The most innovative biotech companies of 2026

The Most Innovative Companies in biotech reflect a year in which companies took big swings—delivering breakthrough treatments to the clinic, bringing groundbreaking tests to the public, and building out the tools to support future breakthroughs.

eGenesis made headlines by transplanting genetically modified pig kidneys into humans—sustaining one patient for 271 days without dialysis, a record, with a second recipient still off dialysis since a June 2025 transplant—and scored FDA clearance for an expanded trial. Insilico Medicine backed up the promise of its AI drug design platform with Phase 2a data showing its lead therapeutic candidate, rentosertib, stabilized lung function in IPF patients. Replicate and Strand Therapeutics demonstrated the potential of next-generation RNA-based technologies for more durable vaccines and targeted cancer treatments, and Faeth Therapeutics saw positive results in a Phase 1b study of its novel tumor-starving drug Piktor.

It was also a big year for preventive screening and precision diagnostics: Exact Sciences, the developer of Cologuard test, launched Cancerguard, a multi-cancer blood test that can detect more than 50 types from one draw, along with a new tool for early detection of cancer recurrence—before Abbott acquired the company for $21 billion.

Biotech toolmakers such as Element Biosciences and Thermo Fisher Scientific launched new platforms to streamline multi-omics sequencing, high-throughput proteomics, and genetic sequencing for guiding cancer treatment. And Cambridge, Massachusetts–based Cellino Biotech announced a partnership with Mass General Brigham to pilot its modular cell-therapy manufacturing platform.

Techbio companies not only expanded platforms that enable fundamental discoveries at scale but rapidly advanced an array of therapeutic candidates into the clinic. Vivodyne’s automated “biological data centers” now test 10,000 human tissues at once, modeling drug toxicity for top pharmas like GSK. U.K.-based Basecamp Research added over 1 million previously undocumented species to its proprietary genomic dataset and, leveraging computing resources from Nvidia, used it to build a first-of-its-kind AI tool for designing custom gene-editing enzymes. And Enveda Therapeutics has expanded and capitalized on its unique dataset of botanical chemistry to advance three drug candidates into human trials—targeting atopic dermatitis, asthma, inflammatory bowel disease, and obesity—just six years after being funded.

1. eGenesis

For revolutionizing life-saving organ transplants

Currently, more than 800,000 Americans suffer from end-stage kidney disease, and over 550,000 are on dialysis. More than 100,000 Americans are on a transplant waitlist, but only about 17,000 people each year receive one. eGenesis, a Cambridge, Massachusetts–based startup cofounded by Harvard geneticist George Church, has an innovative solution to the transplant shortage: kidneys from genetically modified pigs.

Until now, efforts to transplant organs from pigs and others animals to humans have been hampered by the human immune system, which recognizes them as foreign. By using the precision gene-editing technology CRISPR, eGenesis has eliminated naturally occurring porcine genes that can trigger organ rejection and inserted human genes that suppress immune response and increase compatibility.

In 2025, the company completed two successful transplants of kidneys from its genetically modified pigs into volunteers with end-stage kidney disease. The first, 62-year-old Tim Andrew, went 271 days before the organ failed and he went back on dialysis, making him the longest living recipient of a pig kidney (at press time). The second recipient, 54-year-old Bill Stewart, received the transplant in June 2025 and was still living without dialysis as of press time.

Based on these outcomes, in September 2025, the FDA cleared eGenesis to start a large study in patients on the kidney transplant waitlist. 

In April 2025, the FDA also greenlighted a clinical trial of a dialysis-like system that uses a gene-edited pig liver outside of the body, a potentially life-saving bridge to a transplant.

Read more about eGenesis, No. 34 on Fast Company’s list of the World’s 50 Most Innovative Companies of 2026.

2. Exact Sciences

For bringing comprehensive cancer testing to the masses

Madison, Wisconsin–based Exact Sciences is the leader in noninvasive home screening for colorectal cancer in people at normal risk. Its Cologuard test has been used for more than 16 million completed screenings since its launch in 2014, and brought in estimated revenue of more than $2.5 billion in 2025.

