Voyager Therapeutics, a company that specializes in bioinformatics, has announced a significant update to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The company informed on Tuesday, February 11, 2025, that it would be experimenting with other main deliveries for its SOD1 ALS gene therapy program due to the singular nature of the recent preclinical studies’ outcomes. This change means that the company will not have its original timeline for submitting an investigational new drug (IND) application for VY9323, which should have been in mid-2025.

The report was released on the heels of encouraging data from three-month non-human primate (NHP) studies that the research team had not obtained previously. The aforementioned alternate payload discovery was the one point indicated as being a necessity to have the gene therapy of the sought product profile developed. Dr. Alfred W. Sandrock, Jr., the CEO of Voyager, came up with a good excuse for shifting the siRNA payload and mentioned the company’s high standards, which were not met owing to the off-target effect. He specified that the practically extinct therapeutic window came forth because the siRNA payload was identified as being off-target. The executive also underlined the fact that in spite of this setback, there are prospects of the company not only identifying an alternate payload but also charting a path forward for the program, considering the massive unmet demand in the ALS field.

It is worth mentioning that Voyager stated that there are no plans to alter the capsid, which is at the heart of the therapy’s story. The firm stressed that the capsid that was attached to the VY1706 (tau silencing) gene therapy program was successful in triggering the desired activity levels and had no adverse effects in three-month NHP studies. Furthermore, this is also the case with Voyager’s TRACER AAV capsid discovery platform, which has been operating consistently and impressively for several programs in CNS disease treatment. Therefore, the company believes TRACER is likely to be the technology that will revolutionize gene therapy for these diseases.

Voyager’s financial picture and the time to develop it now that they are considering reassessing the SOD1 ALS program will heavily depend on it. The company now expects its cash runway to extend into mid-2027, not including any potential milestone payments from existing partnerships. This additional period will give Voyager enough time to figure out a different approach to the SOD1 ALS program and perhaps come up with a more suitable payload for the therapy. The company has committed to providing updates on the expected timing for its SOD1 ALS program when appropriate as it works to address the challenges identified in the preclinical studies.

Voyager, despite the holdback in the SOD1 ALS program, remains positive about its other gene therapy projects. The company continues to report that IND filings will be done in 2025 for its gene therapy candidates directed at both GBA1 Parkinson’s and other GBA1-mediated diseases, as well as for its program on Friedreich’s ataxia. These programs are the product of the Neurocrine Biosciences collaboration. Moreover, Voyager is planning to submit an IND in 2026 for VY1706, its tau silencing gene therapy program. The company’s diverse pipeline and ongoing partnerships underscore its commitment to advancing neurogenetic medicines across multiple indications.

This announcement has grabbed the attention of the biotechnology and medical areas, thereby eliciting attention and awareness of the challenges in gene therapy for neurodegenerative diseases. This announcement signifies the fact that Voyager’s SOD1 ALS program suffers from a minor hiccup but on the other hand, it proves the firm’s dedication to strong scientific rules and patient safety. The choice to reflect on the cargo instead of a potentially more ideal choice represents a needs-based and responsible development approach in the context of the fulfillment of living expectancy.

Voyager, like an explorer in his expedition, deals with the challenges that are the roadblocks for ALS research and gene therapy development in the field as a whole. The company’s open communication has not only helped in clarifying preclinical findings but also explained it coming from previous and future perspectives which adds to the valuable information about the part of the drug development process that was developed and the importance of comprehensive preclinical assessment. The biotechnology industry will closely monitor Voyager’s progress to the peak of the mountain in this case as they engineer novel technological solutions to SOD1 ALS and various other neurodegenerative diseases.