Voyager Therapeutics' Stock Jumps As FDA Lifts Clinical Hold For Huntington's Disease Gene Therapy

Today FDA has removed the clinical hold on one more gene therapy study, this time for Voyager Therapeutics Inc's VYGR investigational new drug application for VY-HTT01, a gene therapy candidate for Huntington's disease (HD).
The agency has confirmed that the company may proceed with its planned Phase 1/2 clinical trial.
The decision comes after a comprehensive review of the Chemistry, Manufacturing, and Controls information previously submitted to the FDA.
Voyager plans to initiate VYTAL, a Phase 1/2 clinical trial of VY-HTT01, this year.
VYTAL Phase 1/2 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.
VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, thereby altering disease progression.
It includes adeno-associated virus capsid and a proprietary transgene that harnesses the canonical RNA interference pathway to knock down levels of HTT mRNA selectively.
Earlier today, the FDA removed the clinical hold on uniQure NV's QURE hemophilia B gene therapy program.
Price Action: VYGR shares are moving 12.6% higher at $5.25 on the last check Monday.