Ocugen

Ocugen's Dr. Shankar Musunuri to discuss gene therapy advancements at the 2024 Maxim Healthcare Virtual Summit, Oct. 15-17.

Ocugen presented OCU400 phase 1/2 study results at EURETINA, showing excellent safety and tolerability in RP patients. The therapy received FDA and EMA orphan drug designations, and expanded access approval. Ocugen is enrolling for OCU400 phase 3, targeting broader RP indications. Additionally, OCU410 (RORA-based) is in phase 1/2 for geographic atrophy and Stargardt disease, with positive safety profiles.

Ocugen announced the FDA lifted the clinical hold on OCU200, a DME treatment, with a Phase 1 trial planned. OCU200 targets DME mechanisms and could benefit non-responders to current therapies. The study will assess safety and combination with anti-VEGF.

Ocugen announced the FDA lifted the clinical hold on OCU200, a DME treatment, with a Phase 1 trial planned. OCU200 targets DME's underlying mechanisms, potentially benefiting non-responders to current therapies. The study will assess safety and is open-label with dose-ranging.

Ocugen's OCU400, a gene-agnostic gene therapy for inherited retinal diseases (IRD), has entered phase 3 clinical trial (liMeliGhT; NCT06388200). Unlike traditional targeted gene therapies, OCU400 aims to modify the underlying disease pathway affected by various mutations in RP patients, potentially benefiting a wide range of gene mutations. The phase 3 trial will enroll 150 patients, evaluating OCU400's safety and efficacy, with a primary efficacy endpoint of improved mobility under different light conditions.

CGTLive’s Weekly Rewind highlights 5 gene and cell therapy advances: CARTITUDE-4 trial outcomes, Ocugen’s phase 3 gene therapy, Anixa’s investigational new drug clearance, Epic Bio’s technology applications, and CSF analysis reductions in NAA levels.

Ocugen's CEO, Dr. Shankar Musunuri, will present at the 2024 Cell & Gene Meeting on the Mesa, discussing Ocugen’s modifier gene therapy platform and clinical trials for retinitis pigmentosa, geographic atrophy, and Stargardt disease. The event is held October 7-9, 2024, in Phoenix, AZ.

Ocugen is enrolling patients in its OCU400 phase 3 study for a broader retinitis pigmentosa (RP) indication, the first of its kind. OCU400, a gene-agnostic therapy, showed safety and efficacy in a phase 1/2 trial, with 60% of patients responding to a luminance-dependent navigation assessment. The therapy has received orphan drug and regenerative medicines advanced therapy designations.

CGTLive®’s Weekly Rewind highlights 5 gene and cell therapy advances, including FDA actions, research, and interviews. Notable mentions include a gene therapy for Usher syndrome type 1B and a discussion on OCU400 for IRDs.

Dr. Shankar Musunuri, Ocugen's Chairman, CEO, and Co-founder, will speak at the U.S.-India Biotechnology Cooperation workshop in New Delhi, September 9-11, 2024. He will participate in a panel on 'Persistent Challenges: Right-Sizing Regulations and Setting Standards Beyond the Biomedical Sphere' on September 10. The event aims to identify collaboration opportunities in biotechnology between the U.S. and India, organized by the U.S. National Academies of Sciences, Engineering, and Medicine and the Indian National Science Academy.