Cambridge, MA | October 2025 — Transition Bio, a drug discovery company advancing treatments for traditionally “undruggable” targets through biomolecular condensate science, has entered into a drug discovery collaboration and license option agreement with Voyager Therapeutics. The partnership aims to develop small-molecule therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with TDP-43 pathology.
TDP-43 abnormalities are implicated in more than 90% of ALS cases and up to 45% of FTD cases, making the protein a critical but challenging therapeutic target.
Under the agreement, Transition Bio will lead the discovery and optimization of small molecules targeting TDP-43 until a development candidate is nominated. Voyager will then have the option to license exclusive global rights for development and commercialization.
Transition Bio will receive a single-digit million-dollar upfront payment and is eligible for milestone payments of up to $500 million, along with high single-digit to low double-digit royalties on future net sales.
“This collaboration fits into Voyager’s strategy of matching the right therapeutic modality to each target,” said Alfred W. Sandrock, Jr., M.D., Ph.D., President and CEO of Voyager Therapeutics. “TDP-43 has long been difficult to drug due to its complex biology. Transition Bio’s condensate-based approach offers a promising new path to modulate the protein without disrupting its essential functions.”
“By combining Transition Bio’s discovery platform with Voyager’s deep expertise in ALS and FTD, we aim to accelerate the development of much-needed therapies for these devastating conditions,” added G. Kelly Martin, Executive Chairman of Transition Bio.
Transition Bio specializes in identifying and drugging biomolecular condensates to address targets previously considered inaccessible to traditional drug discovery methods. Voyager Therapeutics focuses on advancing genetic medicines for neurological diseases.
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