These 10 biotech companies are changing how we discover new drugs and treat complex diseases

This year’s list of biotech companies reflects the evolving impact of AI, a growing interest in cutting-edge research tools for single-cell analysis and spatial proteomics, and clinical advances for novel drug candidates that promise more convenient and effective treatment for conditions impacting millions of Americans.

AlzPath’s highly sensitive blood test can detect signs of Alzheimer’s disease before symptoms develop—and in time to potentially benefit from new treatments. Genomic sequencing specialist GeneDx is helping parents discover and treat hundreds of rare diseases in newborns. 10x Genomics and Syncell streamline cutting-edge analytics for life science research, while Nabla Bio is using generative AI to design completely new antibodies.

Protagonist Therapeutics is partnering with Janssen on an oral peptide for treating plaque psoriasis, aiming to expand the use of these powerful drugs beyond obesity. And Recursion is harnessing the largest supercomputer in pharma to power a drug discovery platform used by Big Pharma customers, while advancing multiple AI-based candidates of its own through clinical trials.

1. ALZpath

For using a simple blood test to diagnose Alzheimer’s at an earlier stage

Earlier diagnosis of Alzheimer’s disease is seen as increasingly important and worthwhile as a focus for new medications. Currently, AD is typically diagnosed via cognitive testing alongside expensive PET brain scans or invasive cerebrospinal fluid (CSF) tests, which are costly and painful. But recently, a much less invasive blood test has demonstrated comparable accuracy to these methods. ALZpath has developed a proprietary antibody that, when utilized in blood-based tests, can detect tiny concentrations of the biomarker p-tau217, which has long been recognized as a reliable sign of Alzheimer’s but previously could not be identified via blood test. Concentrations of p-tau217 can signal the accumulation of proteins that cause Alzheimer’s years before the onset of cognitive symptoms, making ALZpath’s test an option for earlier diagnosis and treatment.

In March 2024, Neurocode, a clinical laboratory that tests for neurological disorders, became the first U.S. lab to make the test available for clinical diagnostic use, clinical trials, and other research purposes. In June 2024, Roche licensed the ALZpath p-tau217 antibody to develop and commercialize an Alzheimer’s disease in-vitro diagnostic test. The Roche p-tau217-based test was recently granted Breakthrough Device designation by the FDA and will be commercialized as part of an ongoing collaboration between Roche and Eli Lilly. In 2024, ALZpath reached the milestone of obtaining over 50 active partners across pharma, diagnostics, and academia, and has seen a 130% increase in revenue from 2023.

2. GeneDX

For offering diverse newborn populations genomic screenings for rare diseases

The average time for accurate diagnosis of a rare disease is about five years, but in some populations it may take up to a decade. GeneDx aims to shorten that diagnostic journey and get patients the care and treatments they need sooner. In 2024, the company—a leader in genome and exome testing for rare diseases—provided the large-scale sequencing and reporting for the largest study of its kind on genomic screening for rare diseases in newborns.

A collaboration with Columbia University Irving Medical Center, New York-Presbyterian, the New York State Department of Health, and Illumina, the GUARDIAN (Genomic Uniform-Screening Against Rare Diseases in All Newborns) study aims to screen 100,000 newborns in a diverse population for more than 450 actionable genetic conditions not currently included in standard newborn screenings. The study, which is open to babies born at New York-Presbyterian Hospitals, has sequenced more than 17,000 newborns to date. Preliminary results from the first 4,000 newborns, published in October 2024, found that 3.7% of newborns had one of these rare conditions, the majority of which would not have been picked up by traditional newborn screening.

GeneDx has also partnered with the New York Center for Rare Diseases at Montefiore, PacBio, and Google Health to deliver genetic diagnoses for Bronx families living with rare diseases. The company’s customers today typically include clinical geneticists, genetic counselors, pediatric neurologists, developmental pediatricians, and neonatologists, but the goal is to make genomic newborn screening the standard of care nationwide and to have more general pediatricians use genomic screening as a first-line test for children with rare-disease symptoms. The company’s 2024 full-year revenue of $302.3 million was a 56% year-over-year increase over 2023.

3. 10x Genomics

For bringing speed and efficiency to single-cell analysis

In 2024, 10x Genomics worked to cement its status as “the single-cell analysis company,” making upgrades and additions to its portfolio of tools used by researchers in academia and industry to understand molecular activity in the three-dimensional space of cells and tissues.

“Single-cell” analysis refers to the study of genomics, transcriptomics, proteomics, and cell-cell interactions at the level of the individual cell, as opposed to conventional methods that study bulk populations of many cells. This is important because many diseases, particularly cancer, are driven by changes in particular cells, not all cells; precision treatments require pinpointing those that really matter.

In 2024, 10x Genomics launched 10 new products and capabilities across its single-cell and spatial platforms, including GEM-X, an innovative microfluidic chip design that allows for significantly improved sensitivity and efficiency compared to previous versions, enabling researchers to detect up to two times more genes for a more than twofold reduction in cost per cell analyzed. Another new product, the Visium HD Spatial Gene Expression Assay, enables users to layer cell-level gene-expression data on top of histology (tissue) images, with high-resolution, to pinpoint drug targets, for example.

