Profile: Spark Therapeutics Inc (ONCE.OQ)
17 Jul 2018
Spark Therapeutics, Inc. (Spark), incorporated on March 13, 2013, is a gene therapy company. The Company focuses on treating orphan diseases. It has a pipeline of product candidates targeting multiple rare blinding conditions, hematologic disorders and neurodegenerative diseases. Its pipeline includes a product candidate targeting choroideremia (CHM), which is in a Phase I/II clinical trial and a product candidate for hemophilia A, which is in a Phase I/II clinical trial. The Company's product investigational candidate, voretigene neparvovec, is intended to treat a genetic blinding condition or inherited retinal disease (IRD) caused by non sex-linked, or autosomal recessive, biallelic mutations in the RPE65 gene. The Company has received orphan product designation for voretigene neparvovec for the treatment of RPE65-mediated IRD in both the United States and the European Union.
The Company's lead product candidate, SPK-CHM, is indicated for the treatment of CHM. The Company has received orphan product designation for SPK-CHM for the treatment of CHM in both the United States and the European Union. The programs targeting hematologic disorders include SPK-FIX, for which the lead product candidate, SPK-9001, is in a Phase I/II clinical trial for the treatment of hemophilia B in collaboration with Pfizer Inc., as well as SPK-FVIII for the treatment of hemophilia A. SPK-9001 has received both breakthrough therapy and orphan drug designation by the United States Food and Drug Administration (FDA). In its SPK-FVIII program for the treatment of hemophilia A, the Company has initiated a dose-escalating Phase I/II clinical trial for its lead product candidate, SPK-8011. The Company is developing neurodegenerative disease product candidates that are intended to address Tripeptidyl-peptidase 1 (TPP1) deficiency, which is a form of Batten disease and Huntington's disease, among others. The Company has received orphan product designation in the United States for SPK-TPP1 for the treatment of ceroid lipofuscinosis neuronal 2 (CLN2) disease (neuronal ceroid lipofuscinosis (NCL)) caused by TPP1 deficiency. The Company has other neurodegenerative disease programs in preclinical development.
The Company competes with bluebird bio, Inc., Annapurna Therapeutics, Applied Genetic Technologies Corporation, Asklepios BioPharmaceutical, Inc., Audentes Therapeutics, Inc., Avalanche Biotechnologies, Inc., AveXis, Inc., Abeona Therapeutics Inc., Dimension Therapeutics, Inc., GenSight Biologics SA, Horama SAS, Lysogene SAS, MeiraGTx Limited, NightstaRx Ltd., ReGenX Biosciences, LLC, Voyager Therapeutics, Inc., Second Sight Medical Products, Inc., Retina Implant AG, Novelion Therapeutics, Inc., NightstaRx Ltd., Pfizer Inc., Shire, PLC, Sangamo BioSciences, Inc., Freeline Therapeutics, BioMarin Pharmaceutical Inc., Bayer HealthCare, uniQure N.V., Telethon Institute, Biogen Inc., Alnylam Incorporated, Novo Nordisk A/S and Roche Holding AG.
Spark Therapeutics Inc
3737 Market St Ste 1300
PHILADELPHIA PA 19104-5543
Company Web Links
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