Top 50 Drug Discovery startups in USA
Apr 09, 2026
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22
1
Funding: $89.6M
Pinnacle Medicines is a peptide drug discovery company that provides peptide modality for medical needs. The company has built a proprietary platform that integrates emerging AI technologies with physics-based simulations to rationally design and optimize peptide therapeutics. Its initial focus is on immunology and cardiometabolic indications.
Pinnacle Medicines is a peptide drug discovery company that provides peptide modality for medical needs. The company has built a proprietary platform that integrates emerging AI technologies with physics-based simulations to rationally design and optimize peptide therapeutics. Its initial focus is on immunology and cardiometabolic indications.
2
Funding: $2.4B
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
3
Funding: $1.4B
Relay therapeutics is a developer of an allosteric drug-discovery platform intended to apply computational techniques to protein motion.
Relay therapeutics is a developer of an allosteric drug-discovery platform intended to apply computational techniques to protein motion.
4
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.
Eikon is pioneering a new method of drug discovery based on tracking and measuring the movement of individual proteins in living cells.
5
Funding: $1B
Xaira Therapeutics is an integrated biotechnology company driving advances in artificial intelligence.
Xaira Therapeutics is an integrated biotechnology company driving advances in artificial intelligence.
6
Funding: $865.4M
Recursion Pharmaceuticals is a digital biology company, developing a drug discovery platform and pipeline with machine learning.
Recursion Pharmaceuticals is a digital biology company, developing a drug discovery platform and pipeline with machine learning.
8
Funding: $700M
Odyssey Therapeutics is a computational immunology company that develops novel therapeutics for patients with autoimmune diseases.
Odyssey Therapeutics is a computational immunology company that develops novel therapeutics for patients with autoimmune diseases.
9
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.
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.
10
Funding: $693M
Generate Biomedicines is a biotechnological company focusing on creating breakthrough medicines.
Generate Biomedicines is a biotechnological company focusing on creating breakthrough medicines.
11
Funding: $646M
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
12
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.
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.
13
Funding: $530.3M
FogPharma creates a new class of medicines that target human disease drivers currently considered undruggable.
FogPharma creates a new class of medicines that target human disease drivers currently considered undruggable.
14
Funding: $529.9M
Enveda Biosciences is a biotechnology company engineering new drugs from plants.
Enveda Biosciences is a biotechnology company engineering new drugs from plants.
15
Funding: $528M
Formation Bio is a biotech company using AI to speed up drug development, aiming for efficient patient treatment.
Formation Bio is a biotech company using AI to speed up drug development, aiming for efficient patient treatment.
16
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
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
17
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.
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.
18
Funding: $394.3M
Absci is the drug & target discovery company harnessing deep learning AI & synthetic biology to expand the therapeutic potential of proteins
Absci is the drug & target discovery company harnessing deep learning AI & synthetic biology to expand the therapeutic potential of proteins
19
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.
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.
20
Funding: $334.9M
Regeneron Pharmaceuticals specializes in the discovery, development, and commercialization of innovative medicines.
Regeneron Pharmaceuticals specializes in the discovery, development, and commercialization of innovative medicines.
21
Funding: $333.5M
NextCure is a biopharmaceutical company focused on discovering and developing next generation immuno-oncology-based drugs.
NextCure is a biopharmaceutical company focused on discovering and developing next generation immuno-oncology-based drugs.
22
Funding: $319.5M
Alector is a hypothesis-driven, neurology-focused startup that’s pioneering the discovery and development of first-in-class, immuno-modulatory therapies for Alzheimer’s disease and other neurodegenerative disorders. We are combining state-of-the-art antibody technology and recent discoveries in neuro-immunology and human genetics to develop a broad platform of novel therapeutics that harness the immune system to fight dementia and neurodegeneration.
Alector is a hypothesis-driven, neurology-focused startup that’s pioneering the discovery and development of first-in-class, immuno-modulatory therapies for Alzheimer’s disease and other neurodegenerative disorders. We are combining state-of-the-art antibody technology and recent discoveries in neuro-immunology and human genetics to develop a broad platform of novel therapeutics that harness the immune system to fight dementia and neurodegeneration.
23
Funding: $309M
Senda Biosciences is a therapeutics platform company leveraging unprecedented insights into the molecular connections between humans.
Senda Biosciences is a therapeutics platform company leveraging unprecedented insights into the molecular connections between humans.
24
Funding: $305M
Parabilis Medicines has developed drug discovery platform for alpha-helical-linked peptides, primarily composed of specially selected non-canonical amino acids. Using this platform, Parabilis has assembled a large set of non-canonical amino acids and has hundreds of proprietary linkers for stabilizing peptides in the alpha-helical conformation. To fully exploit this diversity, the company has developed a suite of specialized experimental systems that can generate massive amounts of data on a wide range of drug properties, providing both input for building artificial intelligence models and serving as a high-throughput platform for empirically validating model predictions. As a pilot project, the company is advancing a drug against desmoid tumors.
