target-discovery
(10)
Insilico Medicine taught AI to design a drug. Now comes the human test.
A lung disease, an unfamiliar target and a molecule designed by machines: Insilico Medicine has taken its AI discovery experiment into Phase III. The clever part is how it pays for the long wait.
NextRNA Therapeutics
NextRNA Therapeutics was a Boston-based biotechnology company pioneering small-molecule drugs that target long non-coding RNAs (lncRNAs) - a vast, largely undrugged region of the genome that regulates gene expression. Built on the science of Dana-Farber's Carl Novina and led by co-founder and CEO Dominique Verhelle, the company developed a proprietary discovery engine to map disease-relevant lncRNA-protein interactions and drug them with selective small molecules across oncology and immunology. NextRNA raised roughly $56 million in seed and Series A financing and signed a collaboration with Bayer worth up to $547 million before winding down operations in August 2025 amid a difficult biotech market and a missed partnership milestone.
Paratus Sciences
Paratus Sciences is a biotechnology company decoding the extreme biology of bats to discover first-in-class medicines. Bats live long, tolerate viruses, resist cancer, control inflammation and thrive on high-sugar diets - traits they evolved over roughly 65 million years. Paratus integrates cell biology, comparative genomics and informatics to translate these adaptations into drug targets for inflammatory and cardiometabolic diseases. Launched in 2023 with a $100M Series A and dual sites in New York and Singapore, its lead program PS-1001 is a first-in-class pan-inflammasome inhibitor.
Apeximmune Therapeutics
Apeximmune Therapeutics is a South San Francisco / Burlingame-area biotechnology company developing next-generation antibody therapeutics that harness the immune system to treat cancer and autoimmune disease. Using a proprietary target-discovery platform built on bioinformatic analysis of thousands of tumor RNA-seq transcriptomes, the company has identified more than 30 novel immune targets. Its lead program, AI-306, is a first-in-class antagonist antibody against the novel immune checkpoint AIM-103, and its MEBA platform builds bispecific antibodies that engage macrophages to kill tumor cells. The company closed a $21.3 million Series A in 2025 led by PharmaEssentia.
MBrace Therapeutics
MBrace Therapeutics is a clinical-stage oncology company building antibody-drug conjugates (ADCs) against hard-to-drug cancer targets. Its proprietary SPARTA platform screens antibodies directly inside living tumors to surface antigens that are cancer-specific, accessible, and internalizing - the traits an ADC needs to deliver its toxic payload where it counts. Founded in 2020 and headquartered in San Diego, the company raised an $85M Series B in 2023 (led by TPG) to advance a pipeline led by MBRC-101, a first-in-class ADC targeting the EphA5 receptor now in Phase 1/2 clinical trials.
Shennon Biotechnologies
Shennon Biotechnologies is a San Francisco biotech turning immune-cell target discovery from a months-long slog into a same-day job. Its proprietary TCELERATOR platform fuses ultra-high-throughput microfluidics with AI to functionally profile millions of single immune cells in hours, pinpointing the rare T and B cells that recognize cancer antigens. Founded in 2021 by physicist-turned-founder Li Sun, the company is building a pipeline of validated targets for solid tumors and autoimmune disease, backed by a $13M seed round led by DCVC.
Talus Bio
Talus Bio is a Seattle drug-discovery company going after transcription factors and other regulatory proteins long written off as 'undruggable.' Its MARMOT platform measures the human regulome directly in living cells using functional proteomics and high-throughput mass spectrometry, then feeds tens of millions of compound-target interactions into a lab-in-the-loop AI system to design small molecules that switch disease-driving genes on or off. Founded in 2020 by Alex Federation and Lindsay Pino, the company runs preclinical programs in chordoma, non-small cell lung cancer, and prostate cancer.
Cajal Therapeutics
Cajal Therapeutics (originally launched as Cajal Neuroscience) is a Seattle biotechnology company developing novel medicines to restore biological homeostasis, with a focus on neurodegenerative diseases, anemias of inflammation, and iron-related disorders. Built on a platform that pairs integrative human genetics, high-throughput functional genomics, and industrialized whole-brain imaging with deep neuroscience expertise, the company aims to systematically validate disease targets at unprecedented scale and translate them into small molecule and RNA therapeutics. It launched in November 2022 with a $96 million Series A and counts a roster of celebrated neuroscientists among its co-founders.
Verge Genomics
Verge Genomics is a South San Francisco biotech that builds drugs from human biology instead of mouse models. By assembling one of the largest proprietary multi-omics datasets drawn directly from human brain and tissue samples, then mining it with machine learning, its CONVERGE platform identifies disease targets and drug candidates for neurodegenerative diseases like ALS, ALS-FTD and Parkinson's. Founded in 2015 by Alice Zhang and Jason Chen, the company famously moved its first AI-discovered drug from research to the clinic in four years. After that ALS candidate failed its early trial, Verge has refocused on its core asset, the platform, repositioning to supply other drug developers with human-grounded target data.
Vesalius Therapeutics
Vesalius Therapeutics is a Cambridge, Massachusetts biotech founded by Flagship Pioneering that aims to reconceptualize how common diseases are understood and treated. Its premise: illnesses that drive roughly 90% of human disease burden are not single conditions but constellations of genetically and biologically distinct diseases. Vesalius uses AI, human genetics and genomics, and patient-derived iPSC cell models to spot hidden clinical patterns, trace them to causal gene circuits, and discover novel drug targets for conditions like Parkinson's, heart failure, type 2 diabetes, and Alzheimer's.