GeneTherapy: 2nd generation therapies from AMT, INSERM and ArkTherapeutics

AMT, INSERM and Ark Therapeutics shared goal of achieving an approved gene therapy has led them all to explore what are becoming known as the 2nd generation therapies, and they will be discussing these ideas at the World Gene Therapy Congress in London later this year. Prof Alain Fischer from INSERM will be delivering his recent results in his work …

#AMT’s #HemophiliaB #GeneTherapy gains #OrphanDrug Designation

AMT's  gene therapy program for the treatment of hemophilia B has today gained orphan drug designation in the US from the FDA. AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College …