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Introduction
The pharmaceutical industry is undergoing a revolutionary transformation as artificial intelligence reshapes the traditional drug discovery and development landscape. What once took decades and billions of dollars to accomplish is now being accelerated through sophisticated AI platforms that can analyze vast datasets, predict molecular behavior, and identify promising therapeutic targets with unprecedented speed and accuracy. This paradigm shift represents one of the most significant advances in medical research since the advent of modern chemistry.
The companies featured in this comprehensive review represent the vanguard of AI-driven pharmaceutical innovation, each bringing unique technologies and approaches to solve some of healthcare's most challenging problems. From generative AI platforms that design novel proteins to machine learning systems that predict clinical trial outcomes, these organizations are not merely optimizing existing processes—they are fundamentally reimagining how life-saving medicines are discovered, developed, and brought to market.
These ten pioneering companies have collectively raised billions in funding, formed strategic partnerships with pharmaceutical giants, and advanced numerous candidates through clinical trials, demonstrating the tangible impact of AI on drug discovery. Their diverse technological approaches—spanning mRNA biology modulation, small molecule design, protein engineering, and beyond—illustrate the broad applicability of artificial intelligence across therapeutic areas including oncology, immunology, neuroscience, and rare diseases. As we explore each company's innovations, partnerships, and pipeline progress, we witness the emergence of a new era in pharmaceutical development that promises to deliver more effective treatments faster and more cost-effectively than ever before.
Anima Biotech
Technology: mRNA biology modulators
Disease areas: Immunology, oncology and neuroscience
Recent news: Announced promising preclinical data for lead pulmonary fibrosis candidate
Anima Biotech's AI drug discovery technology is built around its mRNA Lightning.AI platform, which images hundreds of cellular pathways in both healthy and diseased cells to train disease-specific AI models, making use of neural networks to help these models distinguish between healthy and diseased cells and identify dysregulated pathways. These pathways are subsequently analyzed to uncover novel targets backed by experimental validation.
Anima currently has 20 preclinical candidates being evaluated for immunology, oncology, and neuroscience indications, with its most advanced candidate indicated for the treatment of lung fibrosis. The company announced in February 2024 that this candidate had shown promising preclinical results and could open up new avenues for treating patients with idiopathic pulmonary fibrosis.
The AI drug discovery company also has ongoing collaborations with several pharma giants. After initially partnering with Eli Lilly in 2018 and Takeda in 2021, its most recent partnership was formed with AbbVie in 2023 for the discovery and development of mRNA biology modulators against oncology and immunology targets.
Atomwise
Technology: TYK2 inhibitor
Disease area: Autoimmune and autoinflammatory diseases
Recent news: Published results showcasing AtomNet's ability for drug discovery
Atomwise is leveraging the power of AI in an attempt to revolutionize small molecule drug discovery. The company wants to tackle the most challenging, seemingly impossible targets and streamline the drug discovery process to give drug developers more shots on goal.
Atomwise's approach to drug discovery shifts the mode of drug discovery away from serendipitous discovery and toward search based on structure, making the drug discovery process more rational, effective, and efficient. The company's AtomNet platform incorporates deep learning for structure-based drug design, enabling the rapid, AI-powered search of its proprietary library of more than three trillion synthesizable compounds.
In April 2024, the AI drug discovery company published results from a 318-target study highlighting AtomNet as a viable alternative to high-throughput screening, with the AI platform identifying structurally novel hits for 235 of the 318 targets evaluated in the study.
In one of Atomwise's biggest deals, the AI drug discovery company signed a strategic multi-target research collaboration with pharma giant Sanofi, which leverages its AtomNet platform for computational discovery and research of up to five drug targets.
Meanwhile, in October 2023, the company also announced the nomination of its first AI-driven development candidate – an orally bioavailable and allosteric TYK2 inhibitor. TYK2 is a key mediator in cytokine signaling pathways linked to a broad range of immune-mediated inflammatory conditions. By modulating the TYK2 pathway, the candidate has the potential to treat a wide range of autoimmune and autoinflammatory diseases, including inflammatory bowel disease, systemic lupus erythematosus, psoriasis, and psoriatic arthritis.
A top priority for Atomwise now is to get this candidate into human testing.
