Voyager Therapeutics Partners with Transition Bio for Small Molecule Neurodegenerative Drug Discovery; CRISPR Therapeutics Ends Work on CAR-T Therapy

Voyager Therapeutics has entered a partnership with Transition Bio to discover and develop small molecule drugs targeting TDP-43, a protein implicated in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)135.

Under the agreement, Transition Bio will use its condensate biology and microfluidics-powered drug discovery platform to identify and optimize small molecules that modify the mislocalization of TDP-43 without affecting its normal function1.

Voyager is paying Transition Bio a single-digit million-dollar upfront payment, with the potential for milestone payments totaling up to $500 million if research, development, commercial, and sales benchmarks are met1.

Voyager will have the right to license and further develop the identified candidates after Transition chooses a lead compound15.

There are currently no approved drugs specifically targeting TDP-43, though several research candidates exist1.

This deal marks Voyager’s first move into small molecule drug development, as the company’s focus has traditionally been on biologics and gene therapies1.

No recent news was found regarding CRISPR Therapeutics ending work on a CAR-T therapy, suggesting either the event was not widely reported or occurred outside the immediate recent news cycle.

Sources:

1. https://www.fiercebiotech.com/biotech/voyager-transition-bio-set-sail-neuro-research-trek-search-small-molecule-candidates

3. https://www.gurufocus.com/news/3198476/voyager-therapeutics-vygr-partners-with-transition-bio-for-als-and-ftd-drug-discovery

5. https://www.streetinsider.com/Corporate+News/Transition+Bio+and+Voyager+partner+on+TDP-43+drugs+for+ALS+and+dementia/25582307.html