OCU410ST


Associated tags: Stargardt, Oxidative stress, AAV, Stargardt disease, Inflammation, Receptor (biochemistry), NHR, Safety, Pharmaceutical industry, Gardian, Vaccine, Clinical trial, Patient, MPH, Șaeș, DSMB, FACS, Cohort, FDA, Doctor of Philosophy, MD, Consultant, University, United, Disease, Retina

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.