RARE Daily

FDA Removes Clinical Hold on Voyager, Clears IND for Huntington’s Gene Therapy

April 27, 2021

Rare Daily Staff

The U.S. Food and Drug Administration has lifted its clinical hold on Voyager Therapeutics’ application to begin human clinical trials of its experimental gene therapy VY-HTT01 for the rare, neurodegenerative condition Huntington’s disease.

The company is now moving forward this year with a phase 1/2 clinical trial following the agency’s review of the Chemistry, Manufacturing and Controls information Voyager previously submitted. Voyager’s VYTAL phase 1/2 clinical trial is a dose escalation study to evaluate the safety and tolerability of VY-HTT01 in patients with early manifest Huntington’s disease. Secondary endpoints include disease biomarkers and clinical outcome measures.

Huntington’s disease is marked by progressive decline of motor and cognitive functions and a range of behavioral and psychiatric disturbances. Currently there are no disease-modifying therapies approved for the treatment of Huntington’s disease, a fatal, inherited neurodegenerative disease caused by toxic gain-of-function mutations in the huntingtin, or HTT, gene. HD affects more than 30,000 people in the United States alone, with symptom onset commonly appearing between the ages of 30 to 50.

VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, thereby altering disease progression. VY-HTT01 is comprised of an adeno-associated virus capsid (AAV1) and a proprietary transgene that harnesses the canonical RNA interference pathway to selectively knock down levels of HTT mRNA. Preclinical data in non-human primates demonstrated robust and durable reduction of HTT mRNA and protein and widespread distribution of VY-HTT01 across the striatum and cortex, which are core areas of disease pathology.

“Our investigational gene therapy has been designed to achieve broad knockdown of HTT mRNA throughout the brain via a one-time MRI-guided neurosurgical delivery,” said Omar Khwaja, CMO and head of Research and Development at Voyager.

Photo: Omar Khwaja, CMO and head of Research and Development at Voyager.



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