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Transition Bio & Voyager collaborate to advance small molecules targeting TDP-43 in ALS and frontotemporal dementia

Cambridge, UK
Wednesday, November 12, 2025, 13:00 Hrs  [IST]

Transition Bio, a drug discovery company focused on unlocking traditionally “undruggable” targets by leveraging biomolecular condensates, and Voyager Therapeutics, a biotechnology company dedicated to leveraging genetics to treat neurological diseases, have entered into a drug discovery collaboration and license option agreement. The companies will collaborate to discover and develop novel, selective small molecules for people suffering from amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology. TDP-43 pathology is common in many neurodegenerative diseases, including over 90% of ALS cases and up to 45% of FTD cases.

Under the terms of the partnership, Transition Bio is responsible for the discovery and optimization of small molecules targeting TDP-43 until nomination of a development candidate, upon which Voyager will have an option to license the worldwide exclusive rights to develop and commercialize the program. Transition Bio has received a single-digit million-dollar upfront payment and is eligible to receive potential research, development, commercial and net sales milestone payments totalling up to $500 million. Transition Bio is also eligible for high single-digit to low double-digit royalties on net sales.

“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.”

“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 programme together to achieve our shared goal of transforming the lives of patients with these devastating diseases.”

Transition Bio, Inc. is a private biotechnology company that is unlocking a fundamental control layer – biomolecular condensate – to discover transformative medicines for diseases driven by “undruggable” targets.

Voyager Therapeutics, Inc. is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases.

 

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