10x’s tools are used in several major collaborative research efforts, including the Human Cell Atlas, the Chan Zuckerberg Initiative, and NIH’s BRAIN Initiative Cell Census Network (BICCN) project.

4. Protagonist Therapeutics

For expanding peptide-based therapies beyond obesity

Protagonist Therapeutics makes novel drugs based on peptides—the building blocks of proteins, and the basis of drugs including insulin and obesity medications like Wegovy and Zepbound. Because peptides are potent, selective in their action, and safe, there is growing interest in their potential therapeutic applications in many areas. But it has been extremely difficult to deliver effective peptide treatments in oral form, because they break down rapidly in the gut.

Protagonist’s lead drug candidate, Icotrokinra (JNJ-2113), is an oral peptide that was jointly discovered with scientists from Johnson & Johnson and saw positive topline results in two phase 3 clinical trials for treating moderate to severe plaque psoriasis. Three more phase 3 studies are ongoing, with results expected in Q2 2025. Additional preliminary results from a phase 2b study in ulcerative colitis are expected in Q1 2025.

Protagonist’s other leading drug candidate, Rusfertide, is an injectable synthetic peptide that mimics the natural hormone hepcidin, which helps regulate how the body processes iron. In January 2024, Takeda and Protagonist announced a global license and collaboration agreement to develop and commercialize Rusfertide as a treatment for blood cancer polychthemia vera, which causes an overproduction of red blood cells. Protagonist reported over $259 million in license and collaboration revenue for the first half of 2024. Net income of nearly $177 million for the first half of 2024 was a significant turnaround from a net loss of $72.2 million in the same period in 2023.

5. Recursion

For mapping a path for AI-driven drug discovery

Founded in 2013, Salt Lake City-based Recursion was a techbio company before there was such a thing. In 2024, it made big moves to stay at the forefront as research and discovery platform for biopharma customers and as a company with multiple clinical-stage therapies of its own.

In January 2024, the company launched its LOWE (LLM-Orchestrated Workflow Engine) platform, which provides a natural language interface for complex drug discovery tasks. Powered by Recursion’s proprietary biological and chemical data, it can orchestrate experiments using the company’s automated wet laboratories. In May, a partnership with NVIDIA allowed the company to upgrade its “tech stack” with an on-premise supercomputer called BioHive-2, which ranked number 35 in the world in 2024 and is believed to be fastest computer in the biopharmaceutical space. In August, Recursion’s first neuroscience “phenomap”—which shows chemical and genetic alterations in neuronal cells—was optioned by Roche Genentech for $30 million as part of a fee structure that could exceed a total of $500 million across multiple maps.

Recursion made steady in its own pipeline, too: In October 2024, the company announced the first patient dosed in its phase 2 clinical trial of REC-3964, a potential first-in-class, nonantibiotic treatment for recurrent Clostridioides difficile (C. diff) infection, and in December, the start of a phase 1/2 trial of REC-1245, a potential first-in-class therapy, called an RBM39 degrader, for certain solid tumors and lymphoma.

In late November, Recursion completed the acquisition of a former rival in AI-driven drug development, Exscientia, bringing the company’s total number of in-house drugs in preclinical and clinical development to 10.

6. Basecamp Research

For recording Earth’s microbial biodiversity and sharing the benefits

Less than 1% of the Earth’s genetic biodiversity has been recorded. But U.K.-based Basecamp Research is working to expand our knowledge of what’s out there. By conducting more than 125 research expeditions across five continents so far, the company has assembled what it bills as the world’s largest genomic database of biodiversity, comprising genetic sequences and protein data from microbes collected in volcanic vents, caves, oceans, jungles, and other extreme and remote ecosystems around the world.

The Basecamp platform integrates environmental data like temperature, pH, and other chemical conditions alongside protein sequences, providing context for understanding protein function. It uses AI models to help customers in pharmaceuticals, materials science, consumer goods, and other areas by identifying and designing novel proteins for drugs, industrial enzymes, and biomaterials. The company licenses its platform to clients and has partnerships with more than 50 companies and research institutions, including Sanofi P&G, and U.K. chemicals company Johnson Matthey.

Basecamp Research has also worked to ensure that the benefits of its discoveries are shared fairly with the communities whose native biodiversity feeds the platform, by tracing the source of proteins that clients use to develop products, and making royalty payments to these communities starting in the R&D phase. At the UN Biodiversity Conference in Montreal in August 2024, Basecamp announced a new, landmark bioprospecting deal with the nation of Cameroon, which in addition to royalty payments provides nonmonetary benefits including training of Cameroonian scientists and academics, sequencing equipment, data for soil and environmental monitoring, and education programs in the areas where the company collects samples. In October, the company raised a $60 million Series B.

