The Healthcare Technology Report is pleased to announce The Top 25 Biotechnology Companies of 2024. Innovation across the biotechnology sector is occurring at an increasing rate now that more advanced tools such as artificial intelligence have become ubiquitous. As in recent years, the number and size of biotechnology companies continue to grow markedly faster. In parallel, the commercialization of new biotech products has ushered in a unique era for healthcare technology, making groundbreaking treatment options more accessible than ever before.
Among this year’s awardees, we highlight BeiGene, a company focused on oncology medications that have already treated almost one million individuals. Meanwhile, CareDx, a medical solutions company focused on transplant patients, continues to grow with its first commercial product, AlloMap® Heart, which is now used in more than 90 percent of transplant centers in the United States. We also feature Sage Therapeutics, a cutting-edge biopharmaceutical company that has developed the only two FDA-approved treatments for postpartum depression.
The companies on this year’s list serve as an impressive reminder of the life-changing effects cutting-edge technology can have on diagnosing and treating life-threatening illnesses in addition to improving people’s well-being. Please join us in recognizing the accomplishments of The Top 25 Biotechnology Companies of 2024
1. Clario
For over 50 years, Clario has established itself as a leading healthcare research and technology company, headquartered in Philadelphia. The company specializes in generating clinical evidence for pharmaceutical, biotech, and medical device partners globally. With a comprehensive endpoint technology platform covering eCOA, cardiac safety, medical imaging, precision motion, and respiratory endpoints, Clario has facilitated over 27,000 trials and secured over 500 regulatory approvals since 2012. Operating in 30 offices in nine countries, the company has established a truly global footprint, impacting over five million patients in 120 countries, and helping to introduce more than 800 new drugs to the market. They’ve made impactful advances in various therapeutic areas, including cardiovascular and metabolic, neuroscience, musculoskeletal, oncology clinical trials, and hepatology.
In recent collaborations, Clario partnered with CellCarta to enhance clinical trial sample management and with Trial Data in China to advance decentralized clinical trial methodologies. Notably, Clario's Precision Motion Opal® technology contributed to a University of Oxford breakthrough in Parkinson's Disease, enabling early disease progression detection.
2. BeiGene
BeiGene, a global biotechnology company founded in 2010 by Xiaodong Wang and CEO John V. Oyler, specializes in discovering and developing innovative oncology treatments to enhance affordability and accessibility for cancer patients worldwide. With a diverse portfolio, BeiGene expedites the development of novel therapeutics through internal capabilities and collaborations, aiming to radically improve medicine access for patients in need. The company boasts a global team exceeding 10,000 colleagues in more than 30 markets across five continents, with administrative hubs in Basel, Beijing, and Cambridge, MA. More than 800,000 patients have been treated with BeiGene’s medicines.
Recent milestones include the European Commission's approval of BRUKINSA® (zanubrutinib) for Relapsed or Refractory Follicular Lymphoma, along with FDA approval for a label update in Chronic Lymphocytic Leukemia. BeiGene's partnership with Ensem Therapeutics signals its commitment to advancing differentiated CDK2 inhibitors. These accomplishments underscore BeiGene's dedication to pioneering oncology solutions and expanding treatment options for cancer patients worldwide.
3. Revvity
Headquartered in Waltham, Massachusetts, Revvity provides health science solutions, technologies, expertise, and services that deliver complete workflows from discovery to development, and diagnosis to cure. With specialized focus areas in translational multi-omics technologies, biomarker identification, imaging, prediction, screening, detection, and diagnosis, Revvity is at the forefront of revolutionizing healthcare. Generating over $3 billion in revenue and employing over 11,000 professionals, Revvity serves customers across pharmaceutical and biotech, diagnostic labs, academia, and governments globally. It is part of the S&P 500 index, with customers in more than 190 countries.
Recently, Revvity collaborated with Element Biosciences to enhance NGS research workflow and launched the EONIS Q system for simplified newborn screening. Revvity has screened 40 million babies annually for life-threatening diseases across 110 countries. Additionally, Revvity partnered with SCIEX to develop neonatal mass spectrometry solutions. Among the company’s other accomplishments, they’ve introduced over 1,500 new antibodies, kits, and reagents annually for life science research, have supplied software solutions to more than two million scientists, and have 700 peer-reviewed publications that describe their TB test as demonstrating excellent performance.
