insitro expands Bristol Myers Squibb partnership with two new ALS targets

insitro announced the expansion of its collaboration with Bristol Myers Squibb (NYSE: BMY) to include two additional therapeutic targets for amyotrophic lateral sclerosis treatment. The AI therapeutics company identified the targets, designated ALS-2 and ALS-3, through its Virtual Human platform.

The new targets join ALS-1, which Bristol Myers Squibb nominated in December 2024. insitro received a $10 million milestone payment for the selection of the two additional targets, according to the company's statement.

The collaboration employs multiple therapeutic approaches for the targets. insitro will develop its own oligonucleotide program for ALS-1 while simultaneously advancing a small molecule program for Bristol Myers Squibb targeting the same designation.

insitro's platform integrates human-derived cell data with machine learning to identify disease drivers. The company focuses on processes that affect TDP-43 mislocalization, a mechanism present in approximately 97% of ALS patients.

In laboratory validation experiments using iPSC-derived motor neurons, target modulation restored neurite growth in ALS cellular models. The experiments also showed reduction of cryptic exons and restoration of full-length transcripts, according to the company.

"Our platform allows us to build a data-driven map of the impact of ALS on motor neurons and identify novel drivers of neurodegeneration," said Daphne Koller, founder and CEO of insitro.

The collaboration aims to develop disease-modifying interventions for ALS patients. insitro has raised approximately $800 million from investors and generated about $150 million in revenue from partnerships with Bristol Myers Squibb, Eli Lilly, and Gilead Sciences.

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