Benzinga | Apr 26, 2021 09:32AM EDT

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 (NASDAQ: 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 (NASDAQ: QURE) hemophilia B gene therapy program.

* Price Action: VYGR shares are moving 12.6% higher at $5.25 on the last check Monday.