BPGbio
Technology: Drug-lipid conjugate nanodispersion
Disease areas: Oncology, neurology and rare diseases
Recent news: Formed 5-year partnership with the University of Oxford for novel protein degradation technologies
Named as the 2024 "BioTech AI Company of the Year" by BioTech Breakthrough Awards, BPGbio has an AI platform called NAi Interrogative Biology, which leverages one of the world's largest non-governmental biobanks with more than 100,000 clinically annotated and multi-omics annotated patient samples. Powered by causal AI and the world's fastest supercomputer, Frontier, at Oak Ridge National Laboratory, the platform accelerates the identification of novel drug targets and biomarkers, enabling BPGbio to advance treatments for critical diseases.
After acquiring all of Boston-based biotech BERG's assets in 2023, BPGbio now has a pipeline of late-stage clinical assets in oncology, rare diseases, and neurology. Its lead asset, BPM31510, is a drug-lipid conjugate nanodispersion containing ubidecarenone (CoQ10) that is being tested as topical, intravenous, and oral formulations. CoQ10 is a vitamin-like fat-soluble substance found in the mitochondria of human cells. Validated by BPGbio's AI platform, BPM31510 has been found to induce a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. Furthermore, in many mitochondrial diseases, restoring CoQ10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction.
The intravenous formulation of BPM31510 is in phase 2 trials for glioblastoma multiforme (GBM) and pancreatic cancer. It has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for both of these indications. Meanwhile, the topical formulation has also recently received orphan drug designation for epidermolysis bullosa, as well as rare pediatric disease designation for primary CoQ10 deficiency.
In October 2024, BPGbio entered into a 5-year collaboration with the University of Oxford in the U.K. focused on advancing novel protein degradation technologies, particularly in oncology and central nervous system (CNS) diseases, with the goal of unlocking new therapeutic pathways for conditions with limited treatment options. This is one of many partnerships the AI drug discovery company has with academic institutions, as well as healthcare organizations. Plus, it also has partnerships with the likes of AstraZeneca and Boehringer Ingelheim formed through BERG.
Cradle Bio
Focus areas: Therapeutics, diagnostics, food, chemicals, and agriculture
Major partnerships: Novo Nordisk, Johnson & Johnson, Grifols and Twist Biosciences
Recent news: Raised $73 million in series B funding
Cradle Bio uses generative AI to help biologists design improved proteins and accelerate research and development, making it easier, quicker, and more cost-effective to create bio-based products for therapeutics, diagnostics, food, chemicals, and agriculture. The company's AI models are trained on billions of protein sequences, as well as data generated in Cradle's own wet lab.
Cradle operates as a platform provider, working with biotech and pharmaceutical companies to accelerate protein engineering. The company has secured partnerships with major industry players, including Novo Nordisk, Johnson & Johnson, Grifols, and Twist Biosciences, helping them refine and optimize proteins for various applications. It is working on multiple projects focused on engineering a wide range of protein modalities, including enzymes, vaccines, peptides, and antibodies across a broad spectrum of desired protein properties, such as stability, expression, activity, binding affinity, and specificity.
In November 2024, Cradle raised $73 million in series B funding to help the company accelerate the adoption of AI-powered protein engineering. This funding took the total raised by the company to date to more than $100 million.
Iktos
Technology: Small molecules
Disease areas: Inflammatory and autoimmune diseases, oncology and obesity
Recent news: Secured a €2.5 million ($2.7 million) grant from the EIC Accelerator
Based in Paris, Iktos is making use of AI and robotics synthesis automation technology for drug discovery and design, using it to rapidly identify small molecules that can become clinical candidates. By using AI, Iktos aims to speed up the drug discovery process while increasing the probability of success of drug candidates reaching clinical development. This approach has already been validated by Iktos through more than 50 academic and industrial collaborations, with pharmaceutical and biotech companies such as Janssen, Merck, Pfizer, Servier, Ono, and Teijin.
The company has multiple AI technologies to help with the discovery process. These are: Makya, a generative AI that generates optimal molecules in silico; Spaya, a retrosynthesis AI platform that processes the molecules in order to identify synthesis routes compatible with Iktos' robots; and Ilaka, an "orchestration" AI platform that takes over to manage the entire workflow, from ordering raw materials to overseeing synthesis campaigns and directing the company's robots to perform the chemistry. From this, the company currently has a pipeline focused on delivering preclinical candidates for inflammatory and autoimmune diseases, oncology, and obesity.
