictions on distribution or use of a product;

•	requirements to conduct post-marketing studies or clinical trials;

•	warning letters or untitled letters;

•	withdrawal of the products from the market;

•	refusal to approve pending applications or supplements to approved applications that we submit;

•	recall of products;

•	damage to relationships with any potential collaborators;

•	unfavorable press coverage and damage to our reputation;

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•	fines, restitution or disgorgement of profits or revenues;

•	suspension or withdrawal of marketing approvals;

•	refusal to permit the import or export of our products;

•	product seizure;

•	injunctions or the imposition of civil or criminal penalties; and

•	litigation involving patients using our products.

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the federal Anti-Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation or arranging of, any good or service, for which payment may be made under a federal healthcare program such as Medicare and Medicaid;

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the federal False Claims Act imposes criminal and civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to be presented, false or fraudulent claims for payment by a federal healthcare program or making a false statement or record material to payment of a false claim or avoiding, decreasing or concealing an obligation to pay money to the federal government, with potential liability including mandatory treble damages and significant per-claim penalties, currently set at $5,500 to $11,000 per false claim;

•	the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;

•	HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and its implementing regulations, also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;

•	the federal Physician Payments Sunshine Act requires applicable manufacturers of covered drugs to report payments and other transfers of value to physicians and teaching hospitals, with data collection beginning in August 2013; and

•	analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws and transparency statutes, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers.

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•	an annual, non-deductible fee on any entity that manufactures or imports specified branded prescription products and biologic agents;

•	an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program;

•	expansion of healthcare fraud and abuse laws, including the civil False Claims Act and the federal Anti-Kickback Statute, new government investigative powers and enhanced penalties for noncompliance;

•	a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% point-of-sale discounts off negotiated prices;

•	extension of manufacturers’ Medicaid rebate liability;

•	expansion of eligibility criteria for Medicaid programs;

•	expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program;

•	new requirements to report certain financial arrangements with physicians and teaching hospitals;

•	a new requirement to annually report product samples that manufacturers and distributors provide to physicians; and

•	a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research.

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•	provide for a classified board of directors such that only one of three classes of directors is elected each year;

•	allow the authorized number of our directors to be changed only by resolution of our board of directors;

•	limit the manner in which stockholders can remove directors from the board of directors;

•	provide for advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors;

•	require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent;

•	limit who may call stockholder meetings;

•	authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a “poison pill” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and

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•	require the approval of the holders of at least 75% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our certificate of incorporation or by-laws.

•	the success of products or technologies that compete with our product candidates, including results of clinical trials of product candidates of our competitors;

•	results of clinical trials for our product candidates and the timing of the receipt of such results;

•	the results of our efforts to in-license or acquire the rights to other products, product candidates and technologies for the treatment of ophthalmic diseases;

•	political, regulatory or legal developments in the United States and other countries;

•	developments or disputes concerning patent applications, issued patents or other proprietary rights;

•	the recruitment or departure of key personnel;

•	the level of expenses related to any of our product candidates or clinical development programs;

•	actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;

•	variations in our financial results or those of companies that are perceived to be similar to us;

•	changes in the structure of healthcare payment systems;

•	market conditions in the pharmaceutical and biotechnology sectors;

•	general economic, industry and market conditions; and

•	the other factors described in this “Risk Factors” section.

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(i)

Reasonable record keeping, audit and reporting obligations sufficient to enable Licensor and Licensee to reasonably verify the payments due to Licensee and to Licensors under such Sublicense and to reasonably monitor such Sublicensee’s progress in developing and/or commercializing Licensed Products, provided that such obligations shall be no less stringent that those provided in this Agreement for Licensee.

(ii)	Infringement and enforcement provisions that do not conflict with the restrictions and procedural requirements imposed on Licensee and do not provide greater rights to Sublicensee than as provided in Section 8.

