Bayer HealthCare and Dimension Therapeutics have entered into a collaboration for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. 

Under the terms, the biotechnology firm based in Cambridge, Massachusetts, USA will be responsible for all pre-clinical development activities and the Phase I/IIa clinical trial, with funding from Bayer.

Depending on the results of this trial, Bayer will conduct 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 Therapeutics, which is focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, will be eligible to receive tiered royalties based on product sales.

The US company'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.

The vectors are enabled by proprietary NAV technology owned by Regenex Biosciences. Founded by Fidelity Biosciences and Regenex in 2013, Dimension Therapeutics is funded by Fidelity Biosciences and OrbiMed.

Bayer's hematology business owns an approved treatment for hemophilia A and compounds in development for hemophilia A, sickle cell anemia, and other blood and bleeding disorders.