Parabilis Medicines has developed drug discovery platform for alpha-helical-linked peptides, primarily composed of specially selected non-canonical amino acids. Using this platform, Parabilis has assembled a large set of non-canonical amino acids and has hundreds of proprietary linkers for stabilizing peptides in the alpha-helical conformation. To fully exploit this diversity, the company has developed a suite of specialized experimental systems that can generate massive amounts of data on a wide range of drug properties, providing both input for building artificial intelligence models and serving as a high-throughput platform for empirically validating model predictions. As a pilot project, the company is advancing a drug against desmoid tumors.
25
Funding: $303M
Iambic Therapeutics is built on technologies for predicting protein-ligand structures, generating molecular designs and other drug discovery tasks. Using the technologies, Iambic claims it can accelerate drug discovery, pointing to the two years it took to take its lead project from program launch to IND to make its case.
Iambic Therapeutics is built on technologies for predicting protein-ligand structures, generating molecular designs and other drug discovery tasks. Using the technologies, Iambic claims it can accelerate drug discovery, pointing to the two years it took to take its lead project from program launch to IND to make its case.
26
Funding: $294M
Cellarity's platform digitizes the molecular information in cells to elucidate how they behave in health and disease.
Cellarity's platform digitizes the molecular information in cells to elucidate how they behave in health and disease.
27
Funding: $287M
Dewpoint Therapeutics is a biotech company which develop a drug platform that targets biomolecular condensates.
Dewpoint Therapeutics is a biotech company which develop a drug platform that targets biomolecular condensates.
28
Funding: $283.4M
Atea Pharmaceuticals is a biopharmaceutical company which is engaged in the acquisition, discovery and development of novel antiviral therapeutics.
Atea Pharmaceuticals is a biopharmaceutical company which is engaged in the acquisition, discovery and development of novel antiviral therapeutics.
29
Funding: $256.1M
Genesis Therapeutics unifies AI and biotech to accelerate the discovery of new medicines
Genesis Therapeutics unifies AI and biotech to accelerate the discovery of new medicines
30
Funding: $250M
Terremoto Biosciences is a biotechnology company leveraging the power of lysine-based covalency to develop optimized medicines.
Terremoto Biosciences is a biotechnology company leveraging the power of lysine-based covalency to develop optimized medicines.
31
Funding: $239.3M
Alto Neuroscience is a clinical-stage biopharmaceutical company that creates precision medicines through its AI-driven biomarker platform.
Alto Neuroscience is a clinical-stage biopharmaceutical company that creates precision medicines through its AI-driven biomarker platform.
32
Funding: $231M
Chai Discovery develops AI models that predict and reprogram biochemical molecule interactions. Chai’s goal is to shorten the drug development timeline so that potential new medicines enter first-in-human studies and reach the market quicker. It can design molecules that have needed properties and tackle challenging targets that have been out of reach. The team has already used its flagship model Chai 2 to develop monoclonal antibodies aimed against tough-to-drug targets, and (accoring to the company) the new designs have properties similar to known antibody drugs.
Chai Discovery develops AI models that predict and reprogram biochemical molecule interactions. Chai’s goal is to shorten the drug development timeline so that potential new medicines enter first-in-human studies and reach the market quicker. It can design molecules that have needed properties and tackle challenging targets that have been out of reach. The team has already used its flagship model Chai 2 to develop monoclonal antibodies aimed against tough-to-drug targets, and (accoring to the company) the new designs have properties similar to known antibody drugs.
33
Funding: $225.1M
Plexium is a drug development startup that leverages techniques from various physical-science and computational disciplines.
Plexium is a drug development startup that leverages techniques from various physical-science and computational disciplines.
34
Funding: $221.6M
Atomwise is the creator of AtomNet, the first Deep Learning technology for novel small molecule discovery, characterized by its unprecedented speed, accuracy, and diversity.
Atomwise is the creator of AtomNet, the first Deep Learning technology for novel small molecule discovery, characterized by its unprecedented speed, accuracy, and diversity.
35
Funding: $220M
Soley Therapeutics has developed a platform that uses computer vision to capture the response of living cells to drug stress, transforming image characteristics into structured data and uses AI to distinguish drug activity from toxicity, identify mechanisms of action, identify pathways for new drug discovery. Computer vision recognizes features invisible to the human eye that result from a variety of intracellular events, such as protein movement, localization, expression and organelle and membrane dynamics. The company's automated infrastructure enables the screening of over 100,000 compounds per week. The company's first drugs have demonstrated high preclinical efficacy in both hematological and solid tumors. They are also being tested for the treatment of neurodegenerative diseases, obesity/metabolic diseases and hair regeneration.