(iii)	Confidentiality provisions with respect to Confidential Information of Penn consistent with the restrictions on Licensee in Section 17 of this Agreement.

(iv)	A requirement of indemnification of Licensors by Sublicensee that is equivalent to the indemnification of Licensors by Licensee under Section 12 of this Agreement.

(v)	A requirement of obtaining and maintaining insurance by Sublicensee that is equivalent to the insurance requirements of Licensee under Section 12 of this Agreement, including coverage under such insurance of Penn as provided in Section 12.

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(vi)	Restriction on use of Licensor’s names etc. consistent with Section 13 of this Agreement.

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1.1

“Penn Intellectual Property” means all inventions, whether patentable or not, that are (a) conceived or (b) conceived and reduced to practice, in either case ((a) or (b)) in the conduct of the Sponsored Research during the term of this Agreement, including all United States and foreign patent applications claiming said patentable inventions, including any divisional, continuation, continuation-in-part (to the extent that the claims are directed to said patentable inventions), and foreign equivalents thereof, as well as any patents issued thereon and reissues, reexaminations and supplemental examinations thereof and extensions of any of the foregoing. For clarity, Penn Intellectual Property also includes all software created in the conduct of the Sponsored Research during the term of the applicable SOW.

1.2	“Principal Investigator” means the Penn employee who has agreed to serve as faculty investigator for the Sponsored Research as identified in the applicable SOW and shall be responsible for the conduct, supervision and administration of the Sponsored Research under that SOW.

1.3	“Product” means the AAV gene therapy product candidate for autosomal dominant retinitis pigmentosa, licensed by Sponsor pursuant to the License Agreement.

1.4	“Research Results” means all data and information which are generated in the performance of the Sponsored Research during the term of the applicable SOW. Research Results expressly excludes Penn Intellectual Property.

1.5	“Sponsored Research” means the research program described in the applicable SOWs.

1.6

“Statement of Work” or “SOW” means any Statement of Work in substantially the form attached hereto as Attachment A that is entered into as of the Effective Date or in the future by Penn and Sponsor specifying the effective date of the SOW and fully detailing the activities and responsibilities to be undertaken with respect to each applicable research program in addition to any other obligations set forth in this Agreement.

1.7	Other Terms. The definition of each of the following terms is set forth in the section of the Agreement indicated below:

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2.1

Conduct. Penn shall promptly commence the Sponsored Research under an applicable SOW after the Effective Date of such SOW and upon payment by Sponsor of any funds owed thereunder, and shall use good faith efforts to conduct such Sponsored Research substantially in accordance with the terms and conditions of this Agreement and the applicable SOW, including the timelines, if any, set forth therein. Sponsor acknowledges that Penn and the Principal Investigator shall have the freedom to conduct and supervise the Sponsored Research in a manner consistent with Penn’s educational and research missions.

2.2

Principal Investigator. If the services of the Principal Investigator become unavailable to Penn in connection with an applicable SOW for any reason, Penn shall be entitled to designate another member of its faculty who is acceptable to Sponsor to serve as the Principal Investigator of the Sponsored Research thereunder. If an acceptable substitute Principal Investigator has not been designated within [**] after the original Principal Investigator ceases his or her services under the applicable SOW, either Party may terminate the SOW upon written notice thereof to the other Party, subject to the provisions of Article 7.

3.1

Reimbursement. Sponsor shall reimburse Penn for an amount equal to its expenditures and reasonable overhead incurred in the conduct of the Sponsored Research as set forth in the applicable SOW. Sponsor acknowledges that this amount is a good faith estimate only and not a guarantee of the cost to conduct the Sponsored Research. If at any time Penn determines that it will require additional funds for the Sponsored Research, it shall notify Sponsor and provide an estimate of the additional amount. Sponsor shall not be liable for any costs in excess of the amount set forth in the applicable SOW unless it has agreed in writing to provide additional funds.

