Ocugen, Inc. Announces Submission of Investigational New Drug Application with U.S. Fda to Initiate a Section 1/2 Clinical Trial Evaluating Gene Treatment Candidate OCU400 (AAV-NR2E3) to Handle Inherited Retinal Degeneration

Modifier gene remedy platform has prospective to handle a number of retinal illnesses with 1 product or service

MALVERN, Pa., Nov. 08, 2021 (Globe NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical corporation targeted on exploring, producing, and commercializing gene therapies to heal blindness ailments and producing a vaccine to fight COVID-19, introduced that it has submitted an Investigational New Drug application (IND) with the U.S. Foodstuff and Drug Administration (Food and drug administration) to initiate a Section 1/2 clinical trial of OCU400 (AAV-NR2E3), a modifier gene therapy candidate for the treatment method of retinitis pigmentosa ensuing from genetic mutations identified in NR2E3 and RHO.

“This critical milestone brings us a person move closer in direction of attaining our company’s vision of fostering a long run wherever no 1 feels hopeless in the face of condition,” reported Dr. Shankar Musunuri, Chairman of the Board, Chief Government Officer, and Co-Founder of Ocugen. “Our novel modifier gene therapy platform is a solid illustration of our technique to drug progress.”

Ocugen’s modifier gene treatment system aims to focus on nuclear hormone receptors (NHRs) that control several functions inside the retina, providing it the potential to tackle many unique gene mutations – and, in change, various retinal illnesses – with a solitary merchandise. Regular gene remedy, which transfers a practical version of a non-useful gene into concentrate on cells, targets only one unique gene mutation at a time.

“The aim with OCU400 (AAV-NR2E3) is to offer individuals living with genetic eyesight conditions – people whose eyesight is slowly deteriorating and who have no existing treatment method choices – a explanation to hope,” commented Arun Upadhyay, PhD, Vice President and Head of Exploration & Advancement at Ocugen. “And we’re very delighted to be on this journey with the creator of our modifier gene therapy platform, Dr. Neena Haider from Harvard Health-related School. Collaboration is a mom of innovation, and our do the job with Dr. Haider is actually driving us to see a entire new likely for care.”

The planned Section 1/2 medical review will appraise the safety and evidence-of-idea of OCU400, using unilateral sub-retinal injection (one particular eye per examine subject matter) in 18 patients, with an extension for contralateral eye and long-term security abide by up. Ocugen has now effectively finished producing at industrial scale (200L) to assistance clinical experiments. Ocugen strategies to comply with the Section 1/2 demo with a Section 3 examine, on more assessment of the details.

In between 2019 and 2020, OCU400 was granted four orphan drug disease designations from the Food and drug administration from dealing with four distinctive gene mutation-connected retinal degenerative conditions. In 2021, the European Medicines Company (EMA) granted Ocugen wide orphan medicinal product designation for OCU400 for the treatment of both retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA), which means that, if accredited, OCU400 by by itself could take care of these ailments that are rooted in mutations of far more than 175 distinct genes.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical firm focused on exploring, producing, and commercializing gene therapies to treatment blindness diseases and establishing a vaccine to save life from COVID-19. Our breakthrough modifier gene treatment platform has the probable to deal with multiple retinal diseases with a single drug – “one to many” and our novel biologic products candidate aims to supply superior treatment to sufferers with underserved illnesses these types of as wet age-similar macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-acquiring Bharat Biotech’s COVAXIN™ vaccine prospect for COVID-19 in the U.S. and Canadian markets. For much more information and facts, be sure to pay a visit to www.ocugen.com.

Cautionary Notice on Ahead-On the lookout Statements
This push launch includes forward-hunting statements within just the which means of The Personal Securities Litigation Reform Act of 1995, which are topic to hazards and uncertainties. We may possibly, in some circumstances, use phrases such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other terms that convey uncertainty of future activities or outcomes to establish these forward-hunting statements. These kinds of forward-wanting statements include things like info about qualitative assessments of offered details, likely rewards, expectations for scientific trials, and predicted timing of clinical trial readouts and regulatory submissions, like with regard to our planned Phase 1/2 demo provided in our Investigational New Drug software (IND) to the U.S. Meals and Drug Administration (Fda) for OCU400. This facts entails hazards and uncertainties that could induce true effects to differ materially from those expressed or implied by such statements. Pitfalls and uncertainties include, among the other factors, the uncertainties inherent in investigation and growth, which includes the means to meet anticipated scientific endpoints, commencement and/or completion dates for scientific trials, regulatory submission dates, regulatory approval dates and/or start dates, as effectively as dangers linked with preliminary and interim info, including the possibility of unfavorable new medical trial knowledge and more analyses of existing medical demo details the chance that the benefits of in-vitro scientific tests will not be duplicated in human clinical trials the danger that clinical trial data are subject to differing interpretations and assessments, together with throughout the peer review/publication process, in the scientific group commonly, and by regulatory authorities whether the Food and drug administration will acknowledge our IND submission for OCU400 without any adjustments, or if we are expected to submit additional facts to the Fda in help of our IND submission, the extent and importance of any such variations regardless of whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway obtainable for vaccines in the United States, Canada or other jurisdictions industry demand for COVAXIN™ in the United States or Canada selections by the Food and drug administration or Health and fitness Canada impacting labeling, manufacturing processes, safety and/or other matters that could have an impact on the availability or industrial likely of COVAXIN™ in the United States or Canada, together with advancement of items or therapies by other companies. These and other hazards and uncertainties are far more absolutely explained in our periodic filings with the Securities and Exchange Fee (SEC), which include the threat things explained in the part entitled “Risk Factors” in the quarterly and annual studies that we file with the SEC. Any forward-looking statements that we make in this push release communicate only as of the day of this press release. Except as needed by law, we presume no obligation to update forward-on the lookout statements contained in this push launch no matter whether as a outcome of new information and facts, upcoming occasions or in any other case, immediately after the day of this press release.

Ocugen Get hold of:
Ken Inchausti
Head, Trader Relations & Communications
[email protected]

Make sure you post trader-linked inquiries to: [email protected]