Voyager Interrupts IND Filing to Consider Other Payloads for SOD1 ALS Gene Therapy
The company will evaluate new payloads for its gene therapy initiative, changing its schedule
Voyager Therapeutics, a biotechnology company that specializes in neurogenetic medicines, shared on February 11, 2025, that it will be looking into different payloads for VY9323, its gene therapy program targeting superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). This decision comes after reviewing new three-month data from a study conducted on non-human primates (NHPs). The data indicate that using a different payload is needed to reach the desired product profile (1). The new plan moves the company’s investigational new drug (IND) application filing, which was first set for mid-2025. The company's cash runway is now anticipated to last until mid-2027, excluding possible milestone payments from current partnerships.
The company stated that there are no planned changes to the novel capsid component of the SOD1 ALS gene therapy program. The capsid used in the VY1706 gene therapy program for tau silencing reached the expected activity levels and demonstrated good tolerance in three-month studies with non-human primates.
“Recent preclinical findings suggest that the siRNA component of VY9323 does not align with our high standards, as it seems to cause an off-target effect that limits the therapeutic window.” Although we are sad that the development candidate VY9323 will not move forward, we are optimistic about the possibility of finding another payload and a way to continue this program, considering the existing need in.
“ALS,” stated Alfred W. Sandrock, Jr., MD, PhD, the chief executive officer of Voyager, in a company press release.
Due to the need to evaluate different payloads, Voyager has put a hold on the IND application and Canadian clinical trial application (CTA) for VY9323, which were expected to be filed in mid-2025. The company plans to share an update on the new expected timing for its SOD1 ALS program at the right time.
The choice regarding the SOD1 ALS program does not affect the company’s other gene therapy initiatives. The company still anticipates IND filings in 2025 from Neurocrine Biosciences for a gene therapy program targeting glucosylceramidase beta 1 (GBA1) Parkinson’s and other GBA1-related diseases, along with a program for Friedreich’s ataxia (FA). In January 2023, Voyager teamed up with Neurocrine Biosciences to focus on three gene therapy programs aimed at rare targets in the central nervous system (CNS). Every program uses Voyager’s innovative approach to redirect the tropism of adeno-associated virus through the specific expression of RNA in different cell types with TRACER capsids. Neurocrine Biosciences agreed to pay Voyager $175 million upfront. Neurocrine Biosciences has also agreed to cover all expenses related to the collaboration.
Voyager is continuing to progress with VY1706 and plans to submit an IND for this gene therapy program in 2026. The company has a range of programs focused on Alzheimer’s disease, ALS, Parkinson’s disease, and several other CNS diseases. The company has several programs that come from its TRACER platform. This platform helps create new capsids and find related receptors, aiming to improve the ability of genetic medicines to reach the brain after being given through an IV.
“We are happy that our new TRACER capsids keep working well in different programs, and we think they could change gene therapy for CNS diseases.” Sandrock, Jr. stated in the press release, "We still anticipate IND filings in 2025 for our gene therapy candidates for GBA1 and FA, and in 2026 for VY1706."
Source: Voyager Therapeutics