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3.2	Equipment. Title to any equipment, laboratory animals, or any other materials made or acquired with funds provided under this Agreement shall vest in Penn, and such equipment, animals, or materials shall remain the property of Penn following termination of the applicable SOW.

4.1

Research Results. Sponsor shall have the right to use, copy and distribute and have used, copied and distributed Research Results disclosed to Sponsor in records and reports for any reasonable purpose including for making IND and other regulatory filings with respect to the Product. The foregoing rights shall not grant Sponsor any rights under other copyrights or claims of patent applications or issued patents owned by Penn.

4.2

Records. Principal Investigator shall maintain accurate and complete records of the results of the Sponsored Research and shall provide Sponsor with (a) [**] reports of the progress and results of the Sponsored Research in accordance with the applicable SOW, and (b) ongoing informal updates on the progress and results of the Sponsored Research. Penn shall maintain accurate and complete records of the use of the funds provided by Sponsor and shall make such records available to Sponsor upon reasonable notice during Penn’s normal business hours, but not more frequently than [**] of the Effective Date of the applicable SOW.

4.3

Research Reports. Penn hereby grants Sponsor a royalty-free, nontransferable, non-exclusive right to copy, reproduce and distribute any research reports furnished to Sponsor under this Agreement. Sponsor may not charge fees for said research reports, use said research reports for advertising or promotional activities, or alter or modify said research reports without the prior written permission of Penn.

5.1	Penn Intellectual Property. Except as otherwise set forth in this Agreement or in any other agreement between the Parties, Penn shall retain all right, title and interest in and to Penn Intellectual Property and any patents, copyrights, software and tangible research materials and other intellectual property related thereto.

5.2

Disclosure. Principal Investigator shall promptly provide Penn and Sponsor a written disclosure of any Penn Intellectual Property. Sponsor shall advise Penn in writing, no later than [**] after receipt of such disclosure, whether it requests Penn to file and prosecute patent applications related to such Penn Intellectual Property. If Sponsor does not request Penn to file and prosecute such patent applications, Penn may proceed with such preparation

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5.3

Prosecution. Penn shall control the preparation and prosecution of all patent applications and the maintenance of all patents related to Penn Intellectual Property. With regard to any patent applications filed at the request and expense of Sponsor, Penn will consult with Sponsor on patent preparation, filing, prosecution and maintenance, including by providing Sponsor with a reasonable opportunity to provide suggestions or comments regarding the same, such suggestions or comments to be reasonably considered for inclusion by Penn in good faith. Sponsor shall reimburse Penn within [**] after receipt of invoice for all documented expenses incurred in connection with the filing and prosecution of the patent applications and maintenance of the patents that Sponsor has requested Penn to prosecute under Section 5.2 hereof.

5.4	Software. Principal Investigator shall provide Penn and Sponsor a written disclosure of any copyrightable software created in the conduct of the Sponsored Research during the term of this Agreement that Principal Investigator reasonably considers to be scientifically valuable, provided that Principal Investigator shall have no obligation to provide the source code for such software.

5.5	Option. In consideration of Sponsor’s funding of the Sponsored Research and payment for intellectual property expenses as provided for in Section 5.3, Penn shall grant Sponsor:

(i)

An exclusive first option to amend the License Agreement to include Related Intellectual Property (as defined below) in the exclusive license granted to Sponsor thereunder, which amendment shall include such Related Intellectual Property on the terms set forth in the License Agreement, without the payment of additional consideration by Sponsor. “Related Intellectual Property”, as used herein, means Penn Intellectual Property that is “fully-funded” by Sponsor and either (a) covered by a Valid Claim of the Background Patents or (b) whose manufacture, use, sale or import would, absent the License Agreement, constitute an infringement, inducement of infringement or contributory infringement of any Valid Claim. “Valid Claim”, means a claim of (x) an issued and unexpired patent in Background Patents which claim has not been revoked or held unenforceable or invalid by a decision of a court or governmental agency of competent jurisdiction from which no further appeal can be taken or has been taken within the time allowed for appeal, and has not been abandoned, disclaimed, denied or admitted to be invalid or unenforceable through reissue or disclaimer; or (y) a pending patent application that is included in Background Patents which was filed and is being prosecuted in good faith, and has not been abandoned or finally disallowed without the possibility of appeal or re-filing of the application.