In November, Exact Sciences announced the launch of a comprehensive cancer-screening blood test, developed at the company, called Cancerguard. Available as a laboratory-developed test in the United States, Cancerguard is the first commercially available multi-cancer early-detection test that analyzes cancer-related proteins and changes in gene expression to help detect more than 50 cancer types and subtypes from a single blood draw, including those that often go undiagnosed until later stages, when there are few treatment options.

To maximize access to Cancerguard, Exact Sciences has partnered with Quest Diagnostics to enable blood collection at more than 7,000 sites nationwide, including mobile phlebotomy services. In late November, the company announced that it would be acquired by Abbott in a deal valuing the company at approximately $21 billion.

3. Insilico Medicine

For bringing AI precision to small-molecule drug design

Lots of companies are promising to revolutionize drug discovery with AI. But Insilico Medicine, founded in 2014, is one of very few that is validating tangible benefits for patients. In 2025, the company published results from its Phase IIa trial of its lead asset, rentosertib, the first wholly AI-discovered and AI-designed small-molecule drug. The trial showed that patients receiving rentosertib—especially at higher doses—generally experienced stabilization or modest improvement in lung function instead of the typical decline seen in IPF, with a favorable safety profile.

Also in 2025, two Phase I trials of ISM5411, an AI-designed treatment for inflammatory bowel disease, yielded positive topline results, and Phase I trials of two more candidates—one targeting mesothelioma and other solid tumors, and another targeting MTAP-deleted advanced solid tumors—does their first patients.

In November, the company announced a $100 million research and licensing deal with Eli Lilly to advance Lilly’s AI-driven drug-discovery efforts. After raising $123 million in a March Series E, one of the largest AI-biotech raises of the year, Insilico started trading on the Hong Kong exchange on December 30, raising about $292 million.

4. Juvena Therapeutics

For helping patients keep muscle while losing weight

One of the best-known side effects of GLP-1 drugs for weight loss is the potential to lose beneficial muscle along with excess pounds. The first AI-discovered therapy from Redwood City, California–based Juvena Therapeutics, JUV-161, could be a game changer for people on those drugs and a critical tool for regenerating muscle and tissue in patients with muscle-wasting conditions.

Juvena kicked off Phase I clinical trials of JUV-161 in the treatment of myotonic dystrophy type 1 (DM1) in May 2025, after receiving FDA Orphan Disease Drug designation in 2024. A first-in-class muscle-regenerating endocrine therapy, JUV-16 aims to restore critical protein signaling to youthful, healthy levels in diseased muscle tissues. In addition to encouraging muscle growth, it modulates insulin resistance and has anti-inflammatory properties.

In June 2025, Juvena signed a deal with Eli Lilly with a potential value of more than $650 million to use Juvena’s AI screening platform to identify therapeutic candidates that improve muscle health and body composition—a potential complement to Lilly’s blockbuster weight-loss drug Zepbound and future products.

5. Enveda

For mining plant chemistry to build better medicines

While nearly half of all oral medicines in use today trace their origins to molecules first discovered in nature, more than 99% of nature’s chemistry is still hidden from science because isolating, identifying, and characterizing molecules from complex natural compounds has been historically slow and expensive.

By massively streamlining the process, using a combination of cutting-edge mass spectrometry for sample analysis and algorithms for interpreting the resulting data—Boulder, Colorado–based Enveda Therapeutics aims to unlock nature’s pharmacopeia for a new generation of cures.

In 2025, the company move several of its drug candidates through clinical trials. Its lead asset, ENV-294, a first-in-class oral anti-inflammatory, is being evaluated for the treatment of both atopic dermatitis and asthma, moving into Phase II trials for both after completing Phase I last year. Additionally, in December, the company dosed its first patient in a Phase I trial for a once-daily oral therapy targeting obesity and muscle preservation. The same month, the FDA cleared a Phase I trial of ENV-6946, a first in class “multi-biologic in a pill” for the treatment of inflammatory bowel disease. All are based on natural molecules found in botanical samples.

