Transition Bio And Voyager Therapeutics Partner To Tackle TDP-43 Pathology In ALS And FTD; The $500M Collaboration Aims To Develop First-in-Class Small Molecules

Transition Bio, a biotech company pioneering therapies for traditionally “undruggable” targets through its biomolecular condensate platform, has entered a drug discovery collaboration and license option agreement with Voyager Therapeutics. The collaboration aims to develop novel small molecules targeting TDP-43 pathology, a hallmark of several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 abnormalities are found in more than 90% of ALS and up to 45% of FTD cases, representing a critical unmet medical need.

“This collaboration with Transition Bio fits into Voyager’s vision of building a multi-modality neurotherapeutic pipeline that matches the optimal modality to each target,” said Alfred W. Sandrock, Jr., M.D., Ph.D., President & CEO of Voyager and member of the Transition Bio Board of Directors. “Historically, TDP-43 has been difficult to address therapeutically because of the complexity of targeting toxic forms of the protein without impacting the nontoxic forms that are necessary to the cell. Transition Bio’s molecular condensate technology uniquely identifies small molecules that aim to precisely correct the mislocalization of TDP-43 without abolishing its important functional activity.”

Under the agreement, Transition Bio will lead the discovery and optimization of selective small molecules directed at TDP-43 until a development candidate is nominated. At that point, Voyager will have the option to license worldwide exclusive rights to develop and commercialise the therapy. Transition Bio has received an upfront payment in the single-digit millions and stands to earn up to $500 million in potential research, development, and commercial milestones, plus royalties in the high single-digit to low double-digit range on net sales.

“By working closely with the Voyager team, we will be able to leverage a world-leading translational team with deep expertise in ALS and FTD,” said G. Kelly Martin, Executive Chairman of Transition Bio. “We look forward to advancing this program together to achieve our shared goal of transforming the lives of patients with these devastating diseases.”

Transition Bio’s broader pipeline includes programs targeting MYC-driven cancers, neurological disorders such as ALS, FTD, and myotonic dystrophy type 1, and other hard-to-treat diseases. Its platform integrates microfluidics and machine learning to discover and refine small molecules. Voyager Therapeutics, meanwhile, continues to advance its gene therapy pipeline for multiple central nervous system disorders, utilising its proprietary TRACER AAV capsid discovery platform to enhance the brain penetration of genetic medicines. The collaboration combines Transition Bio’s expertise in condensate biology with Voyager’s strength in neurological drug development to pursue innovative treatments for neurodegenerative diseases.