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(ii)

An exclusive first option to negotiate to acquire an exclusive license on commercially reasonable terms to all or any portion of Penn Intellectual Property that is not Related Intellectual Property (“Unrelated Intellectual Property”). Penn and Sponsor will negotiate in good faith to determine the terms of a license agreement as to each item of Unrelated Intellectual Property for which Sponsor has agreed to make payment for intellectual property expenses as provided for in Section 5.3, if any.

(iii)	If Sponsor fails to exercise its option within [**] after disclosure of any Penn Intellectual Property to Sponsor, or if Sponsor fails to make payment for intellectual property expenses as provided for in Section 5.3, Penn shall be free to license the applicable Penn Intellectual Property to any party upon such terms as Penn deems appropriate, without any further obligation to Sponsor.

5.6

Government Rights. Any license granted to Sponsor pursuant to Section 5.5 hereof shall be subject to Penn’s right to use and permit other non-profit organizations to use Penn Intellectual Property for educational and academic research purposes and, if applicable, to the rights of the United States government reserved under Public Laws 96-517, 97-256 and 98-620, codified at 35 U.S.C. 200-212, and any regulations issued thereunder.

6.1

Confidential Information. Sponsor shall not disclose confidential information to Penn unless it is related to the Product or necessary to the performance of the Sponsored Research. Any confidential information provided by Sponsor will be in writing and clearly marked by Sponsor as “Confidential” or, if disclosed orally, written notice will be provided within [**] of disclosure (“Confidential Information”).Penn shall protect Confidential Information with the same degree of care as Penn’s own confidential information. Penn’s and the Principal Investigator’s obligations of confidentiality will exist during the performance of the applicable SOW and for [**] following termination or expiration of the applicable SOW, unless disclosure is required by law or regulation.

(i)	Known by Penn or Principal Investigator without restriction prior to disclosure under this Agreement;

(ii)	Disclosed to Penn or Principal Investigator by a third party without an obligation of confidentiality;

(iii)	Available to the public not through a breach of this Agreement by Penn;

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(iv)	Independently developed by Penn or Principal Investigator without knowledge or use of Confidential Information disclosed by Sponsor under this Agreement;

(v)	Published or disclosed in accordance with the terms of this Agreement; or

(vi)	Required to be produced in litigation or a public investigation. To the extent feasible and permitted by law, Penn will give reasonable notice to Sponsor to allow Sponsor to offer its objections to the production of Confidential Information.

6.2

Penn Intellectual Property. In order to preserve the patentability of Penn Intellectual Property and to preserve Penn’s publication rights, Sponsor shall maintain Penn Intellectual Property, Research Results and information provided by Penn pursuant to the Sponsored Research (whether oral or written) as confidential and shall not disclose such information to any third party until the publication of such information by the Principal Investigator or until Penn provides Sponsor with written verification that all desirable patentable inventions have been protected, whichever occurs sooner. In the event that Sponsor wishes to disclose Research Results or any other information provided by Penn pursuant to the Sponsored Research to a third party prior to the publication of such information by the Principal Investigator or such time as Penn provides Sponsor with written verification that all desirable patentable inventions have been protected, whichever occurs sooner, Sponsor shall obtain Penn’s written permission for such disclosure pursuant to a confidentiality agreement with such third party (provided that no Confidentiality Agreement is required for disclosure to a regulatory agency in connection with any IND or other regulatory filing with respect to the Product), which permission shall not be unreasonably withheld delayed or conditioned; provided that, such permission shall not be required with respect to disclosure of information to a regulatory agency in connection with any IND or other regulatory filing with respect to the Product that occurs [**] or more after the termination or expiration of the applicable SOW. For the sake of clarity, once permission has been granted for a disclosure in connection with any IND or other regulatory filing with respect to the Product, no additional permission shall be required for subsequent disclosures in connection with additional IND or other regulatory filings with respect to the Product.