So far, says CEO Viswa Colluru, Enveda’s program has come from analyzing fewer than 100 plants. The company is now up to about 400, and expects to hit 12,000 by the end of 2026—what will be the world’s largest chemical map. After raising $150 million in a February Series C, including $20M strategic investment from Sanofi, Enveda closed a Series D in September, which, valued the company at more than $1 billion.

6. Replicate Bioscience

For showing the potential of self-replicating RNA in metabolic disease and beyond

San Diego-based Replicate Bioscience is leading the way in exploring the use of self-replicating RNA (srRNA) to overcome key limitations of current mRNA vaccines and therapeutics. Replicate’s platform is based on next‑generation srRNA vectors that self‑amplify for a limited period, so cells produce therapeutic proteins at much higher mRNA levels than usual while using minimal synthetic RNA input. The vectors can also encode multiple mutations or antigens, supporting applications in immuno‑oncology, vaccines, and autoimmune diseases.

In February 2025, Replicate announced positive results from a Phase II trial of its lead asset, RBI‑4000, an srRNA rabies vaccine that has shown protective immunity at doses lower than any other reported mRNA or srRNA rabies vaccine.

In August 2025, Replicate signed a multiyear research collaboration agreement allowing Novo Nordisk to leverage its proprietary srRNA technology to develop novel therapies targeting obesity, type 2 diabetes, and other cardiometabolic diseases. Replicate Bioscience will receive research funding and is eligible to receive up to $550 million in payments. In January 2026, the Gates Foundation awarded the company $7 million to develop candidate therapies for malaria and HIV.

7. Strand Therapeutics

For programming mRNA therapeutics that help the immune system fight cancer

Stand Therapeutics makes programmable, self-replicating mRNA medicines that behave like tiny computer programs. By embedding “logic circuits” into mRNA molecules, Strand can design drugs that turn on only in specific cells, allowing the production of an immune-boosting protein only in cancer cells, for example, while staying silent in healthy cells. The approach provides unprecedented control over where, when and how much protein is made.

The company’s lead candidate, STX‑001, uses mRNA injected directly into a tumor; it carries instructions for cells to make interleukin‑12 (IL-12), a protein that revs up the immune system. Data from ongoing phase 1 trials released in 2025 showed encouraging signs of antitumor activity as a standalone therapy in patients with certain solid tumors, including melanoma Several patients whose cancer had stopped responding to drugs saw visible disease disappear completely. Because the mRNA produces IL‑12 only in the tumor, this treatment has fewer side effects than traditional ones.

Strand’s next program, STX‑003, aims to show that the same principle works when the drug is injected through the bloodstream, with the mRNA load only turning on inside of tumors. This would open the door to treating tumors that are hard to reach directly with a needle, such as lung cancers. STX‑003 is expected to enter human testing this year. In August 2025, Strand raised $153 million in new funding, from Investors included Kinnevik, Regeneron Ventures, ICONIQ, AmgenVentures, and Eli Lilly.

8. Cellino Biotech

For bringing cell and tissue manufacturing into the future

Cell-based therapies hold enormous potential across many chronic degenerative diseases. But manufacturing autologous stem cell treatments, which are derived from a patient’s own cells, has been a complex, time-consuming challenges in biomanufacturing relying on a small number of experts.

With its automated, modular biomanufacturing platform, Cambridge, Massachusetts-based Cellino Biotech aims to overcome these challenges, making the production of high-quality iPSCs for clinical trials and commercial therapies more precise, reproducible, and scalable. Cellino’s Nebula system grows cells in a sterile, closed cassette, and uses cameras, AI algorithms, and lasers to continuously monitor and control cell growth and cell density.

In February 2025, the company announced a partnership with Mass General Brigham to build what it calls the world’s first hospital-based “foundry” for iPSCs enabling on-site production of personalized cell and tissue therapies. In May, Cellino’s platform received FDA Advanced Manufacturing Technology designation for safety and innovation, making it one of just two companies to get the certification. In 2025, the company announced collaborations with Matricelf, focused on celll-based treatments for spinal cord injury, and with Karis Bio, to explore regenerative therapies for cardiovascular disease, the world’s leading cause of death.

