Ophthotech is Advancing an Impressive Portfolio of Cutting-Edge Therapies for Retinal Diseases
Audio version: Ophthotech is a biopharmaceutical company committed to developing therapeutics and gene therapy solutions to treat retinal diseases. The company is aggressively pursuing therapies for orphan conditions like retinitis pigmentosa (RP), Stargardt disease , and Leber congenital amaurosis (LCA), as well as common indications such as wet and dry age-related macular degeneration (AMD). Most impressive, the company is taking on a multi-track strategy that includes retinal gene-therapy development, including delivery of over-sized genes and design of a two-step process of gene knockdown and replacement for autosomal dominant conditions. While these are the scientific challenges that keep most retinal researchers awake at night, Ophthotech thrives on them and sees them as opportunities to meet significant unmet medical needs for people with retinal diseases. Kourous Rezaei, MD, the company’s chief medical officer, says his team’s clinical and drug-development expertise, as...