Cambridge, United Kingdom and Lexington, Mass. — November 10, 2025 — Leads & Copy — Transition Bio and Voyager Therapeutics are partnering to discover and develop small molecules for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology. The agreement includes a license option.
Transition Bio, a drug discovery company focused on unlocking traditionally “undruggable” targets, will use its biomolecular condensates technology in the collaboration. Voyager Therapeutics (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, will collaborate with Transition Bio.
Under the terms of the agreement, Transition Bio is responsible for discovering and optimizing small molecules targeting TDP-43 until a development candidate is nominated. Voyager will then have the option to license worldwide exclusive rights to develop and commercialize the program.
Transition Bio received a single-digit million-dollar upfront payment and is eligible for 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.
Alfred W. Sandrock, Jr., M.D., Ph.D., President & CEO of Voyager and a member of the Transition Bio Board of Directors, said the collaboration with Transition Bio fits into Voyager’s vision of building a multi-modality neurotherapeutic pipeline that matches the optimal modality to each target.
Sandrock added that TDP-43 has been difficult to address therapeutically because of the complexity of targeting toxic forms of the protein without impacting the nontoxic forms necessary to the cell.
G. Kelly Martin, Executive Chairman of Transition Bio, said that by working closely with Voyager, they will be able to leverage a world-leading translational team with deep expertise in ALS and FTD. He added that they look forward to advancing this program together to achieve their shared goal of transforming the lives of patients with these devastating diseases.
TDP-43 pathology is common in many neurodegenerative diseases, including over 90% of ALS cases and up to 45% of FTD cases.
Voyager Therapeutics’ pipeline includes programs for Alzheimer’s disease, Friedreich’s ataxia, Parkinson’s disease, ALS, and multiple other diseases of the central nervous system. Many of Voyager’s programs are derived from its TRACER™ AAV capsid discovery platform, which it has used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of its programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc.
Voyager Therapeutics® is a registered trademark; TRACER™ and Voyager NeuroShuttle™ are trademarks, of Voyager Therapeutics, Inc.
Transition Bio Contact:
Martin Kulander, Co-President & COO, mkulander@transitionbio.com
Voyager Contacts:
Trista Morrison, NACD.DC, tmorrison@vygr.com
Investors: Sarah McCabe, smccabe@jpa.com
Media: Adam Silverstein, adam@scientpr.com
Source: Transition Bio
