Voyager Therapeutics & CHDI Foundation Collaborate to Develop Novel Gene Therapy for Huntington’s Disease

Voyager Therapeutics, Inc. recently announced a research collaboration with CHDI Foundation, Inc. (CHDI) to advance Voyager’s VY-HTT01 program, an adeno-associated virus (AAV)-mediated gene-silencing therapy for Huntington’s disease. The collaboration builds upon a previous collaboration between CHDI and Sanofi Genzyme and includes funding from CHDI to help support preparation for and filing of an investigational new drug application, as well as completion of a Phase I clinical trial. CHDI will be reimbursed for its support of Voyager’s program upon VY-HTT01 achieving certain commercial milestones.

“Voyager is pleased to collaborate with CHDI to help advance our gene therapy program for Huntington’s disease,” said Steven Paul, MD, President and Chief Executive Officer of Voyager Therapeutics. “The effort and expertise that Voyager and Sanofi Genzyme continue to commit to the VY-HTT01 program, now further strengthened with CHDI’s extensive experience in Huntington’s disease research, puts us in a strong position to advance the clinical development of a potential disease-modifying medicine for patients suffering from Huntington’s disease.”

“CHDI is committed to identifying and facilitating the development of a diverse pipeline of therapeutic strategies for Huntington’s disease,” said Robi Blumenstein, President of CHDI Management, Inc. “Voyager is a leader in gene therapy, particularly for central nervous system diseases, and we are confident that their expertise will allow us to further extend the important progress that Sanofi Genzyme has already made in our collaboration.”

Huntington’s disease is an inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG repeat to occur many more times than normal. Each child of a parent with a mutation in the huntingtin gene has a 50% chance of inheriting the mutation. As a result of carrying the mutation, an individual’s brain cells degenerate leading to behavioral, cognitive, and motor impairments that, over the course of the disease, significantly reduce the individual’s quality of life and ultimately cause death within 15 to 25 years of overt clinical onset. It is estimated that around one person in 10,000 carries the mutated huntingtin gene. There are currently no therapeutics approved that slow the progression of Huntington’s disease.

CHDI Foundation, Inc. is a privately funded nonprofit biomedical research organization that is exclusively dedicated to rapidly developing therapies that slow the progression of Huntington’s disease. As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. CHDI scientists work closely with a network of more than 700 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. For more information, visit www.chdifoundation.org.

Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the CNS. Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. The company’s pipeline is focused on severe CNS diseases in need of effective new therapies, including advanced Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, Huntington’s disease, spinal muscular atrophy (SMA), frontotemporal dementia, Alzheimer’s disease, and severe, chronic pain. Voyager has broad strategic collaborations with Sanofi Genzyme, the specialty care global business unit of Sanofi, and the University of Massachusetts Medical School. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference, and neuroscience, Voyager Therapeutics is headquartered in Cambridge, MA. For more information, visit www.voyagertherapeutics.com.