Massachusetts based biotech company, Shire, has submitted an investigational new drug (IND) application to the FDA concerning SHP654 – a gene therapy for the treatment of Hemophilia A.
SHP654 bypasses the traditional factor – based treatment route by inducing liver cells into producing their own source of the blood clotting factor VIII (FVIII), the genetic deficiency of which characterises Hemophilia A. The therapy involves the delivery of a codon-optimized, B-domain deleted FVIII (BDD-FVIII)via a recombinant adeno-associated virus serotype 8 (rAAV8) vector.
Clearing the IND application give Shire the go ahead to administer the therapy in humans. The company plans to initiate a global multi-center study evaluating safety and dosage, given the desired IND outcome. Ultimately, the company aims to bring the therapy to the worldwide market for the long term treatment of hemophilia A through sustained FVIII expression.
Medical Director Paul Monahan commented, ‘Shire is leveraging decades of scientific leadership in hemophilia to advance research in gene therapy for this community. Drawing from our rich heritage, Shire is well equipped to sustainably support the development of gene therapies that aim to advance current standards of care and minimize the burden of this disease. SHP654 uses a proprietary technology platform designed to produce sustained levels of factor similar to the natural mechanisms of the body. Our goal with gene therapy for hemophilia is to uphold the highest standards for safety and efficacy.’
Source: Shire submits investigational New Drug Application to FDA for Gene Therapy candidate SHP654 for treatment of Hemophilia A Press Release