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Voyager Therapeutics Selects SOD1-ALS siRNA Development Candidate

NEW YORK – Voyager Therapeutics on Wednesday said it has selected a drug that it will evaluate as a treatment for patients with SOD1-mutated amyotrophic lateral sclerosis (ALS). 

The investigational agent is designed to intravenously deliver a capsid that penetrates the blood-brain barrier with a small interfering RNA and decrease expression of SOD1. In a preclinical study of nonhuman primates, a single dose of the treatment reduced SOD1 expression by 73 percent in cervical spinal cord motor neurons.

Voyager is hoping to file an investigational new drug application for this agent with the US Food and Drug Administration in mid-2025. 

The Lexington, Massachusetts-based firm developed the capsids for this product using its adeno-associated virus capsid discovery platform, called Tropism Redirection of AAV by Cell-type-specific Expression of RNA, or Tracer for short.

"We believe our development candidate could represent a significant advancement in the treatment of SOD1-mutated ALS by offering the potential for durable SOD1 knockdown with a single IV administration," Voyager CEO Alfred Sandrock said in a statement. "We plan to utilize established cerebrospinal fluid and plasma biomarkers in early clinical studies to efficiently achieve potential proof of concept."

ALS, also called Lou Gehrig's disease, is a progressive neurodegenerative disease. SOD1-ALS is the most common genetic form of the disease, accounting for roughly 2 percent of ALS cases. Earlier this year, the FDA approved Biogen's antisense oligonucleotide drug Qalsody (tofersen) for SOD1-ALS, making it the first approved therapy for a genetic form of the disease.