9. Faeth Therapeutics

For fighting endometrial cancer with a three-pronged approach

In 2025, San Francisco-based Faeth Therepeutics made significant progress advancing therapies that fight cancer by targeting tumor metabolism. Its core principle: cutting the fuel supply weakens the disease.

The company’s lead asset, PIKTOR, combines two inhibitors of tumor growth which work together to shut down one of the main circuits determining how cancers grow and consume fuel. Mutations in this pathway are common across many types of solid tumors, including endometrial, breast, lung, and ovarian. Attempts to block it with a single drug have often failed to yield a durable response.

A phase 1b study of PIKTOR, alongside the chemotherapy paclitaxel as a treatment for endometrial cancer, showed a remarkable 80% overall response rate and median progression-free survival of 11 months, versus the historical three to four months with chemotherapy alone. A phase 2 trial is currently enrolling patients. The novel mechanism of the drug is enhanced with an insulin-suppressing diet designed to deprive the cancer of a main fuel source.

Beyond cancer, Faeth’s program in Tyrosinemia Type I, a rare pediatric disorder that can lead to cognitive decline, is expected to advance into phase 1 clinical trials this year. In October 2025, the company raised $25 million in funding led by S2G Ventures, with Khosla Ventures and others, bringing total capital raised to $92 million.

10. Orca Bio

For making high-precision cancer immunotherapies more scalable

For many patients with multiple myeloma, leukemia, and other blood cancers, stem cell transplants from donors offer the best chance of survival. But patients have a high likelihood of developing chronic graft-versus-host disease (GVHD) and eventual organ failure.

Orca Bio has developed an alternate approach that aims to reduce the risk of GVHD. The company’s Orca-T is a precision immunotherapy that’s composed of a mixture of highly purified donor regulatory T cells, purified stem cells that give rise to other blood cell types, and conventional T cells from a donor. The idea is to minimize T cells that drive GVHD, while preserving enough conventional donor ones to maintain graft‑versus‑leukemia effect, in which donor immune cells recognize and eliminate residual malignant cells the recipient.

The therapy also uses a novel, proprietary method to conditioning a patient to receive the graft, requiring just one immunosuppressive drug rather than the standard combination treatment, which is linked to toxicity issues.

In a phase 3 study of patients with multiple types of blood cancer, announced in March, Orca-T doubled the rate at which patients survived without developing chronic graft-versus-host disease at one year, compared with conventional alloHSCT. An FDA approval decision is set for early April.

To prepare for commercialization, Orca Bio has built a centralized 100,000 square foot commercial manufacturing facility to produce its cell therapies scale. In December 2025, the company announced a Series F financing round led by Lightspeed Venture Partners.

11. Epicrispr Biotechnologies

For pioneering treatments that control gene expression to help people with neuromuscular disease

Epicrispr’s epigenetic editing platform is designed to activate or repress gene targets without cutting DNA, offering a potentially safer and more durable approach to treating diseases caused by aberrant genes.

After granting it Fast Track, Rare Pediatric Disease, and Orphan Drug designations, in April 2025, the FDA cleared Episcripr to launch a phase 1/2 clinical trials of EPI-321, its treatment for facioscapulohumeral muscular dystrophy (FSHD). This is the first trial of an epigenetic editing therapy for a neuromuscular disease, and the first and only open epigenetic editing Investigational New Drug (IND) authorization in the United States.

In January 2026, Epicrispr shared results from the first three patients to complete the study. Across these participants, researchers observed improvements in a broad range of strength and functional measures, which generally exceeded outcomes observed in an external comparator cohort. No serious adverse events and no severe adverse events have been reported to date among study participants.

In March 2025, the company closed a $68 million Series B funding round, led by Ally Bridge Group, with participation from SOLVE FSHD, a venture philanthropy organization founded by Chip Wilson, founder of Lululemon Athletica and an FSHD patient, along with other new and existing investors.

