Bayer

Bayer

First results for Bayer’s Phase I/II trial of AAVhu37.hFVIIIco were reported at the ISTH Virtual 2020 Congress [1]. Outcomes were available for three pairs of patients given different doses (0.5, 1.0 and 2.0 x 1013 GC/kg).

  • The first two had been followed up for 52 weeks; factor VIII activity was 5% – 20% and one patient had used no prophylaxis for 7 months; no adverse events were reported
  • The second pair were followed up for 28 weeks, Both had stopped prophylaxis, with factor VIII activity of 8% – 40%. One had mild elevation of alanine aminotransferase/aspartate aminotransferase which responded to steroids.
  • The third pair were followed up for 10 weeks. Both had stopped prophylaxis for 4 weeks. Both developed mild-to-moderate elevation in alanine aminotransferase/aspartate aminotransferase which responded to steroids.

 

References

  1. Pipe SW, Hay CRM, Sheehan J, et al. First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). Accessed 30 September 2020.