Empowered Patient Podcast
Gene Therapies for Rare Blindness Diseases with Dr. Shankar Musunuri Ocugen
Dr. Shankar Musunuri, Chairman of the Board, CEO, and Co-Founder of Ocugen, is developing gene therapies for rare blindness diseases like retinitis pigmentosa and Stargardt disease, which have significant unmet medical needs. The modifier gene therapy approach aims to reset homeostasis and create a healthy environment for photoreceptors to stabilize vision loss. Ocugen is also working on a gene therapy for dry age-related macular degeneration, which has the potential to address the late-stage geographic atrophy form of the disease.
Shankar explains, "Vision is the most important part for all of us. Many years ago, when we founded Ocugen, it was based on targeting the ophthalmology disease area. We looked into many blindness diseases related to the back of the eye/retina. We have diseases such as AMD- and many people get into that – age-related macular degeneration. Then, there are diseases that are inherited in nature, like retinitis pigmentosa and Stargardt disease. A lot of these diseases impact many families globally. Therefore, our passion for patients has driven that. If there is a significant unmet medical need, we want to take that as a challenge and provide solutions for patients and provide that hope."
"There are two rare blindness diseases. One is retinitis pigmentosa. There are about two million people globally who struggle with it. About 300,000 patients are in the US and EU. Effects in about 100 genes can cause retinitis pigmentosa. With our modifier game-changing gene therapy technology, potentially, our one product, which is currently going through a Phase 3 clinical trial, can target this entire population, rather than building or developing 100 products with the traditional gene therapy gene. The second inherited retinal disease we are working on, Stargardt, affects about 100,000 patients in the US and EU. Similar to RP, there are no therapies today to treat these patients’ significant unmet medical needs."
#Ocugen #CourageousInnovation #GeneTherapy #ModifierGeneTherapy #BlindnessDiseases #RetinitisPigmentosa #GeographicAtrophy #Stargardt
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