SPK-8011

Unassigned

New Medicines

Haemophilia A

Information

Advanced therapy medicinal product (ATMP)
Spark Therapeutics
Spark Therapeutics

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes
Yes

Category

Single-dose recombinant adeno-associated viral (rAAV) vector composed of a bio-engineered capsid with liver specific enhanced tropism and a codon-optimized expression cassette that encodes the SQ-FVIII variant of a B-domain-deleted (BDD) human F8 gene.
It affects 1:4,000 to 1:5,000 live male births worldwide [1].
Haemophilia A
Intravenous

Trial or other data

Jan 20 · No UK trial sites