Veeral Sheth, MD, is joined by Rich Small, the CEO of Neurotech, to discuss the historic approval of revakinagene taroretcel (ENCELTO) a groundbreaking encapsulated cell therapy for macular telangiectasia type 2 (MacTel). This approval marked the first and only FDA–approved treatment for MacTel, a neurodegenerative disease of the retina that can cause progressive and irreversible vision loss.
Speaking with Sheth, Small recounted the long journey of Neurotech, which began in 2007, emphasizing the perseverance required to bring ENCELTO to market. He detailed his transition from Chief Financial Officer to CEO in 2016 and highlighted his team’s commitment, many of whom have been with the company for over a decade.
The conversation explored the scientific foundation of ENCELTO, which uses genetically modified cells to release neurotrophic factors that protect photoreceptors in the retina. Unlike previous treatments that relied on best corrected visual acuity (BCVA) as a clinical endpoint, ENCELTO’s approval was enabled by advancements in imaging technology that allowed for direct measurement of photoreceptor health. Sheth and Small discussed the ideal patient profile for ENCELTO, noting that early intervention is key to preserving vision in MacTel patients.
With ENCELTO’s approval secured, Small indicated the next step is widespread adoption, introducing ENCELTO.com as a patient service hub designed to streamline diagnosis, treatment, and access to care.
#Ophthalmology #Podcast #RareDisease
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