Voyager Therapeutics Provides Regulatory Update on VY-HTT01 Program
- Category: DNA RNA and Cells
- Published on Tuesday, 13 October 2020 11:59
- Hits: 1190
CAMBRIDGE, MA, USA I October 12, 2020 I Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced that it has received feedback from the U.S. Food and Drug Administration (FDA) on the Investigational New Drug (IND) submission for VY-HTT01 for the treatment of Huntington’s disease. Voyager submitted the IND for VY-HTT01 in September 2020 and has been notified that the IND was placed on clinical hold pending the resolution of certain chemistry, manufacturing and controls (CMC) matters. The Company expects to receive specific feedback from the FDA on these matters within 30 days and plans to work closely with the agency to resolve them and promptly begin the clinical evaluation of VY-HTT01.
Huntington’s disease is a fatal, inherited neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain, resulting in a range of motor, cognitive, and psychiatric symptoms. Symptom onset typically occurs between the ages of 30 to 50 and the average length of survival after clinical diagnosis is typically 10 to 20 years. Huntington’s disease is the most common monogenic neurological disorder in the developed world and is estimated to affect over 40,000 people in the U.S. Currently, there are no approved treatments targeting the underlying cause of the disease.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyager’s wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinson’s disease, Huntington’s disease, Friedreich’s ataxia, and other severe neurological diseases. For more information on Voyager Therapeutics, please visit the company’s website at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.
SOURCE: Voyager Therapeutics