6.3

Publications. Penn shall have the first right to publish, present or otherwise disclose (each a “Publication”) Research Results or other information and material resulting from the Sponsored Research for any academic purpose. Penn shall furnish the Sponsor with a copy of any proposed Publication at least [**] in advance of the date of the submission of said proposed Publication in order for Sponsor to review and comment on said proposed Publication to (a) determine whether such contains any Confidential Information and (b) enable Sponsor to identify any Penn Intellectual Property that it wishes Penn to file patent applications on or to seek other intellectual property protection for. If within the [**] review period (i) Sponsor notifies Penn that the Sponsor requires deletion from the publication or

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7.1	Term. The initial term of this Agreement shall begin on the Effective Date of this Agreement and shall end three (3) years from the Effective Date unless terminated sooner pursuant to Sections 2.2 or 7.2 hereof. This Agreement may be extended or renewed only by mutual written agreement executed by duly authorized representatives of the Parties.

7.2

Termination. In addition to the termination right set forth in Section 2.2 hereof, either Party may terminate this Agreement or any SOW effective upon written notice to the other Party, if the other Party breaches any of the terms or conditions of this Agreement or the applicable SOW and fails to cure such breach within [**] after receiving written notice thereof. In the event of an incurable breach, the non-breaching Party may terminate this Agreement or the applicable SOW effective immediately upon written notice to the breaching Party.

7.3	Effects of Termination.

(i)

In the event of termination of any SOW prior to its stated term, Penn shall be entitled to payment for the work in progress up to the date of termination, and for allowable costs. Allowable costs include, without limitation, all costs or non-cancellable commitments incurred prior to the receipt, or issuance, by Penn of the notice of termination, and the full cost of each employee, student and faculty member supported hereunder through the end of such commitments to the extent they cannot be transferred to other projects, each to the extent incurred in accordance with the terms and conditions of this Agreement and in any event, in the case of costs for employees, students and faculty members, for a period not in excess of [**] from the Effective Date of the SOW. In the event of termination, Penn shall submit a final report of all costs incurred and all funds received under the applicable SOW within [**] after the effective termination date. The report shall be accompanied by a check in the amount of any excess of funds advanced over costs and allowable commitments incurred. In case of a deficit of funds, Sponsor shall pay Penn the amount needed to cover costs and allowable commitments incurred by Penn under the applicable SOW.

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(ii)	In the event of termination of this Agreement, any SOW(s) in effect at the time of such termination shall continue in effect until expiration or termination of such SOW(s) and the terms of this Agreement shall remain applicable to such SOW(s).

(iii)	Termination of any individual SOW will not result in termination of this Agreement or any other SOW unless the Parties specify such intention in the termination notice.

(iv)	Termination of this Agreement shall not affect the rights and obligations of the Parties accrued prior to termination hereof. The provisions of ARTICLE 3; ARTICLE 4; ARTICLE 5; ARTICLE 6; ARTICLE 7; ARTICLE 8; and ARTICLE 9, shall survive such termination.

