Voyager Therapeutics selects development candidate for SOD1 program

Voyager Therapeutics announced that it has selected a lead development candidate for its superoxide dismutase 1-mutated amyotrophic lateral sclerosis gene therapy program. The Company anticipates filing an investigational new drug, IND, application with the Food and Drug Administration for the candidate in mid-2025. Voyager’s SOD1-ALS lead candidate combines a highly potent siRNA construct to decrease the expression of SOD1 with an IV-delivered, blood-brain barrier-penetrant TRACER capsid. In a non-human primate study, the candidate demonstrated 73% reduction of SOD1 in cervical spinal cord motor neurons following a single intravenous dose in cynomolgus macaques. The candidate demonstrated robust knockdown of SOD1 across all levels of the spinal cord and motor cortex. Further, the candidate demonstrated an ability to transduce both neurons and astrocytes, two cell types thought to play an important role in ALS. Voyager expects to present additional data on the candidate at an upcoming scientific conference. In data previously shared at the 2022 American Society of Gene and Cell Therapy annual meeting, another Voyager SOD1-ALS preclinical candidate increased survival by a median of 152 days in a G93A mouse model, with survival in some animals exceeding 430 days. “Our SOD1-ALS program is one of several TRACER-powered gene therapy programs we expect to see advance to IND in 2025,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “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. SOD1 is a validated target, and we plan to utilize established cerebrospinal fluid and plasma biomarkers in early clinical studies to efficiently achieve potential proof of concept.”