12. Vivodyne

For enabling researchers to get “human data” on new drugs—before clinical trials

University of Pennsylvania spinout Vivodyne wants to reduce reliance on animal studies, with its highly automated laboratories that use living human tissue, in various states of health and disease, to realistically predict drug response.

In 2025, the company finished CK building out biological data centers in San Francisco and Philadelphia, 48,000 square feet in all, where robotic systems can cultivate, dose, and analyze more than 10,000 individual human tissues simultaneously.

The tissue samples actively metabolize drugs, develop immune responses, and exhibit emergent tissue-level behaviors over weeks. This allows researchers to monitor disease progress and the development of drug resistance or toxicity—how cardiac tissue develops arrhythmias from drug accumulation, for example, or how tumors recruit blood vessels to resist therapy and immune cells infiltrate organs to drive inflammation.

A majority of the top 10 pharmaceutical companies are running experiements at what Vivodyne calls its “biological data centers.” In December, Vivodyne and GlaxoSmithKline published a peer-reviewed study in Cell Stem Cell showing that its bone-marrow models accurately identified dose-linked toxicity in preclinical studies. In May, the company raised $40 million from Khosla Ventures and other investors.

13. Opus Genetics

For focusing on a therapy to restore vision in congenitally blind children

In 2025, patient-founded Opus Genetics advanced its mission of developing new therapies that restore and protect vision. In April 2025, the company announced the first pediatric data from their Phase 1/2 trial of a one-time gene therapy for Leber congenital amaurosis type 5 (LCA), a rare inherited eye disease that appears at birth or in the first few months of life and affects about 1 in 40,000 newborns.

After receiving a subretinal injection in one eye, the majority of patients in the trial had clinically meaningful gains in cone‑mediated vision, visual acuity, retinal sensitivity, and functional navigation. In May 2025, 12-month data from adults with condition confirmed durable vision restoration, leading to FDA Regenerative Medicine Advanced Therapy designation, which is given to drug candidates intended to treat serious or life-threatening conditions under the 21st Century Cures Act that allows for accelerated approval based surrogate or intermediate endpoints.

In August, the FDA cleared another therapeutic candidate to start clinical trials. OPGx-BEST1, an adeno‑associated virus–based gene therapy that targets macular dystrophy caused by pathogenic variants in the BEST1 gene. In addition, the company reported positive phase 3 data for VEGA-3, the company’s phentolamine eye drops for treating presbyopia, blurry vision at close range associate with aging.

14. Gilgamesh Pharma

For making psychedelic medicine more manageable

Psychedelic therapies could be transformative for conditions including depression, PTSD, and substance-abuse disorders. But their unpredictability and a psychoactive affects that can endure for several hours represent a significant hurdle to their approval and practical deployment in clinical settings. Gilamesh Pharma’s lead drug candidate, a next-generation psychedelic called bretisilocin could be the breakthrough that the field has been waiting for.

Bretisilocin is a synthetic tryptamine psychedelic in the same broad family as short-acting DMT and long-acting psilocybin. By making a few slight chemical alterations, Gilgamesh scientists developed a treatment with psychoactive effects that last 60-90 minutes—midway between DMT and psylocybin—and that increases antidepressant neuroplasticity without the usual intense hallucinations. In phase 2 trials, patients with major depressive disorder. showed a 94% remission rate within 30 days of receiving the drug intravenously. In August 2025, Abbvie secured the rights to bring bretisilocin to market, in a deal worth up to $1.2 billion.

Other candidates in Gilgamesh’s pipeline include GM-3009, an ibogaine-inspired analog designed to treat opioid use disorder, PTSD, and traumatic brain injury. And in January, the company announced positive topline results from its phase 2a study of Blixeprodil, an NMD receptor antagonist that is structurally related to ketamine, which demonstrated rapid, robust, and durable antidepressant effects in patients with moderate-to-severe major depressive disorder.

