title: Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results.
abstract: Investor interest in gene therapy has increased substantially over the past few years, and the next major catalyst for the field is likely to be Spark Therapeutics's phase III trial for the treatment of visual impairment caused by RPE65 gene mutations (often referred to as Leber congenital amaurosis type 2, or LCA2, but may include other retinal disorders). Analysis of the approach from the basic genetics, underlying visual mechanisms, clinical data, and commercialization considerations helps frame investor expectations and the potential implications for the broader field.
authors.affiliation: Piper Jaffray & Co. , New York, New York.
authors.forename: Joshua
authors.initials: J
authors.lastname: Schimmer
authors.affiliation: Piper Jaffray & Co. , New York, New York.
authors.forename: Steven
authors.initials: S
authors.lastname: Breazzano
mesh_terms: D001709:Biotechnology / Q000191:economics / Q000458:organization & administration; D017326:Clinical Trials, Phase III as Topic; D066264:Datasets as Topic; D055808:Drug Discovery; D015316:Genetic Therapy / Q000191:economics* / Q000639:trends; D006801:Humans; D007449:Investments; D057130:Leber Congenital Amaurosis / Q000235:genetics* / Q000628:therapy*; D009154:Mutation; D019745:cis-trans-Isomerases / Q000235:genetics; D000099178:Retinoid Isomerohydrolase
chemical_list: D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase