Bayer HealthCare will use Dimension Therapeutics’ adeno-associated virus (AAV) technology under a collaboration by the companies to develop and commercialize a new gene therapy for hemophilia A. The deal could net Dimension up to $252 million.
The AAV vector technology is designed to allow for systemic intravenous administration of the clotting factor gene in vivo. The technology has been shown in preclinical studies to target the liver, resulting in long lasting expression of FVIII protein at therapeutic levels. Dimension’s vectors apply the NAV® technology platform of co-owner ReGenX Biosciences.
NAV includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. The company says NAV provides further advantages over traditional AAV vectors, including greater efficiency for delivering genes in vivo, faster onset of gene expression, high tissue selectivity, and high-titer manufacture.
The technology was discovered in the lab of ReGenX’ scientific founder James M. Wilson, M.D., Ph.D., director of the gene therapy program at the University of Pennsylvania. Dr. Wilson also chairs Dimension’s Scientific and Technical Advisory Board, and serves as editor-in-chief of Human Gene Therapy, a journal published by GEN publisher Mary Ann Liebert Inc.
In October, Dimension entered into an exclusive license and collaboration with ReGenX that gave Dimension preferred access to NAV vector technology and rights in ReGenX product programs in multiple rare disease indications.
“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,” Thomas R. Beck, M.D., Dimension’s CEO, said in a statement.
Dimension will receive $20 million upfront from Bayer, and is eligible for up to $232 million in payments tied to development and commercialization milestones.
Dimension will oversee all preclinical development activities and the Phase I/IIa clinical trial, with funding from Bayer. Depending on results of the Phase I/IIa trial, Bayer will advance the gene therapy to a Phase III trial, make all regulatory submissions, and hold worldwide rights to commercialize the potential treatment for hemophilia A. Dimension is also eligible for tiered royalties based on product sales.
Separately, Dimension said it closed on a $30 million financing, with new investor OrbiMed joining existing investor Fidelity Biosciences in the round. Fidelity—whose partners include Beck and Dimension Chairman Ben Auspitz—joined ReGenX in launching the company last year, focusing on developing gene therapies for rare diseases.
Dimension continues to advance a wholly owned hemophilia B program towards clinical development. The program has progressed past candidate selection toward IND-enabling work in advance of a submission and Phase I trial.