In March 2023, the company announced that it had closed a €15.5 million ($16.4 million) series A financing round, enabling it to further develop its AI and drug discovery capabilities, expand its existing SaaS software offering, as well as launch Iktos Robotics – an end-to-end, drug discovery platform that combines AI and automation of chemical synthesis to significantly accelerate drug discovery timelines.
More recently, in February 2025, Iktos secured a €2.5 million ($2.7 million) grant from the European Innovation Council (EIC) Accelerator to advance its AI and robotics technology. Plus, in January, the company signed a small molecule AI drug discovery collaboration with Cube Biotech. This partnership will leverage Iktos's AI platform and Cube Biotech's advanced protein technologies to develop novel agonists of the Amylin Receptor.
Insilico Medicine
Technology: Small molecule inhibitor
Disease area: Fibrosis
Recent news: Raised $110 million in series E round
Insilico Medicine is intent on using AI for every step of pharmaceutical research and development, in an effort to significantly reduce the time and cost associated with bringing life-saving medicines to patients. To achieve this, the company connects biology, chemistry, and clinical trial analysis using next-generation AI systems. Its fully integrated drug discovery suite, Pharma.AI, consists of PandaOmics (to discover and prioritize novel targets), Chemistry42 (to generate novel molecules), and InClinico (to design and predict clinical trials).
In June 2023, Insilico's small molecule inhibitor drug candidate, INS018_055, for the treatment of idiopathic pulmonary fibrosis, became the first entirely AI-discovered and AI-designed drug to enter a phase 2 clinical trial, representing an important milestone for the industry. The company then announced in June 2024 that it had completed patient enrollment in a phase 2a study of the candidate in China. The AI company also has two more drugs in clinical stages that have been partially generated by AI. One is for COVID-19, and the other is for solid tumors.
It is worth mentioning, too, that in November 2022, Insilico signed a major collaboration deal with Sanofi, worth up to $1.2 billion. The agreement stated that Sanofi would leverage Insilico's Pharma.AI to identify disease targets, generate new molecular data, and predict clinical trial results to advance drug candidates for up to six new targets.
Meanwhile, earlier this month, Insilico secured $110 million in series E financing, which will go toward advancing its drug development pipeline and AI platform developments.
insitro
Disease areas: Neuroscience and metabolic diseases
Recent funding: Raised $400 million in series C round
Recent news: Formed collaboration with Eli Lilly to advance novel treatments for metabolic diseases, including MASLD
By generating high-throughput, functional genomic data sets that align with patient data, and interpreting those data via novel machine learning methods, insitro builds predictive models that can accelerate target selection and the design of effective therapeutics. This AI-assisted drug discovery has built the foundation of insitro's pipeline, which includes candidates in neuroscience and metabolic diseases that are being advanced both internally and through strategic partnerships.
Speaking of strategic partnerships, insitro has so far established several of them. Shortly after launching in 2018, it formed a partnership with Gilead to discover and develop novel therapies for nonalcoholic steatohepatitis (NASH). Then, in 2020, the company signed a deal with Bristol Myers Squibb (BMS) to discover new therapies for amyotrophic lateral sclerosis (ALS) – insitro received a $25 million milestone payment as part of this agreement in December last year for the achievement of discovery milestones and the selection of the first novel target for ALS. And, most recently, insitro teamed up with Eli Lilly to advance novel treatments for metabolic diseases, including metabolic dysfunction-associated steatotic liver disease (MASLD), based on targets identified by insitro using the company's AI/ML-based platform.
In terms of funding outside of these collaborations, insitro also managed to bring in an impressive $400 million in series C financing back in 2021, which went toward further expanding the company's platform capabilities and pipeline.
Isomorphic Labs
Drug discovery collaborations: Eli Lilly and Novartis
Recent achievement: Co-developed AlphaFold3 with Google DeepMind
Recent news: Expanded the scope of small molecule drug discovery agreement with Novartis
As the sister company of the prominent AI research laboratory Google Deepmind, Isomorphic Labs is working to develop cutting-edge computational techniques in fields like deep learning, reinforcement learning, active learning, representation learning, and more, to solve some of the toughest challenges in drug discovery, and some of the most stubborn scientific problems in biology, chemistry and medical research today.