8.1

Both Parties represent that its execution of this Agreement and its performance of its obligations hereunder do not conflict with any agreement with or obligation to any third party. Penn further represents that the Principal Investigator and any other Penn personnel assisting the Principal Investigator with performance of the Sponsored Research on behalf of Penn shall be under a duty to assign their entire right, title and interest in and to Penn Intellectual Property to Penn. Without limiting Sponsor’s remedies with respect to any breach of Penn’s representations and covenants hereunder, if at any time the staff of the Penn Center for Innovation becomes aware of any inaccuracy in, noncompliance with, or change in the foregoing representations and covenants of Penn, it will provide Sponsor with prompt written notice thereof. EXCEPT FOR THE FOREGOING REPRESENTATIONS AND COVENANTS, PENN MAKES NO WARRANTIES, EXPRESS OR IMPLIED, AS TO ANY MATTER WHATSOEVER, INCLUDING, WITHOUT LIMITATION, WARRANTIES WITH RESPECT TO THE CONDUCT, COMPLETION, SUCCESS OR PARTICULAR RESULTS OF THE SPONSORED RESEARCH, OR THE CONDITION, OWNERSHIP, MERCHANTABILITY, OR FITNESS FOR A PARTICULAR PURPOSE OF THE SPONSORED RESEARCH OR ANY PENN INTELLECTUAL PROPERTY OR RESEARCH RESULTS OR THAT USE OF PENN INTELLECTUAL PROPERTY OR RESEARCH RESULTS WILL NOT INFRINGE ANY PATENT, COPYRIGHT, TRADEMARK OR OTHER INTELLECTUAL PROPERTY RIGHT OF A THIRD PARTY. WITHOUT LIMITING PENN’S OBLIGATIONS TO PERFORM THE SPONSORED RESEARCH IN ACCORDANCE WITH THIS AGREEMENT, PENN SHALL NOT BE LIABLE FOR ANY DIRECT, INDIRECT, CONSEQUENTIAL, PUNITIVE OR OTHER DAMAGES SUFFERED BY SPONSOR OR ANY OTHER PERSON RESULTING FROM THE SPONSORED RESEARCH OR THE USE OF ANY PENN INTELLECTUAL PROPERTY, ANY RESEARCH RESULTS OR ANY PRODUCTS RESULTING THEREFROM. NOTWITHSTANDING THE FOREGOING, ABSENT SPONSOR’S

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8.2	Indemnification.

(i)

Sponsor shall indemnify, defend and hold harmless Penn and its respective trustees, officers, faculty, students, employees, contractors and agents (the “Penn Indemnitees”) from and against any and all liability, damage, loss, cost or expense (including reasonable attorneys’ fees), which the Penn Indemnitees may hereafter incur, or be required to pay as a result of third party claims based on Sponsor’s use of the results of Sponsored Research or any Penn Intellectual Property or Research Results or as a result of any third party claims based on a breach of this Agreement or any act or omission of Sponsor, its employees, affiliates, contractors, licensees or agents, provided that Sponsor’s obligations pursuant to this Section 8.2(i) shall not apply to the extent such claims or suits result from the negligence or willful misconduct of any of Penn Indemnitees as determined by a court of law.

(ii)

As a condition to a Penn Indemnitee’s right to receive indemnification under this Section 8.2, Penn shall: (a) promptly notify Sponsor when it becomes aware of a claim or suit for which indemnification may be sought pursuant hereto; (b) cooperate with Sponsor in the defense, settlement or compromise of such claim or suit; and (c) permit the Sponsor to control the defense, settlement or compromise of such claim or suit, including the right to select defense counsel. In no event, however, may Sponsor compromise or settle any claim or suit in a manner which (a) admits fault or negligence on the part of Penn or any other Penn Indemnitee; or (b) commits Penn or any other Penn Indemnitee to take, or forbear to take, any action, without the prior written consent of Penn. Penn shall reasonably cooperate with Sponsor and its counsel in the course of the defense of any such suit, claim or demand.

9.1

Force Majeure. Neither Party shall be liable for any failure to perform as required by this Agreement to the extent such failure to perform is due to circumstances reasonably beyond such Party’s control, including, without limitation, labor disturbances or labor disputes of any kind, accidents, failure of any governmental approval required for full performance, civil disorders or commotions, terrorism, acts of aggression, acts of God, energy or other conservation measures imposed by law or regulation, explosions, failure of utilities, mechanical breakdowns, material shortages, disease, or other such occurrences.

