12/25/2023 0 Comments Spark therapeutics![]() ![]() (Trial identifier NCT03003533)Ībout Roche and Spark Therapeutics gene therapy research in hemophilia A to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A. The Phase 1/2 study, titled “A Gene Transfer Study of SPK-8011 for Hemophilia A,” is being conducted by Spark Therapeutics, Inc. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the U.S., while the European Commission has granted orphan designation to SPK-8011. 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. The remaining 16 participants maintained FVIII expression, of which 12 were followed for over 2 years and demonstrated no apparent decrease in one-stage FVIII activity over time. As previously reported, two participants lost all FVIII expression due to an anti-AAV capsid cellular immune response, unresponsive to immunosuppression. In the safety analysis, 33 treatment-related adverse events (AEs) occurred in 8 participants of which 17 were vector-related, including one serious AE, and 16 were glucocorticoid-related. “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. 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 ![]()
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |