#Spark therapeutics trial#

Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. “This Phase 1/2 study for SPK-8011 demonstrates our commitment to following the science to develop gene therapies for hemophilia A that demonstrate safety, predictability, efficacy and durability at the lowest effective dose and with an optimal immunomodulatory regimen.” “We are thrilled that the New England Journal of Medicine chose to publish data from our Phase 1/2 study of investigational SPK-8011 and highlight its potential for patients living with hemophilia A,” said Gallia Levy, M.D., Ph.D., Chief Medical Officer, Spark Therapeutics. The updated analysis (cutoff May 3, 2021) of all 18 study participants, following the initial data presentation at the International Society of Thrombosis and Hemostasis (ISTH) 2021 Virtual Congress in July, demonstrated a 91.5% reduction (95% CI: ) in annualized bleed rate (ABR) and a 96.4% reduction (95% CI: ) in annualized number of FVIII infusions. The latest results were published online in the New England Journal of Medicine (NEJM).

The study found that, at a median efficacy follow-up of 33.4 months (range, 3.7-47.6), 16 of 18 study participants had sustained factor VIII (FVIII), which permitted prophylaxis cessation and reduction in bleeding episodes.

#Spark therapeutics trial#

17, 2021 (GLOBE NEWSWIRE) - Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced data from its Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A.

Data from Spark Therapeutics’ Phase 1/2 gene therapy study published in New England Journal of Medicine