Promising Results Show OCU400 Gene Therapy Could Treat Retinitis Pigmentosa and Leber Congenital Amaurosis with Positive Safety and Efficacy!

Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing gene and cell therapies, biologics, and vaccines, has announced promising preliminary data from their Phase 1/2 trial assessing the safety and efficacy of OCU400 for patients with retinitis pigmentosa (RP), Leber Congenital Amaurosis (LCA), or mutations in the NR2E3 and Rhodopsin (RHO) genes.

These preliminary results provide promising evidence that OCU400 may be a potential gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration, and could be a major advance in the treatment of these conditions.

Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen, was thrilled to see the promising preliminary results of the company’s novel modifier gene therapy approach. This is the first clinical validation of the platform, with patient responses across multiple genetic mutations indicating that OCU400 has the potential to bring much-needed relief to those living with debilitating blindness diseases.

This Phase 1/2 trial is an exciting open-label, dose ranging study that is being conducted across multiple centers. So far, 18 participants have been enrolled, ranging in age from 18 to 77 years old and spanning a variety of RHO and NR2E3 gene mutations.

To make the trial even more comprehensive, we are now expanding it to include LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO, and CEP290 mutations. We can’t wait to see the results!

In an exciting clinical trial, 7 participants with severe vision impairment due to Retinitis Pigmentosa associated with RHO and NR2E3 gene mutations were injected with either a low (1.66 x 1010 vg/mL) or medium (3.33 x 1010 vg/mL) dose of OCU400, split between Cohort 1 and 2 respectively. Preliminary data analysis from 9-month follow-up of 3 subjects from Cohort 1 and 6-month follow-up of 4 subjects (including 1 from Cohort 1 and 3 from Cohort 2) has been evaluated.

The safety profile of OCU400 was favorable, and treatment with it resulted in visible improvements in both multi-luminance mobility testing (MLMT) and best corrected visual acuity assessment (BCVA). Patients reported an overall satisfaction with the results of the treatment.

The results of the trial of OCU400, a potential therapeutic solution for Retinitis Pigmentosa, have far exceeded expectations. David Birch, PhD, Scientific Director of the Retina Foundation of the Southwest, who served as the principal investigator of the study, has been delighted by the improvements in visual function seen in the advanced stages of the retinal disease. He is hopeful that this treatment could restore independence to those affected by RP, in addition to restoring their vision.

The promising results of the Phase 1/2 clinical trial have given us cause for optimism in the potential of modifier gene therapy technology to address the unmet medical needs of patients with RP and LCA. With a favorable safety profile and positive efficacy signals, we are excited to observe the longer-term data and possibly move forward with Phase 3 trials in the U.S. and EU.

Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen, expresses his enthusiasm for the paradigm-shifting possibilities of this technology.

Ocugen is dedicated to providing a potential treatment option to RP and LCA patients, and is diligently tracking the long-term safety and efficacy data from the treated patients to ensure that OCU400 advances in development. With this promising new option, Ocugen is committed to bringing hope to those suffering from these eye conditions.

Ocugen has partnered with CanSinoBIO to make their Phase 1/2 trial of OCU400 a success, with the latter providing all the necessary CMC development and clinical supplies.

About Modifier Gene Therapy

Ocugen’s modifier gene therapy platform is pioneering a new approach to treating retinal diseases such as RP, LCA, Stargardt disease and dry AMD. Our platform utilises the power of NHRs, master gene regulators that can restore homeostasis in the retina.

Unlike single-gene replacement therapies, our platform is designed to address multiple genetic mutations in one product, and to address complex diseases caused by imbalances in multiple gene networks. Our three modifier gene therapy programs, OCU400 (RP, LCA), OCU410 (dry AMD) and OCU410ST (Stargardt disease) are specifically designed to fulfil unmet medical needs and revolutionise the way retinal diseases are treated.

About OCU400

OCU400 is a revolutionary gene therapy product that could offer relief to patients suffering from inherited retinal diseases. It works by using the NHR gene, NR2E3, to reset altered cellular gene-networks, restoring balance and potentially improving retinal health and function.

It is a gene-agnostic modifier therapy that regulates a variety of physiological functions in the retina, such as photoreceptor development, metabolism, phototransduction, inflammation and cell survival. OCU400 could be a game changer for those living with inherited retinal diseases, allowing them to reclaim their sight and improve their quality of life.

About Ocugen, Inc.

Ocugen, Inc. is a pioneering biotechnology company on a mission to revolutionize healthcare. We are committed to discovering, developing, and commercializing innovative gene and cell therapies, biologics, and vaccines that can profoundly improve the lives of patients around the world.

Our cutting-edge modifier gene therapy platform has the potential to treat multiple retinal diseases with one product, and we are also researching infectious and orthopedic diseases to address unmet medical needs. Through our courageous innovation, we strive to make a meaningful difference in the lives of those we serve.

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