Soley Therapeutics has developed a platform that uses computer vision to capture the response of living cells to drug stress, transforming image characteristics into structured data and uses AI to distinguish drug activity from toxicity, identify mechanisms of action, identify pathways for new drug discovery. Computer vision recognizes features invisible to the human eye that result from a variety of intracellular events, such as protein movement, localization, expression and organelle and membrane dynamics. The company's automated infrastructure enables the screening of over 100,000 compounds per week. The company's first drugs have demonstrated high preclinical efficacy in both hematological and solid tumors. They are also being tested for the treatment of neurodegenerative diseases, obesity/metabolic diseases and hair regeneration.
36
Funding: $200M
Terray Therapeutics is a biotechnology company that propels drug discovery into the information age.
Terray Therapeutics is a biotechnology company that propels drug discovery into the information age.
37
Funding: $191.4M
Stealth BioTherapeutics is an innovative biopharmaceutical company.
Stealth BioTherapeutics is an innovative biopharmaceutical company.
38
Funding: $186M
BioXcel Therapeutics is a clinical stage biopharmaceutical company that utilizes novel artificial intelligence, or AI, to identify the next wave of medicines across neuroscience and immuno-oncology.
BioXcel Therapeutics is a clinical stage biopharmaceutical company that utilizes novel artificial intelligence, or AI, to identify the next wave of medicines across neuroscience and immuno-oncology.
39
Funding: $176.2M
METiS Therapeutics uses integrated drug discovery to develop medicines in a variety of illness areas.
METiS Therapeutics uses integrated drug discovery to develop medicines in a variety of illness areas.
40
Funding: $172M
vTv Therapeutics, a pharmaceutical company, focuses on the discovery and development of human therapeutics to fill unmet medical needs. Its mission is to utilize its innovative technology to rapidly translate the functional modulation of human proteins into safe and effective medicines.
vTv Therapeutics, a pharmaceutical company, focuses on the discovery and development of human therapeutics to fill unmet medical needs. Its mission is to utilize its innovative technology to rapidly translate the functional modulation of human proteins into safe and effective medicines.
41
Funding: $153M
Superluminal Medicines is a generative biological and chemical company accelerating new possibilities in drug discovery and development.
Superluminal Medicines is a generative biological and chemical company accelerating new possibilities in drug discovery and development.
42
Funding: $147.6M
Palleon Pharmaceuticals develops drugs that target sugar-sensing molecules on the surfaces of cells. It has integrated technologies and insights from world-renowned scientific leaders in glycoscience and human immunology to create the first Glycoimmune Checkpoint Inhibitors to treat cancer patients.
Palleon Pharmaceuticals develops drugs that target sugar-sensing molecules on the surfaces of cells. It has integrated technologies and insights from world-renowned scientific leaders in glycoscience and human immunology to create the first Glycoimmune Checkpoint Inhibitors to treat cancer patients.
43
Funding: $142M
EvolutionaryScale is a developer of biological artificial intelligence models for therapeutic design.
EvolutionaryScale is a developer of biological artificial intelligence models for therapeutic design.
44
Funding: $135.5M
The Cour particle platforms provide a novel and highly specific immune targeting capability which may be safely applied to severe inflammatory modulation, and immune (antigen) tolerization.
The Cour particle platforms provide a novel and highly specific immune targeting capability which may be safely applied to severe inflammatory modulation, and immune (antigen) tolerization.
45
Funding: $134.1M
We use genomic data to find better drugs, faster. We find new cures for brain diseases 1000X more cheaply and quickly using our proven network algorithms.
We use genomic data to find better drugs, faster. We find new cures for brain diseases 1000X more cheaply and quickly using our proven network algorithms.
46
Funding: $128.3M
Alloy Therapeutics makes medicine by democratizing access to foundational drug discovery platforms and services.
Alloy Therapeutics makes medicine by democratizing access to foundational drug discovery platforms and services.
47
Funding: $115M
Octant Bio is a developer of synthetic technology used to improve health and treat disease.
Octant Bio is a developer of synthetic technology used to improve health and treat disease.
48
Funding: $100M
Atavistik Bio's drug development platform that focuses on allosteric control mechanisms, which is powered by nature's own evolution, helps to tackle metabolic diseases and cancers
Atavistik Bio's drug development platform that focuses on allosteric control mechanisms, which is powered by nature's own evolution, helps to tackle metabolic diseases and cancers
49
Funding: $99.3M
BigHat Biosciences is a protein therapeutics company that develops an antibody design platform guided by artificial intelligence.
BigHat Biosciences is a protein therapeutics company that develops an antibody design platform guided by artificial intelligence.
50
Funding: $71M
Vilya is a biotechnology company that plans to design novel artificial molecules with customized biologic-like properties that can hit difficult-to-drug therapeutic targets in a broad set of indications.
Vilya is a biotechnology company that plans to design novel artificial molecules with customized biologic-like properties that can hit difficult-to-drug therapeutic targets in a broad set of indications.
