Top 20 Drug Discovery startups in USA

Nov 24, 2025
1
Funding: $13M
Onepot.ai automates synthesis of small molecules from basic building blocks using chemical reactions. The company operates a small molecule synthesis lab, POT-1 and an LLM, which learns from all successful and unsuccessful experiments to predict what works and what doesn't when creating compounds and suggests potential molecules. As a result, Onepot has a catalog of molecules it can manufacture. Clients (biotech and pharma companies) select the desired compounds and Onepot's technology then synthesizes these molecules and ships them to the client for use in their experiments. They ship physical products either as dry compounds or as solutions in tablets or vials.
2
Funding: $2.3B
Revolution Medicines is developing new therapies through an innovative approach that harnesses the complex chemicals of life by reconfiguring natural substances into best-in-class medicines.
3
Funding: $1.6B
Arrowhead Research is focused on developing, commercializing, and licensing a range of nanotechnology products and applications.
4
Funding: $1.4B
Relay therapeutics is a developer of an allosteric drug-discovery platform intended to apply computational techniques to protein motion.
5
Funding: $1.1B
Eikon is pioneering a new method of drug discovery based on tracking and measuring the movement of individual proteins in living cells.
6
Funding: $1B
Xaira Therapeutics is an integrated biotechnology company driving advances in artificial intelligence.
7
Funding: $865.4M
Recursion Pharmaceuticals is a digital biology company, developing a drug discovery platform and pipeline with machine learning.
8
Funding: $743M
Insitro is a machine-learning driven drug discovery and development company.
9
Funding: $700M
Odyssey Therapeutics is a computational immunology company that develops novel therapeutics for patients with autoimmune diseases.
10
Funding: $694.1M
IDEAYA is an oncology-focused biotechnology company committed to the discovery of breakthrough synthetic lethality medicines for genetically defined patient populations and immuno-oncology therapies targeting immuno-metabolism and innate immunity.
11
Funding: $693M
Generate Biomedicines is a biotechnological company focusing on creating breakthrough medicines.
12
Funding: $646M
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
13
Funding: $569.1M
Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered zinc finger DNA-binding proteins (ZFPs) as novel ZFP Therapeutics targeting various monogenic and infectious diseases with unmet medical needs.
14
Funding: $529.9M
Enveda Biosciences is a biotechnology company engineering new drugs from plants.
15
Funding: $530.3M
FogPharma creates a new class of medicines that target human disease drivers currently considered undruggable.
16
Funding: $528M
Formation Bio is a biotech company using AI to speed up drug development, aiming for efficient patient treatment.
17
Funding: $460M
Valo Health is a biotech company that is recreating the future of medicine. Valo is using computers to accelerate drug development while reducing the failure rate
18
Funding: $407.2M
Synergy is a gastrointestinal-focused biopharmaceutical company committed to science-based innovation, providing novel treatment options for patients and healthcare providers, rewarding opportunities for our employees, and creating exceptional value for our shareholders.
19
Funding: $394.3M
Absci is the drug & target discovery company harnessing deep learning AI & synthetic biology to expand the therapeutic potential of proteins
20
Funding: $366.2M
Alzheon is committed to developing innovative medicines for patients suffering from Alzheimer's disease and other neurological and psychiatric disorders. Our Alzheon’s drug development platform which comprises proprietary chemistries, robust clinical efficacy and safety datasets in patients and genetically-defined subpopulations, and the ability to optimize molecules to have the desired therapeutic attributes.
Editor: Jason Kwon
Jason Kwon is a senior editor for MedicalStartups. He has previously covered the pharmaceutical and medical research industries for FDAnews and worked as a head of marketing for medical startup Sonic Therapeutics. Before that, he co-founded a startup consulting business for emerging entrepreneurial hubs in Asia. Jason graduated from St. Bonaventure University’s journalism school. In his free time, Jason enjoys yoga, watching movie trailers, traveling to places where he can't get cell service. You can contact Jason at jaskwon(at)medicalstartups(dot)com