Gardian

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2024 Annual Meeting

Retrieved on: 
Friday, April 26, 2024
Stargardt disease, The Association, Safety, Retinitis pigmentosa, Armada, NR2E3, Gardian, Research, Doctor of Philosophy, Vaccine, Retina, Research and development, Patient, Geographic atrophy, Clinical trial, Annual general meeting, ARVO, Pharmaceutical industry

MALVERN, Pa., April 26, 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, and vaccines, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410 for the treatment of geographic atrophy (Phase 1/2 ArMaDa clinical trial), and OCU410ST for the treatment of Stargardt disease (Phase 1/2 GARDian clinical trial) at The Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting in Seattle, WA from May 5-9, 2024.

Key Points: 
  • MALVERN, Pa., April 26, 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, and vaccines, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410 for the treatment of geographic atrophy (Phase 1/2 ArMaDa clinical trial), and OCU410ST for the treatment of Stargardt disease (Phase 1/2 GARDian clinical trial) at The Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting in Seattle, WA from May 5-9, 2024.
  • “With three gene therapies to treat blindness diseases currently in the clinic, now is an exciting time for Ocugen and the patients who can potentially benefit from our first-in-class modifier gene therapy platform,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Radius.
  • “We look forward to sharing more about the scientific foundation of our programs and providing clinical updates with industry leaders during ARVO.”
    Ocugen’s presence at ARVO 2024 includes:
    Title: OCU400 Nuclear Hormone Receptor-Based Gene Modifier Therapy: Safety and Efficacy from Phase 1/2 Clinical Trial for Retinitis Pigmentosa Associated with NR2E3 and RHO Mutations
    Presenter: Arun Upadhyay, PhD, Chief Scientific Officer, Head of Research & Development, Ocugen
    Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally.

Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease

Retrieved on: 
Monday, April 1, 2024
Stargardt disease, Gardian, Patient, Cohort, Șaeș, Clinical trial, MPH, Safety, DSMB, FACS, Vaccine, FDA, Pharmaceutical industry

Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.

Key Points: 
  • Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.
  • An additional three patients will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.
  • “The DSMB has recommended moving forward to medium dose for dosing subjects with Stargardt disease,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial.
  • Phase 1 is a multicenter, open-label, dose ranging study consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)].

Ocugen, Inc. Announces Dosing Completion of Subjects with Stargardt in Cohort 1 of Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST

Retrieved on: 
Thursday, February 22, 2024
Doctor of Philosophy, MD, Consultant, Gardian, Vaccine, Safety, University, Stargardt disease, Patient, Clinical trial, United, Disease, Retina, Pharmaceutical industry

Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States alone.

Key Points: 
  • Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States alone.
  • “This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen.
  • “Although Stargardt is one of the most common inherited retinal diseases, there remains no treatment option to address this condition.
  • In the first cohort, 3 subjects received 200L single subretinal administration of the low dose (3.75x1010 vg/mL) of OCU410ST.

Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST—Modifier Gene Therapy—for Stargardt Disease

Retrieved on: 
Friday, November 10, 2023
Stargardt disease, Gardian, Safety, Retina, RAR-related orphan receptor, RORA, Patient, Vaccine, Oxidative stress, Consultant, MD, NHR, Doctor of Philosophy, AAV, Inflammation, Receptor (biochemistry), Pharmaceutical industry, Stargardt, Research

“There is a significant unmet medical need for the approximate 35,000 patients in the U.S. living with Stargardt disease,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen.

Key Points: 
  • “There is a significant unmet medical need for the approximate 35,000 patients in the U.S. living with Stargardt disease,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen.
  • OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene.
  • “It is important and exciting to pursue novel therapies for untreatable blinding diseases,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas.
  • “Initiation of this trial program investigating a new mechanism of action for the treatment of Stargardt disease is inspiring and brings hope to patients and families.”