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9.2	Relationship of the Parties. Nothing in this Agreement is intended or shall be deemed, for financial, tax, legal or other purposes, to constitute a partnership, agency, joint venture or employer-employee relationship between the Parties. The Parties are independent contractors and at no time will either Party make commitments or incur any charges or expenses for or on behalf of the other Party.

9.3	Expenses. Except as otherwise provided in this Agreement, each Party shall pay its own expenses and costs incidental to the preparation of this Agreement and to the consummation of the transactions contemplated hereby

9.4	Third Party Beneficiary. No party, other than Penn or Sponsor shall be entitled to any rights whatsoever by virtue of the relationships created by or arising under this Agreement, including, without limitation, rights as a third party beneficiary

9.5

Use of Names. Except as otherwise agreed in writing, Sponsor and its affiliates may not use the name, logo, seal, trademark, or service mark (including any adaptation of them) of Penn or any Penn school, organization, employee, student or representative, without the prior written consent of Penn. Notwithstanding the foregoing, Sponsor may use the name of Penn in a non-misleading and factual manner solely to state Sponsor’s funding of this Sponsored Research. Penn shall not use Sponsor’s name without Sponsor’s prior written consent except that Penn may acknowledge Sponsor’s funding of this Sponsored Research and any scientific contributions in scientific publications, in listings of sponsored research projects and for other academic purposes.

9.6	No Discrimination. Neither Penn nor Sponsor will discriminate against any employee or applicant for employment because of race, color, sex, sexual or affectional preference, age, religion, national or ethnic origin, handicap, or veteran status

9.7	Successors and Assignment.

(i)	The terms and provisions hereof shall inure to the benefit of, and be binding upon, the Parties and their respective successors and permitted assigns.

(ii)

Sponsor may not assign or transfer this Agreement, any of Sponsor’s rights or obligations created hereunder, any SOW, or any of Sponsor’s rights or obligations thereunder, by operation of law or otherwise, without the prior written consent of Penn, except to an affiliate or in connection with the sale or transfer of all or substantially all of Sponsor’s business or assets relating to the subject matter of this Agreement, whether by merger, sale of assets or otherwise provided, that, in either case, (a) there exists no material breach by Sponsor of any material term of this Agreement and/or the applicable SOW; (b) Sponsor provides prompt written notice of the affiliate assignment or transaction to Penn; and (c) the assignee agrees in

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(iii)	Any assignment not in accordance with this Section 9.7 shall be void.

9.8	Further Actions. Each Party agrees to execute, acknowledge and deliver such further instruments and to do all such other acts as may be necessary or appropriate in order to carry out the purposes and intent of this Agreement.

9.9

Entire Agreement of the Parties; Amendments. This Agreement and the Schedules and Attachments hereto constitute and contain the entire understanding and agreement of the Parties respecting the subject matter hereof and cancel and supersede any and all prior negotiations, correspondence, understandings and agreements between the Parties, whether oral or written, regarding such subject matter. No waiver, modification or amendment of any provision of this Agreement shall be valid or effective unless made in a writing referencing this Agreement and signed by a duly authorized officer of each Party.

9.10	Governing Law. This Agreement shall be governed by and interpreted in accordance with the laws of the Commonwealth of Pennsylvania, excluding application of any conflict of laws principles that would require application of the law of a jurisdiction outside of the Commonwealth of Pennsylvania.

9.11	Dispute Resolution. If a dispute arises between the Parties concerning this Agreement, then the Parties will confer, as soon as practicable, in an attempt to resolve the dispute. If the Parties are unable to resolve such dispute amicably, then the Parties will submit to the exclusive jurisdiction of, and venue in, the state and Federal courts located in the Eastern District of Pennsylvania.

