Sanofi's Genzyme Jumps Into Neuro Gene Therapies With $845 Million R&D Voyager Pharmaceutical Pact
February 11, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based Voyager Therapeutics announced today a collaboration agreement with Boston-based Genzyme Corporation to develop therapeutics for the treatment of severe CNS disorders.
What brings the two companies together is a strong background in gene therapy research. Genzyme has depth in adeno-associated virus (AAV) gene therapy. Voyager’s major platform is a “Product Engine” that focuses on AAV process development, production, optimization and engineering.
The collaboration will focus on multiple gene therapy programs, including for Parkinson’s disease, Huntington’s disease and other CNS disorders. Genzyme will bring in $100 million upfront, including $65 million in cash and a $30 million equity investment in Voyager. Voyager has the possibility of receiving up to $745 million in future milestone payments and royalties.
On Dec. 11, 2014 Voyager announced a collaboration with MassBiologics, part of the University of Massachusetts Medical School (UMMS). The goal of that collaboration is scalable processes for manufacturing recombinant adeno-associated viral (rAAV) vector products. It will combine Voyager’s rAAV production platform with Mass Biologics’ manufacturing expertise and infrastructure.
The company was launched by Third Rock Ventures in February 2014 with $45 million in Series A financing. The company’s initial pipeline is geared toward gene therapy for Parkinson’s disease, a monogenic (single gene) form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) and Friedreich’s ataxia.
In June 2014, Voyager signed a licensing deal with Washington, DC-based REGENX Biosciences, LLC to use RegenRx’s proprietary NAV vectors for gene therapy applications. The NAV vector technology platform also involves the use of various novel AAV vectors to insert therapeutic genes into human tissues.
AAV-mediated gene delivery for potential gene therapy is considered to be very promising. As Melissa Kotterman and David Schaffer write in a Nature Reviews Genetics article “Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval.”
“Through our combined efforts, Voyager and Genzyme are at the forefront of converting the promise of gene therapy into innovative therapies for CNS disorders that make a meaningful difference in patients’ lives,” said Steven Paul, president and chief executive of Voyager in a statement. “Voyager is proud to be collaborating with one of the biotechnology industry pioneers of gene therapy to not only advance development of our lead programs, but also expand our pipeline into new CNS diseases in need of new treatments.”
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