Voyager Therapeutics is shoving off on a new journey to discover and develop small molecules targeting protein bundles that contribute to neurodegenerative diseases. Voyager is paying travel partner Transition Bio a single-digit million-dollar upfront payment, with additional payments up to $500 million possible if certain research, development, commercial and sales milestones are met.
In return for the cash, Transition will work to identify small molecules that can bind to TDP-43, a protein that bunches together in certain patients with neurodegenerative conditions such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.
Voyager will then have the option to license and develop candidates from Transition’s work, the company announced in a Nov. 10 release.
Despite its promise, there are currently no approved drugs targeting TDP-43 on the market, though a range of candidates exists.
“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,” Voyager’s president and CEO Alfred Sandrock Jr., M.D., said in the release. “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.”
With a presence in both Cambridge, Massachusetts, and Cambridge, England, Transition Bio uses a drug discovery platform powered by microfluidics to find potential new therapeutics. The company secured a $50 million series A round in 2022 and has its own preclinical pipeline led by a YTHDC1 inhibitor (PDF) in development for multiple cancers.
The Transition pact represents Voyager’s first foray into the small molecule space. The neuro-focused biotech is keen on biologics, with its sole clinical asset a tau-targeting antibody in a phase 1 study for Alzheimer’s disease. Voyager is also tackling ALS through gene therapy but, earlier this year, had to delay its efforts in order to find a new payload.
Voyager is also collaborating with Neurocrine Biosciences on gene therapies for central nervous system diseases. In May, Neurocrine returned two discovery-stage programs from this pact to Voyager, though the overall collaboration remains afloat.