Date: 2016-06-13
Type of
information: Results
phase: preclinical
Announcement: results
Company: Spark Therapeutics (USA - PA)
Product: SPK-8011
Action
mechanism:
- gene therapy. SPK-8011 was developed using Spark's proprietary technology platform and uses Spark200, a novel bio-engineered adeno-associated virus (AAV) capsid optimized for more efficient transduction of human hepatocytes, and contains an optimized B-domain deleted FVIII expression cassette. Spark retains global commercialization rights to its SPK-FVIII program for hemophilia A.
Disease: hemophilia A
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details:
Latest
news:
- • On June 13, 2016, Spark Therapeutics highlighted preclinical results from SPK-8011, the lead investigational candidate in its SPK-FVIII program for hemophilia A. Data presented at the meeting of the American Society of Gene and Cell Therapy in May demonstrated strong preclinical data, specifically FVIII expression in the 15% to 35% range in preclinical models at clinically relevant doses of 2 x 1012 vg/kg. Spark Therapeutics is now on track to progress SPK-8011 for hemophilia A into a Phase 1/2 clinical trial in the second half of 2016, and to potentially see the first human data from this program in the first half of 2017.
Is
general: Yes
