Voyager Therapeutics has a new candidate for the treatment of Huntington’s disease. The selected gene therapy is called VY-HTT01 and aims to prevent the production of protein from the mutant huntingtin gene (HTT).
The compound is now undergoing preclinical studies of drug characteristics and toxicology as the company plans to file an investigational new drug (IND) application — necessary to allow the treatment to be tested in patients — with the U.S. Food and Drug Administration in 2018.
The treatment makes use of an adeno-associated virus (AAV) to deliver the gene therapy, which acts to block the production of huntingtin protein by controlling a natural mechanism called RNA interference. Various strains of this harmless virus are often used in gene therapy applications, but each treatment needs to adapt the viral carrier to the particular treatment situation.
“We systematically and thoroughly optimized the AAV capsid and transgene before selecting VY-HTT01 as the lead clinical candidate for Huntington’s disease with scientists at Sanofi Genzyme, as part of our research alliance,” Dinah Sah, PhD, chief scientific officer at Voyager, said in a press release.
“In preclinical models, a single administration of VY-HTT01 was well-tolerated and resulted in robust and widespread knockdown of HTT messenger RNA in disease-relevant regions of the non-human primate central nervous system,” Sah added.
The studies showed the treatment suppressed mutant huntingtin production by more than 50 percent, researchers said.
The company also performed studies to demonstrate that the treatment could be produced in quantities and of the quality required to enter clinical trials.
CHDI, a nonprofit research foundation dedicated exclusively “to rapidly develop therapeutics that slow the progression of Huntington’s disease,” will provide funding to support the preparation and filing of the IND application, and also cover the expenses of a Phase 1 clinical trial.
Once the treatment is approved and on the market, CHDI will be reimbursed for its financial support.
“CHDI Foundation is delighted to be collaborating with Voyager, a leader in developing gene therapy programs for severe neurological diseases,” said Robi Blumenstein, president of CHDI Management.
“The selection of VY-HTT01 as their lead clinical candidate is a significant milestone that brings us an important step closer to a clinical trial of a therapeutic for Huntington’s disease,” Blumenstein added.