Daily Stock Analysis, ONCE, Spark Therapeutics Inc, priceseries

Spark Therapeutics, Inc. engages in gene therapy research, development, manufacturing and clinical trials. The company is developing one-time, life-altering gene therapy treatments to transform the lives of patients and re-imagine the treatment of debilitating diseases. Its initial focus is on treating orphan diseases, and it has demonstrated promising clinical outcomes with its first product candidate targeting rare blinding conditions, which has received both breakthrough therapy and orphan product designation and is in a fully enrolled, pivotal Phase 3 clinical trial. The company also has built a pipeline of product candidates targeting additional blinding conditions, hematologic disorders and neurodegenerative diseases, including a second product candidate targeting another rare blinding condition. Spark Therapeutics was founded by Jeffrey D. Marrazzo, Katherine A. High and Jennifer Wellman on March 13, 2013 and is headquartered in Philadelphia, PA.

Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. Its products include voretigene neparvovec, which is in Phase III clinical trial for the treatment of genetic blinding conditions called inherited retinal diseases caused by non sex-linked, autosomal recessive, or biallelic mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia. The company's products also comprise SPK-7001 that is in Phase 1/2 trial for choroideremia; SPK-9001, which is in Phase 1/2 trial for hemophilia B; and SPK-8011 that is in Phase 1/2 trial hemophilia A. In addition, its product candidates and development programs include SPK-FVIII program to treat hemophilia A; SPK-TPP1 program for the treatment of a form of Batten disease and Huntington's disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and SPK-LHON for treating Leber hereditary optic neuropathy, as well as preclinical programs in development for the treatment of rare genetic blinding conditions, hematologic disorders, and other neurodegenerative diseases. 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. Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.