Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, held in Washington, DC, on May 4-7.
Hemophilia A, also known as factor VIII (FVIII) deficiency, is a genetic disease caused by missing or defective factor VIII, a protein that is involved in clotting processes, estimated to affect 20,000 people in the US alone.
Dimension is a biopharmaceutical company that has been focusing on developing AAV gene therapies for patients with rare diseases associated with the liver. One of their products, DTX201, is designed to deliver FVIII gene expression for long periods, potentially reducing the need for intravenous injections of concentrated recombinant FVIII into hemophilia A patients.
In collaboration with Bayer, Dimension is advancing investigational new drug (IND)-enabling studies for DTX201 in hemophilia A treatment since June 2014, to obtain permission to ship the product to clinical investigators and start Phase 1 clinical trials. Dimension will be responsible for Phase 1/2 clinical trials and developmental activities and, if the treatment proves safe and efficacious, Bayer will conduct a confirmatory Phase 3 trial.
Results from a collaboration with researchers at the University of Pennsylvania (“PENN”) have shown that specific product components, such as the virus capsid, or the enhancer and promoter regions of the FVIII gene, can be selected to further optimize DTX201 effects, including the long-term expression of FVIII. A 30-week study has revealed that FVIII expression levels upon DTX201 administration supported the advancement of the product into IND-enabling studies for treatment of hemophilia A patients. Preclinical studies completed thus far also suggest that DTX201 has the potential to be an effective and well-tolerated treatment for hemophilia A.
“As a leader in hemophilia and evolving new treatment approaches for patients, Bayer has been an ideal partner for us to pursue our program in hemophilia A,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension in a press release. “We continue to benefit greatly from our research collaboration with PENN that has enabled the delivery of high quality in vivo studies to support our Hemophilia A program.”