insitro Expands ALS Drug Development Collaboration with BMS

Summary

Share Amyotrophic Lateral Sclerosis (ALS) presents one of the most formidable challenges in medical science. For too long, the search for effective treatments has been hampered by a temptation to revisit familiar hypotheses and established biological pathways. While understandable given the slow progress and frequent setbacks, this approach rarely advances the science. At the heart of insitro’s collaboration with Bristol Myers Squibb (BMS) is the deliberate decision to boldly seek novel biology. We generated billions of multimodal data points from human-derived cellular systems and used machine learning and AI to guide our understanding of ALS biology. Our unbiased approach revealed novel biological targets – points of intervention that have been previously invisible. Today, we are excited to announce an expansion of our collaboration with BMS toward the development of new therapeutic approaches for the treatment of ALS. BMS nominated two additional ALS targets – ALS-2 and ALS-3 – under our collaboration. insitro will advance its own oligonucleotide program for ALS-1 while simultaneously progressing a small molecule for BMS. This expansion underscores the value of taking a fundamentally different approach in looking for treatments in a disease that has challenged every conventional approach. The Virtual HumanTM: Letting the Data Lead ALS is heterogeneous, its biology is complex, and many of the most intuitive hypotheses have already been tested, mostly unsuccessfully. This is precisely where insitro’s Virtual Human™ Causal Discovery Platform becomes uniquely enabling. Our platform is built on the premise that to truly understand the drivers of complex disease, one must measure it across multiple biological domains and modalities. This allows us to employ machine learning to disentangle intricate biology and uncover patterns that are invisible to conventional human inspection. To demonstrate the power of this premise, we worked with over 200 distinct ALS cell lines – both engineered models and patient-derived lines, spanning familial (inherited) and sporadic disease. We developed protocols to generate motor neurons robustly and rapidly enough to support large-scale experimentation while maintaining disease-relevant biology. Crucially, we established an imaging and screening infrastructure, including our POSH technology for pooled optical screening, capable o…