Bayer, Dimension to develop novel gene therapy for hemophilia A

Dimension Therapeutics is focused on developing new adeno-associated virus (AAV) gene therapy treatments for rare diseases.

Bayer HealthCare member of the Executive Committee and head of Global Drug Discovery Andreas Busch said," 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."

As part of the deal, Dimension Therapeutics will get an upfront payment of $20m and will be eligible for potential development and commercialization milestone payments of about $232m.

Dimension will be responsible for all pre-clinical development activities and the Phase I/IIa clinical trial, with funding provided by Bayer.

Dimension Therapeutics chief executive officer Thomas Beck said existing 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.

"Gene therapy offers the potential to transform the treatment of hemophilia by inserting a correct version of the faulty gene responsible for the disease," Beck said.

Based on the results of the Phase I/IIa clinical trial, Bayer will carry out the confirmatory Phase III trial, make all regulatory submissions, and will have worldwide rights to commercialize the potential future product for the treatment of hemophilia A.

Dimension is also eligible to receive tiered royalties based on product sales.