Advanced therapy medicinal product (ATMP)
Development and Regulatory status
Phase III Clinical Trials
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 .
Trial or other data
Jan 20 · No UK trial sites