Ocugen Sees Ray of Light with Gene Therapy for Retinitis Pigmentosa

Ocugen, which has faced a string of delays with its Bharat Biotech-partnered vaccine, reported some good news today.

The company, based in Malvern, Pennsylvania, reported that the independent Data and Safety Monitoring Board (DSMB) involved in the Phase I/II trial of OCU400, a gene therapy in development for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients. The trial is evaluating the modifier gene therapy OCU400 for RP caused by mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes. Ocugen recently dosed the first patient in the study. The DSMB recommended the company continue enrolling the remaining subjects in the current cohort at the target dose level.

RP is a family of rare eye diseases affecting the retina. RP results in the retina breaking down over time, causing vision loss. Symptoms typically begin in childhood, but the majority of patients eventually lose most of their vision. The most common early symptom is loss of night vision.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Add a Comment

Your email address will not be published. Required fields are marked *