Shire Submits Investigational NDA To FDA For Gene Therapy Candidate SHP654

(RTTNews) - Shire plc (SHPG, SHP.L) announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A.

SHP654 aims to protect hemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression. The IND filing for SHP654 represents the latest step forward for Shire's gene therapy program, which shows promise for both hemophilia A and B populations.

Shire's gene therapy program for hemophilia A uses a recombinant adeno-associated virus serotype 8 (rAAV8) vector, which selectively targets the liver.1,2 It involves the delivery of a functional copy of FVIII to the body's liver to enable its own production of FVIII, rather than relying on a factor-based treatment.