Novel Gene Therapy in Development for Hemophilia A

September 10, 2014
Hemophilia Reports, August 2014, Volume 1, Issue 2

Bayer Healthcare and Dimension Therapeutics recently announced a partnership to advance innovative treatment options for patients with hemophilia A.

Patients diagnosed with hemophilia A could have a new way to treat their condition in the future, following the announcement of a partnership between Bayer Healthcare and Dimension Therapeutics to develop a “novel gene therapy.”

A press release from Bayer said the collaboration should provide considerable benefits in the future. “Bayer is a worldwide leader in the treatment of hemophilia A and we are highly committed to advancing innovative treatment options for patients with hemo."philia A,” said professor Andreas Busch, MD, a member of the Bayer HealthCare Executive Committee and Head of Global Drug Discovery.

Busch added, “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.”

Representatives from Dimension said the new drug could provide a whole new method of treatment for people with hemophilia. “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,” Thomas R. Beck, MD, Dimension’s CEO said. “Gene therapy offers the potential to transform the treatment of hemophilia by inserting a correct version of the faulty gene responsible for the disease.”

Being able to tackle this potential breakthrough with Bayer was something Beck said his company was also looking forward to. “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.”

The initial agreement allows for a $20 million payment up front to Dimension. The total financial number could increase to as much as $232 million depending on development and commercialization milestone payments. Dimension will be tasked with handling the pre-clinical development activities and the Phase I/IIa clinical trials which will be funded by Bayer according to the release.

Bayer, depending on the success of those trials, would then conduct the Phase III trial, make all regulatory submissions and would have worldwide rights to sell the eventual product with Dimension receiving tiered royalties based on sales.