15. Element Biosciences

For simplifying the sequencing of DNA, RNA, protein, and cell morphology all at once

In the past several years, sequencing applications have expanded beyond simply analyzing DNA at the organism level to focus increasingly on single-cell multiomics, which reveals how DNA expression varies across specific cell types and populations. That has created a demand among researchers for new, more nimble tools that help illuminate a range of complex molecular processes.

Element Biosciences is leading the way. Its AVITI24 5D Multiomics System, first delivered to customers in late 2024, is the first and only truly integrated multiomics system available today. It delivers spatial, single-cell multiomics—capturing DNA, RNA, protein, and cell structural features—all in one run, capturing high-quality data affordably. It includes Direct-in-Sample Sequencing, the first technology to enable a true “sample in, answer out” workflow—eliminating traditional library prep and allowing researchers to load samples directly and begin sequencing at the push of a button.

Element’s systems are now installed in more than 40 countries worldwide, and cited in 300-plus peer-reviewed publications, reflecting rapid adoption across academic, clinical, and biopharma research. The company has ongoing collaborations with SOPHiA Genetics, Twist Bioscience, and Qiagen, among others.

16. Argo Biopharma

For putting Chinese biotech innovation on the map

Until recently, China’s biotech industry has been built around providing contract research offices for foreign drug makers. But that paradigm is shifting, as illustrated by a flurry of 2025 dealmaking between global pharma players and Chinese biotech startups. By value, nearly a third of pharmaceutical out-licensing deals worldwide in the first half of 2025 involved China-made assets, according to analysts at Jefferies.

Shanghai-based Argo Biopharma, founded in 2021, is a clinical-stage company developing next-generation RNA interference (RNAi) therapeutics for chronic diseases including cardiovascular, metabolic, and rare disorders. Cofounders Dr. Dongxu Shu  and Dr. Patrick Shao (started the company after working on siRNA delivery platforms at U.S. RNAi specialist Arrowhead Pharmaceuticals.

In September 2025, the company announced a collaboration with Novartis worth up to $5.2 billion. The collaboration focuses on the development of RNAi drugs targeting ANGPTL3, a protein linked to cardiovascular and metabolic diseases. The company hopes to leverage Argo’s proprietary RNA platform in therapies that require just one injection per year for certain indications. Under the terms of the agreement, Novartis will gain exclusive global rights to develop and commercialize Argo’s RNA-targeting therapies for ANGPTL3. This is one of several partnerships Novartis has formed with Chinese biotech firms in recent years.

17. Absci

For bringing AI-designed antibodies to the clinic

With its integrated drug development platform—leveraging its own wet-lab data and generative AI—Vancouver, Washington-based Absci (Nasdaq: ABSI) is designing novel antibodies and biologics from scratch, claiming shorter development timelines and a higher success rate than conventional approaches.

In May 2025, the company announced the first patients dosed in its phase 1 trials of ABS-101, an AI-designed antibody for inflammatory bowel disease. Interim data shared in November showed a favorable safety profile and an extended half-life compared to first-generation drugs targeting inflammation.

The company is also company is doubling down on a second asset, ABS-201, a first-in-class potential treatment for baldness. In December 2025, it announced the first patients dosed in phase 1/2a trial of the injectable therapy. The company plans to pursue trials of ABS-201 into patients with endometriosis.

In August 2025, Absci expanded a collaboration with Barcelona-based Almirall to use its AI platform to target dermatological indications. And in September, the company announced a collaboration with Oracle Cloud Infrastructure and Advanced Micro Devices that will let it leverages OCI’s AI infrastructure and AMD’s latest hardware to consolidate its infrastructure, run large-scale molecular-dynamics simulations, and accelerate its biologics design cycles.

18. Thermo Fisher Scientific

For empowering scientists with next generation sequencing

In 2025, Thermo Fisher Scientific—the world’s leading provider of scientific instruments, laboratory products, and speciality diagnostics—announced significant new approvals and partnerships that impact clinical workflows in oncology and support ongoing large-scale research projects.