The company helped Google DeepMind to develop the acclaimed AI model AlphaFold3, which can accurately predict the 3D structure of proteins. The technology has been heralded as a game-changer for drug discovery, as it has the potential to allow researchers to identify promising drug candidates much more quickly and precisely than current methods, especially since the companies decided to open-source it in November last year. Isomorphic Labs is now using AlphaFold3 internally to speed up its own drug discovery efforts.
Over the last year or so, Isomorphic Labs has formed a couple of high-profile partnerships with big pharma companies. It kicked off 2024 by announcing two strategic collaborations with Eli Lilly and Novartis that have the potential to be worth nearly $3 billion to Isomorphic Labs, excluding any royalties that may result from future drug sales. Last month, the company said that it had also expanded the scope of its small molecule drug discovery agreement with Novartis, adding up to three additional research programs.
Generate Biomedicines
Lead asset technology: Anti-TSLP antibody
Disease areas: Immunology, infectious disease and immuno-oncology
Recent news: Entered into collaboration with Novartis to discover and develop protein therapeutics
After emerging from stealth in 2020, Generate Biomedicines has made notable progress as a leader in the field of generative biology, using AI to discover and develop innovative new drug candidates. The company's AI technology is called the Generate Platform, which works as a continuous loop to generate protein sequences to answer a specific therapeutic question, build computationally generated sequences as real proteins at scale, measure critical molecular characteristics and functions of generated proteins, and learn in order to drive improvement in the company's engine and every molecule that is generated.
Since its inception, Generate has expanded the Generate Platform into new modalities, including into bispecifics, enzymes, T‑cell engagers, and cell therapies. The company now has a robust pipeline across immunology, infectious disease, and immuno-oncology. Its lead asset is an anti-TSLP antibody called GB-0895 for the treatment of severe asthma that is currently in phase 1 studies.
The AI drug discovery company is considered to be a "biotech unicorn" and has raised a substantial amount of money since its emergence from stealth. When it bagged $273 million in series C financing in September 2023 – in the largest private biotech fundraising round of that month – it brought its total capital raised since 2020 to nearly $700 million.
Additionally, Generate has formed some high-profile partnerships with big pharma. It partnered with Amgen in 2022 to discover and create protein therapeutics for five clinical targets across several therapeutic areas and multiple modalities. More recently, in September last year, Generate entered into an agreement with Novartis in one of the biggest biotech deals of that month. The two companies are collaborating to discover and develop protein therapeutics across multiple disease areas with the help of Generate's generative AI platform.
Conclusion
The ten AI drug discovery companies profiled in this comprehensive review represent the cutting edge of pharmaceutical innovation, collectively demonstrating how artificial intelligence is fundamentally transforming the way we approach drug development. From Anima Biotech's mRNA Lightning.AI platform to Generate Biomedicines' generative biology approach, these companies are not merely incremental improvements on traditional methods—they represent a paradigm shift toward more intelligent, efficient, and successful drug discovery processes.
The financial validation of this sector is undeniable, with these companies having collectively raised billions of dollars and secured partnerships worth over $4 billion with pharmaceutical giants including Eli Lilly, Novartis, Sanofi, AbbVie, and others. The fact that major pharmaceutical companies are investing so heavily in AI partnerships signals their confidence that these technologies will become essential to remaining competitive in the modern drug development landscape.
Perhaps most significantly, we are witnessing the first generation of entirely AI-designed drugs entering clinical trials, with Insilico Medicine's INS018_055 breaking new ground as the first fully AI-discovered and designed drug to reach Phase 2 trials. This milestone, achieved in record time, validates years of technological development and investment while setting a new standard for what's possible in pharmaceutical development.
The diversity of approaches represented by these companies—from small molecule design and protein engineering to mRNA modulation and clinical trial prediction—illustrates the broad applicability of AI across the entire drug discovery value chain. Whether addressing oncology, immunology, neuroscience, or rare diseases, these companies are tackling some of healthcare's most challenging problems with unprecedented tools and methodologies.
Looking ahead, the success of these pioneering companies is likely to accelerate the adoption of AI throughout the pharmaceutical industry. As their platforms mature and their clinical candidates advance through trials, we can expect to see more rapid drug development timelines, higher success rates, and ultimately, more effective treatments reaching patients faster than ever before. The future of pharmaceutical development is being written by these ten companies and their AI-powered innovations, promising a new era of precision medicine and accelerated therapeutic discovery that will benefit patients worldwide.