Date: 2014-06-23
Type of information: Development agreement
Compound: adeno-associated virus (AAV) gene therapy
Company: Bayer (Germany) Dimension Therapeutics (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: development commercialisation
Action mechanism: gene therapy. Dimension Therapeutics’ 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 Therapeutics’ vectors are enabled by REGENX Biosciences’ proprietary NAV® technology.
Disease: hemophilia A
Details: * On June 23, 2014, Bayer and Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, have entered into a collaboration for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. Dimension Therapeutics 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 the Phase I/IIa clinical 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.
Financial terms: Under the terms of the agreement, Dimension Therapeutics 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 Therapeutics is eligible to receive tiered royalties based on product sales.
Latest news:
