Setting off with Voyager Therapeutics

CAMBRIDGE, Mass.—Third Rock Ventures, LLC has announced the launch of Voyager Therapeutics, a new gene therapy company that will focus on the development of treatments for fatal and debilitating central nervous system (CNS) diseases. The company was founded with a $45 million Series A financing, and will hit the ground running with several preclinical and clinical product programs.

“CNS disorders are one of the largest areas of unmet medical need,” Philip Reilly, M.D., J.D., interim chief medical officer of Voyager and venture partner at Third Rock, commented in a statement. “By expressing the right amount of protein in the right cell at the right time or knocking down a gene to decrease levels of a disease-causing protein, we believe our gene therapy approach is uniquely suited to targeting these devastating diseases and has the potential to fundamentally change the face of treatment for many patients.”

Voyager Therapeutics will focus on an adeno-associated virus (AAV) approach to gene therapy, and will be investing in areas such as vector optimization and engineering, dosing techniques and process development and production. As the company notes on its website, it is “investing in advancing the science and technology around the three key elements of AAV vectors: capsid, promoter and transgene.” The new company’s founders include experts and leaders in the fields of neuroscience, AAV gene therapy and expressed RNA interference.

The company’s pipeline includes VY-AADC01, a compound indicated for the treatment of Parkinson’s disease. The product candidate is currently in a Phase 1b study with Voyager’s collaborators at the University of California, San Francisco (UCSF). Voyager also has two candidates in the preclinical stage, one for the treatment of a monogenic form of amyotrophic lateral sclerosis and one for the treatment of Friedreich’s ataxia.

“We believe the time is right for gene therapy, and we have assembled the expertise, technology and strategies to convert the promise of AAV gene therapy into meaningful treatments for patients,” Mark Levin, interim CEO of Voyager and partner at Third Rock, said in a press release regarding the new company. “Our world-class founders have driven significant advances in their respective fields, and the combination of their deep clinical and scientific knowledge, our experienced management team and Third Rock’s investment positions Voyager to deliver breakthrough therapies to patients suffering from devastating CNS disorders.”

Voyager Therapeutics has a broad strategic collaboration in place with the University of Massachusetts Medical School (UMMS) and has established license and other agreements with UMMS, UCSF and Stanford University for access to pertinent technology and data. The partnerships and agreements include certain rights to intellectual property related to expressed RNA interference technology discovered at UMMS, clinical data related to the VY-AADC01 Parkinson’s disease program from UCSF and certain rights to AAV intellectual property developed at Stanford University. Voyager Therapeutics also noted that it has “strong relationships with leading patient education and research foundations such as the Friedreich’s Ataxia Research Alliance and The Michael J. Fox Foundation for Parkinson’s Research.”

SOURCE: Voyager Therapeutics press release