Technical Analysis for ONCE - Spark Therapeutics, Inc.

Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. The company is developing SPK-RPE65, which is in Phase III clinical trial for the treatment of inherited retinal dystrophies, a group of rare blinding conditions caused by non-sex linked, or autosomal recessive, mutations in the RPE65 gene. It is also developing SPK-CHM for the treatment of choroideremia; and preclinical programs in development for the treatment of hemophilia A and neurodegenerative diseases, as well as a pipeline of product candidates targeting rare blinding conditions. The company has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B; and development and manufacturing collaboration with Genable Technologies Limited for the manufacture and provision of development advice in the development of Genable's therapeutic product candidate, GT038, to treat rhodopsin-linked autosomal dominant retinitis pigmentosa. The company was founded in 2013 and is based in Philadelphia, Pennsylvania.
Biology Emerging Technologies Gene Therapy Molecular Biology Medical Genetics Blindness Neurodegenerative Diseases Retinitis Pigmentosa Genetic Diseases Gene Delivery Treatment Of Hemophilia Gene Therapy Products Hemophilia A Choroideremia Genetic Disorder Hemophilia B