Advanced therapy medicinal product (ATMP)
Development and Regulatory status
Jun 19 · Spark Therapeutics initiated an observational phase III, six-month run-in study (NCT03432520) with an estimated primary completion date of December 2022 
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 .