title: Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results.
abstract: Spark Therapeutics recently reported positive phase III results for SPK-RPE65 targeting 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), marking an important inflection point for the field of gene therapy. The results highlight the ability to successfully design and execute a randomized trial of a gene therapy and also reinforce the potentially predictive nature of early preclinical and clinical data. The results are expected to pave the way for the first approved gene therapy product in the United States and should sustain investor interest and confidence in gene therapy for many approaches, including retina targeting and beyond.
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: D017326:Clinical Trials, Phase III as Topic / Q000191:economics*; D015316:Genetic Therapy / Q000009:adverse effects / Q000191:economics* / Q000379:methods; D006801:Humans; D007449:Investments / Q000191:economics*; D057130:Leber Congenital Amaurosis / Q000235:genetics / Q000628:therapy*; D019745:cis-trans-Isomerases / Q000235:genetics; D000099178:Retinoid Isomerohydrolase
chemical_list: D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase