The research alliance with CHDI builds upon an earlier partnership between CHDI and Sanofi Genzyme and includes financial support from CHDI to help in the preparation and filing of an FDA investigational new drug application. The collaboration also includes the completion of a Phase 1 clinical trial in patients with Huntington’s disease.
Voyager, a company that discovers, develops, and commercializes adeno-associated virus (AAV) gene therapies for severe central nervous system (CNS) disorders, will refund CHDI when VY-HTT01 accomplishes certain commercial milestones.
“Voyager is pleased to collaborate with CHDI to help advance our gene therapy program for Huntington’s disease,” Dr. Steven Paul, MD, president and CEO of Voyager Therapeutics said in a press release. “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.”
Gene therapy commonly uses AAVs to deliver genes into the nuclei of cells where they can be expressed. In some applications, the genes replace nonfunctional gene copies responsible for disease, while in others, the genes delivered generate antibodies or fluorescent proteins to aid research.
Huntington’s disease is a brain disorder caused by a mutation of the huntingtin gene, which produces fragments of toxic protein that build up in the brain. The build-up eventually leads to characteristic uncontrolled movements, as well as cognitive and psychiatric problems.
This disease tends to occur in middle adulthood (mid-30s to mid-40s) and causes a loss of muscle coordination and eventually psychiatric symptoms as well as a loss in memory.
According to the Huntington Disease Society of America, the average lifespan after onset of the disease is 10 to 20 years. There is currently no effective cure for Huntington’s, and Voyager’s VY-HTT01 program, an AAV-mediated gene-silencing therapy, holds promise as a treatment for this debilitating 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. “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.”