Preclinical testing will soon begin in VY-SOD101, a compound seen as a potential treatment for a certain gene mutation found in people with familial amyotrophic lateral sclerosis (ALS). Its developer, Voyager Therapeutics, hopes to follow this work, if successful, with clinical trials in patients in about two years.
Patients with ALS typically develop weakness in one body region (upper or lower limb or bulbar), before symptoms of progressive motor neuron problems are evident. The majority of ALS cases occur sporadically and with unknown cause, but in about 10 percent of patients, the cause is familial and can be linked to a genetic mutation. Mutations in the gene superoxide dismutase 1 (SOD1) have been linked to about 20 percent of familial forms of ALS.
Voyager selected VY-SOD101 as a clinical candidate for ALS due to mutations in SOD1 gene. The drug is composed of a proprietary adeno-associated virus (AAV) capsid and transgene with a micro RNA expression cassette that silences the expression of the mutant SOD1 gene. The company believes that VY-SOD101 may reduce the levels of the toxic SOD1 protein in the central nervous system and slow the progression of ALS through the loss of motor neurons, or nerve cells.
The company intends to complete preclinical studies evaluating the safety and effectiveness of its VY-SOD101 program for this type of ALS within two years, and file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA), an essential step in beginning a human clinical trial.
“Voyager’s exceptional performance and accomplishments during 2016 created strong momentum for the company for 2017 and beyond as we continue to focus our efforts on developing gene therapies for devastating diseases of the CNS [central nervous system],” Steven Paul, president and CEO of Voyager Therapeutics, said in a press release.
Based in Cambridge, Massaschusettes, Voyager Therapeutics is focused on the development of gene therapies for the treatment of severe central nervous system diseases that lack effective treatment strategies, such as advanced Parkinson’s disease, the monogenic (caused by a single gene) form of ALS, Friedreich’s ataxia, Huntington’s disease, and Alzheimer’s disease.