Core Viewpoint - Ocugen, Inc. has completed dosing ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410, a novel gene therapy for geographic atrophy (GA), which is a severe form of dry age-related macular degeneration (dAMD) [1][3] Company Summary - Ocugen is a biotechnology company focused on developing gene and cell therapies, biologics, and vaccines [10] - The company aims to address significant unmet medical needs in the field of ophthalmology, particularly for conditions like dAMD and GA [10] Clinical Trial Details - The ArMaDa clinical trial is assessing the safety and efficacy of OCU410, with 51 patients randomized into treatment and control groups [4][7] - OCU410 is designed as a one-time treatment, potentially reducing the burden of frequent injections required by current therapies [4][5] - The trial has shown promising preliminary results, including a 44% slower lesion growth in treated eyes compared to untreated eyes at 9 months [5] Disease Context - dAMD affects approximately 10 million Americans and over 266 million people globally, with GA impacting around 2-3 million people in the U.S. and Europe [1][8] - Current treatment options for dAMD are limited and often involve frequent injections with significant side effects [2][4] OCU410 Mechanism - OCU410 utilizes an adeno-associated virus (AAV) platform to deliver the RORA gene, targeting multiple pathways associated with dAMD pathophysiology [9] - The therapy aims to improve lipid metabolism, reduce oxidative stress, and inhibit inflammation, offering a multifaceted approach to treatment [9]

Core Viewpoint - Ocugen, Inc. has received a positive opinion from the European Medicines Agency (EMA) for the Advanced Therapy Medicinal Product (ATMP) classification of its gene therapy OCU400, which is aimed at treating retinitis pigmentosa (RP) [1][2] Company Developments - The ATMP classification is a significant milestone for Ocugen, allowing the company to advance its clinical and commercial strategy for OCU400, with the goal of providing this therapy to RP patients in the U.S. and Europe by 2027 [2] - The ongoing Phase 3 trial, named liMeliGhT, is currently enrolling 150 participants, divided into two arms based on gene mutations, with a treatment group receiving OCU400 and a control group [4][5] - Ocugen plans to file for Marketing Authorization Application (MAA) in Europe and a Biologics License Application (BLA) in the U.S. simultaneously upon completion of the Phase 3 trial [3] Industry Context - Retinitis pigmentosa affects approximately 310,000 patients across the U.S., EU, and Canada, with no approved treatments currently available to slow or stop the progression of the disease [5] - OCU400 is designed as a gene-agnostic modifier gene therapy based on the NR2E3 gene, which plays a crucial role in retinal health and function [6]

MALVERN, Pa., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. OCU400 is the first gene therapy to enter Phase ...

OCU500 will be administered via inhalation and as a nasal sprayCOVID-19 remains a substantial public health threat in the U.S. and around the worldPhase 1 clinical trial is anticipated to start in 2Q 2025 MALVERN, Pa., Jan. 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. ("Ocugen" or the "Company") (NASDAQ:OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the U.S. Food and Drug Administration (FDA) ha ...

OCU500 will be administered via inhalation and as a nasal sprayCOVID-19 remains a substantial public health threat in the U.S. and around the worldPhase 1 clinical trial is anticipated to start in 2Q 2025 MALVERN, Pa., Jan. 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the U.S. Food and Drug Administration (FDA) h ...

MALVERN, Pa., Jan. 16, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. ("Ocugen" or the "Company") (NASDAQ:OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME). "OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) ...

100% (9/9) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years100% (9/9) of treated evaluable subjects demonstrated improvement or stabilization in mobility testing, which was only performed up to one yearImprovement in visual function was statistically significant (p=0.01, treated vs untreated eyes), regardless of mutation at two yearsFavorable long-term safety and tolerability profile with no serious adverse events ...

MALVERN, Pa., Dec. 11, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen will present at the Oppenheimer Movers in Rare Disease Summit at the Westin Grand Central, New York on Thursday, December 12, 2024. “I have witnessed firsthand the impact that Ocugen’s modifie ...

MALVERN, Pa., Nov. 27, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen will present at NobleCon20 – Noble Capital Markets’ 20th Annual Emerging Growth Equity Conference at Florida Atlantic University, Executive Education Complex, in Boca Raton, FL—taking place De ...

Core Viewpoint - Ocugen, Inc. has received orphan medicinal product designation from the European Medicines Agency (EMA) for its treatment OCU410ST aimed at addressing ABCA4-associated retinopathies, including Stargardt disease, which currently lacks available therapies [1][2]. Group 1: Regulatory Designation and Benefits - The EMA's orphan medicinal product designation provides various advantages for drug developers, including protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity post-approval [3]. - The U.S. Food and Drug Administration (FDA) had previously granted orphan drug designation to OCU410ST in April 2023, highlighting the drug's significance in treating rare diseases [2]. Group 2: Clinical Trial Progress - Dosing for the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease has been completed, with the Data and Safety Monitoring Board (DSMB) recommending progression to Phase 2 due to a favorable safety and tolerability profile [4]. - Preliminary data from the Phase 1 dose-escalation portion of the trial indicated an 84% reduction in atrophic lesion growth in treated eyes compared to untreated eyes after six months [5]. Group 3: Future Plans and Technology - Ocugen plans to pursue an accelerated marketing authorization application (MAA) for OCU410ST, indicating a commitment to expedite the drug's availability [7]. - OCU410ST employs an AAV delivery platform for retinal delivery of the RORA gene, representing Ocugen's innovative modifier gene therapy approach that targets multiple physiological functions [6].