In July, the FDA approved the company’s Oncomine Dx Express Test on its Ion Torrent Genexus Dx next-generation sequencing platform as a companion diagnostic for Dizal’s lung cancer drug Zegfrovy and for tumor profiling. Unlike older methods that could only look at one or a few genes at a time, the platform allows rapid sequencing of a large number of genes to identify the specific genetic mutations that are driving a patient’s cancer, delivering results in as little as 24 hours.

Early in the year, the company announced that its Olink Explore platform, a high-throughput system for proteomics (analyzing an entire set of proteins), was selected by the UK Biobank Pharma Proteomics Project to support the world’s largest human proteomics study ever. The project aims to analyze more than 5,400 proteins from 600,000 samples to find new protein biomarkers that can be used to predict, diagnose and treat diseases.

In June, the Regeneron Genetics Center selected Thermo Fisher’s next-generation for a large-scale proteomics investigation involving nearly 200,000 patient samples. In April, the company announced a $2 billion investment in US operations over the next four years to bolster domestic innovation and manufacturing, consisting of $1.5 billion in capital expenditures to expand manufacturing and $500 million towards R&D aimed at supporting high-impact innovations.

19. Basecamp Research

For massively expanding the library of known protein sequences

In 2025, UK-based techbio startup Basecamp Research massively expanded its digital “tree of life,” with the integration of over 1 million previously undocumented species into its BaseData proprietary dataset. Billed as “the world’s largest ethically-sourced and globally representative biological dataset,” it now contains more than 10 billion unique protein sequences, a tenfold gain over all existing public databases combined.

In September 2025, Nvidia CEO Jensen Huang announced a direct investment in Basecamp Research as part of a £2 billion ($2.7 billion) national AI initiative in the UK. The GPU maker also provided Basecamp with the computing infrastructure to build out a new family of evolutionary AI models called EDEN. By January 2026, that collaboration had already yielded a breakthrough—an AI platform called AI-Programmable Gene Insertion. The platform could help overcome key challenges with CRISPR-based technology, which now can only make small edits and must damage DNA to do so, limiting its use. Basecamp Research is the first to demonstrate that AI can design enzymes capable of performing large gene insertion at defined sites in the human genome, key to truly programmable therapies. 

In the lab, the platform was able to design multiple gene-insertion enzymes to target specific diseases in the human genome with a 100% success rate. If the approach proves robust beyond the lab, it could significantly expand the scope of cell and gene therapies for cancer and inherited diseases. The same AI model helped University of Pennsylvania scientists design novel antimicrobial peptide molecules with a 97% lab-confirmed success rate, including candidates that appear potent against multidrug-resistant “superbugs.” Basecamp Research partners with biopharma companies and academic institutions worldwide, and has more than a dozen pilot partnerships in industrial biochemistry on track to progress to full commercial programs.

20. Manifold Bio

For inventing a novel way to track drug distribution in living systems

With its innovative protein-barcoding technology, Boston startup Manifold Bio aims to reduce the number of animals that are needed for pharmaceutical R&D while increasing the amount learned from each experiment by several orders of magnitude.

The company’s M-Code technology—which builds on tools developed in the lab of George Church at Harvard—uses unique, inert peptide sequences, or tags to track where certain therapeutic proteins end up in an animal’s tissues. This yields more useful information about drug delivery than testing in cell cultures can.

The platform can be used to tag up to 100 or more proteins simultaneously, which can be injected at one time into a lab animal. Later, the peptide “barcodes” are converted to DNA barcodes via a proprietary enzymatic process and next-generation sequencing is used to reveal precise details about multiple ways the proteins behave. This parallel readout helps to identify top-performing candidates quickly, and this data is fed back into the company’s AI platform.

The company has already lined up pharma customers including Roche, which in November announced a collaboration with Manifold to work on engineered proteins that are capable of “shuttling” neurological treatments across the blood-brain barrier. The deal is worth up to $2 billion.

Explore the full 2026 list of Fast Company’s Most Innovative Companies, 720 honorees that are reshaping industries and culture. We’ve selected the companies making the biggest impact across 59 categories, including advertising, applied AI, biotech, retail, sustainability, and more.