7. Nabla Bio

For harnessing AI to build truly novel antibodies from scratch

So-called multipass membrane proteins, which comprise two-thirds of all cell surface proteins and play a major role in controlling cell behavior, represent some of the most promising targets for antibody drugs. But developing antibodies that recognize these proteins has been notoriously difficult. As of September 2023, only three multipass membrane proteins had been successfully targeted by antibodies. Nabla’s generative protein-design platform promises to unlock hundreds of these previously inaccessible targets, potentially doubling the number of disease-relevant drug targets the industry goes after. This fall, Nabla released a technical paper demonstrating the successful use of its AI platform to design several therapeutic antibodies “from scratch,” given just a target protein sequence and/or structure—the first evidence of computationally designed antibodies that can target a subset of these proteins called G protein-coupled receptors. On the promise of its platform, Nabla has inked more than $550 million in total deals with Big Pharma customers including AstraZeneca, Bristol Myers Squibb, and Takeda. Since launching in 2021, the Cambridge, Massachusetts-based company has raised $37 million in venture funding, including a $26 million Series A round in January 2024.

8. DarwinHealth

For matching cancer patients with experimental treatments

Today, overall cure rates for advanced lung, breast, and colon cancer are typically less than 50%. Finding a plan B for patients when standard-of-care therapy has failed comes down to high-stakes and time-consuming guesswork to navigate a maze of potential treatments. There are 250 FDA-approved cancer drugs and 200 different types of cancer—meaning, there are tens of thousands of possible drug-cancer combinations and billions more if you combine multiple drugs.

DarwinHealth uses AI—its VIPER algorithm—to take experimentally measurable data from a patient’s tumor, such as gene expression profiles, and make predictions about which existing drugs are most likely to be effective against the “Tumor CheckPoint” proteins driving their particular type of cancer. In 2024, DarwinHealth introduced innovations across its platform, speeding its adoption in multiple clinical trials in rare and treatment-orphan malignancies (including renal carcinoma, gastroenteropancreatic neuroendocrine tumors, breast cancer, prostate cancer, and pancreatic carcinoma), and in drug development, where customers including BMS have used it analyze more than 1,500 oncology-related compounds. Trial results published in 2024 showed 100% accuracy of the DarwinOncoTarget tool at predicting response to drugs called HDAC6 inhibitors in breast cancer, and a 91% accuracy of DarwinOncoTreat at predicting response of multiple tumor types. Estimated revenue doubled from $4M in 2023 to $8 million in 2024, and biopharma partnerships increased from 3 to 8.

9. IO Biotech

For developing a one-two shot of immunotherapy for melanoma

Copenhagen-based IO Biotech is developing novel dual-action cancer vaccines that harness the immune system by directly stimulating immune T cells as well as important immune-suppressive cells in the tumor microenvironment. Unlike mRNA vaccines that are personalized to each patient, IO’s peptide-based vaccines are “off-the-shelf,” meaning patients can start treatment more quickly. The treatment could offer an alternative with less potential for systemic toxicity than RNA-based cancer vaccines (one of which, from Moderna, is in phase 3 trials). IO’s lead candidate, IO102-IO103, for the initial treatment of advanced melanoma, has shown promising clinical efficacy and safety in phase 1/2 studies in combination with Keytruda, demonstrating an 80% overall response rate and 50% complete response rate. This data led to FDA Breakthrough designation and allowed the company to move directly into phase 3 trials. Phase 2 trials of IO102-IO103 in combination with Keytruda are also underway in lung and head and neck cancer patients with solid tumors. In December, IO Biotech entered a loan agreement of up to €57.5 million from the European Investment Bank, the long-term lending institution of the European Union, to fund further development and pre-commercialization work.

10. Syncell

For speeding up spatial proteomics discovery

In 2024, life science tools startup Syncell launched its ultra-high-speed, real-time targeted microscopy-based illumination platform called Microscoop, which offers a new level of precision for researchers studying spatial proteomics—three-dimensional protein expression—in cancer and other diseases. Syncell’s Microscoop platform technology uses microscopy-guided photo-labeling to isolate proteins from disease-related regions of interest, followed by mass spectrometry analysis, to identify novel proteins that could serve as targets for new drugs.

Syncell offers a complete system—including proprietary reagents that are used to prepare cell and tissue samples for labeling and analysis. The technology is applicable to a broad scope of biological problems, including the identification of new disease biomarkers and novel targets for drugs. It has already been used to enable the discovery of novel protein constituents in beta amyloids associated with Alzheimer’s disease, TDP-43 aggregates associated with ALS disease, a marker of triple negative breast cancer, and more. In December, the Taipei and Watertown, Massachusetts-based company raised a $15 million Series A financing round led by Taiwania Capital.

Explore the full 2025 list of Fast Company’s Most Innovative Companies, 609 organizations that are reshaping industries and culture. We’ve selected the companies making the biggest impact across 58 categories, including advertising, applied AI, biotech, retail, sustainability, and more.