9.12

Notices and Deliveries. Any notice, request, approval or consent required or permitted to be given under this Agreement shall be in writing and directed to a Party at its address shown below or such other address as such Party shall have last given by notice to the other Party. A notice will be deemed received: if delivered personally, on the date of delivery; if mailed, five (5) days after deposit in the United States mail; if sent via courier, one (1) business day after deposit with the courier service.

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9.13

Waiver. A waiver by either Party of any of the terms and conditions of this Agreement in any instance shall not be deemed or construed to be a waiver of such term or condition for the future, or of any other term or condition hereof. All rights, remedies, undertakings, obligations and agreements contained in this Agreement shall be cumulative and none of them shall be in limitation of any other remedy, right, undertaking, obligation or agreement of either Party.

9.14

Severability. When possible, each provision of this Agreement will be interpreted in such manner as to be effective and valid under law, but if any provision of this Agreement is held to be prohibited by or invalid under law, such provision will be ineffective only to the extent of such prohibition or invalidity, without invalidating the remainder of this Agreement. The Parties shall make a good faith effort to replace the invalid or unenforceable provision with a valid one which in its economic effect is most consistent with the invalid or unenforceable provision.

9.15	Interpretation. The words “include,” “includes” and “including” shall be deemed to be followed by the phrase “without limitation.” All references herein to Articles, Sections, and Schedules shall be deemed references to Articles and Sections of, and Schedules to, this Agreement unless the context shall otherwise require.

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9.16	Counterparts. This Agreement may be executed in counterparts, each of which will be deemed an original, and all of which together will be deemed to be one and the same instrument. A portable document format (PDF) or electronic copy of this Agreement, including the signature pages, will be deemed an original.

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1.	Scope. This document constitutes a Statement of Work as defined in the Agreement. The Collaborative Research described in this SOW are to be conducted in accordance with the terms and conditions of the Agreement.

2.	Penn Principal Investigator.

a.	Principal Investigator Name: [Insert PI name]

b.	Principal Investigator Address: [Insert PI address]

c.	Principal Investigator email and fax: [Insert PI email and fax]

3.	Sponsored Research. A specific description of the Sponsored Research to be conducted pursuant to this SOW is set forth below:

4.	Period of Performance.

5.	Report Schedule.

6.	Payment Terms.

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7.	Term. The term of this SOW will commence on the SOW Effective Date and will terminate on [date] unless earlier terminated in accordance with Article 7 of the Agreement.

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8.	Amendments. No modification, amendment, or waiver of this SOW shall be effective unless in writing and signed by a duly authorized representative of each Party.

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1.	Section 7 of the Employment Letter is hereby replaced in its entirety by the following:

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(i)	“Change in Ownership or Control” shall mean a change in the ownership or effective control of the Company or in the ownership of a substantial portion of the assets of the Company determined in accordance with Section 280G(b)(2) of the Code.

(ii)	“Contingent Compensation Payment” shall mean any payment (or benefit) in the nature of compensation that is made or made available (under this Agreement or otherwise) to a “disqualified individual” (as defined in Section 280G(c) of the Code) and that is contingent (within the meaning of Section 280G(b)(2)(A)(i) of the Code) on a Change in Ownership or Control of the Company.

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1.	In the first sentence of Section 6 of the Employment Letter, the words “any reason” are hereby deleted and replaced with the words “Good Reason”.

2.	In the form of separation agreement attached as Exhibit A to the Employment Letter as the “Separation Agreement and Release of Claims,” the words “any reason” in the preamble and Section 1 thereof are hereby deleted and replaced in each case with the words “Good Reason”.

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1.	I have reviewed this Quarterly Report on Form 10-Q for the quarter ended June 30, 2018 of Ophthotech Corporation;

2.	Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.	Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.	The registrant's other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

5.	The registrant's other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant's auditors and the audit committee of the registrant's board of directors (or persons performing the equivalent functions):