By: IPP Bureau
Last updated : November 14, 2025 8:45 pm
The partnership aims to develop novel, selective small molecules targeting TDP-43 pathology
Transition Bio, a pioneering drug discovery company harnessing biomolecular condensates to tackle traditionally “undruggable” targets, has entered into a collaboration and license option agreement with Voyager Therapeutics, a biotech leader in genetic therapies for neurological diseases.
The partnership aims to develop novel, selective small molecules targeting TDP-43 pathology, a protein abnormality found in over 90% of amyotrophic lateral sclerosis (ALS) cases and up to 45% of frontotemporal dementia (FTD) cases, marking a significant step toward new treatments for these devastating neurodegenerative disorders.
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 totaling 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, 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 program together to achieve our shared goal of transforming the lives of patients with these devastating diseases.”