Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Jun 23 2014

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A.

Under the terms of the agreement, Dimension will receive an upfront payment of $20 million and will be eligible for potential development and commercialization milestone payments of up to $232 million. Dimension will be responsible for all pre-clinical development activities and the Phase 1/2a clinical trial, with funding from Bayer. Depending on the results of the Phase 1/2a clinical trial, Bayer will conduct the confirmatory Phase 3 trial, make all regulatory submissions, and will have worldwide rights to commercialize the potential future product for the treatment of hemophilia A. Dimension is eligible to receive tiered royalties based on product sales.

"Currently available replacement therapies for hemophilia A are often administered intravenously multiple times a week and may be required for life, depending on the severity of a patient's disease," said Thomas R. Beck, M.D., chief executive officer of Dimension Therapeutics. "Gene therapy offers the potential to transform the treatment of hemophilia by inserting a correct version of the faulty gene responsible for the disease. We are proud to partner with Bayer, a leader in the treatment of hemophilia A, to develop a therapy with the potential to significantly change the treatment landscape."

"Bayer is a worldwide leader in the treatment of hemophilia A and we are highly committed to advancing innovative treatment options for patients with hemophilia A," said Prof. Dr. Andreas Busch, member of the Bayer HealthCare executive committee and head of global drug discovery. "We are excited to partner with Dimension Therapeutics to jointly harness the power of gene therapy to drive the development of new long-term options in treating this disease."

Hemophilia is a rare bleeding disorder in which patients produce little or no clotting factor, a protein needed for normal blood clotting, which can cause life threatening bleeding. Hemophilia A is the most common type of hemophilia in the United States, with approximately one in 5,000 males born with the condition.

Dimension's AAV vector technology allows for systemic intravenous administration of the clotting factor gene in vivo, which has been shown in preclinical studies to target the liver, resulting in long lasting expression of FVIII protein at therapeutic levels. Dimension's vectors are enabled by REGENX Biosciences' proprietary NAV® technology.