4. Ginkgo Bioworks
Ginkgo Bioworks was founded in 2008 by five MIT scientists, headed by Jason Kelly. Headquartered in Boston, the company programs cells to make everything from food to therapeutics. It has established itself as a leading horizontal platform for cell programming, offering comprehensive solutions across various sectors, from agriculture to pharmaceuticals and beyond. Their biosecurity and public health unit, Concentric by Ginkgo, is dedicated to fortifying global defenses against biological threats, empowering governments and public health leaders worldwide. Ginkgo harnesses biology to shape the future, collaborating with partners to innovate products across industries. They facilitate the creation of sustainable vaccines, agricultural solutions, and alternative proteins, among other developments. Embracing diversity, equity, and inclusion as integral values, Ginkgo fosters an inclusive culture to drive innovation and ensure widespread access to the benefits of its platform.
In recent collaborations, Ginkgo Bioworks has joined forces with OneOne Biosciences to advance nitrogen fixation microbial products and partnered with SaponiQx to develop next-generation vaccine adjuvants. These initiatives underscore Ginkgo's commitment to driving scientific progress and addressing global challenges through biotechnology.
5. PacBio
Pacific Biosciences is a leading life science technology company headquartered in Menlo Park, CA. They specialize in designing, developing, and manufacturing advanced sequencing solutions that empower scientists and clinical researchers to enhance their comprehension of the genome and tackle genetically intricate issues. Their innovative products and technologies, including the HiFi long-read sequencing technology and the short-read Sequencing by Binding (SBBTM) technology, prioritize accuracy, quality, and completeness. With a diverse portfolio catering to various fields such as human genetics, plant and animal sciences, infectious diseases, oncology, and more, Pacific Biosciences addresses a wide range of applications.
Recently, they unveiled the Nanobind PanDNA Kit, further optimizing DNA extraction solutions for HiFi sequencing. Additionally, in collaboration with GeneDx and the University of Washington, they launched a research initiative focused on leveraging long-read whole genome sequencing to enhance diagnostic yield in neonatal care. Through continuous innovation and strategic partnerships, Pacific Biosciences remains at the forefront of revolutionizing genomic analysis. The company’s 2023 financial results showed a total revenue of $200 million, a 56% increase compared to a total revenue of $128 million in 2022.
6. CareDx
Based in Brisbane, California, CareDx, Inc. is focused on developing and commercializing high-value healthcare options for transplant patients and caregivers, to improve transplant patient outcomes through innovative and intelligent solutions. For over 20 years, the company has provided testing services, products, and digital healthcare solutions throughout the transplant patient journey, establishing itself as a leading provider of genomics-based information for transplant patients and guided by its five core values of innovation, patient-centricity, accountability, continuous improvement, and collaboration.
For heart transplant patients, CareDx launched its first commercial product, AlloMap® Heart, in 2005 and then its first multimodal solution HeartCare in 2020, which includes both AlloMap Heart and AlloSure Heart. In 2017 CareDx introduced AlloSure Kidney and in 2021 AlloSure Lung. Both AlloMap gene expression profiling and AlloSure donor-derived cell-free DNA are among the most widely used non-invasive tests of their kind to identify immune quiescence and the risk of graft injury in transplant recipients. Recently, CareDx acquired MediGO, an Organ Transplant Supply Chain and Logistics Company, expanding its presence in the organ procurement transplant landscape. Additionally, CareDx has partnered with the National Foundation for Transplants to improve post-transplant medication adherence. These strategic moves underline CareDx's commitment to advancing patient care and driving innovation in the field of transplant medicine.
7. Sage Therapeutics
Sage Therapeutics, headquartered in Cambridge, Massachusetts, is a pioneering biopharmaceutical company dedicated to revolutionizing brain health treatments. Since its founding in 2010, Sage has remained steadfast in its mission to develop life-changing medicines for individuals to thrive, with a focus on addressing unmet needs of brain health disorders, including tremors and cognitive impairment associated with specific neurodegenerative diseases. Sage is focused on pursuing new pathways when it comes to brain health while changing the way disorders are addressed. Sage Therapeutics focuses its research and development efforts on GABA and NMDA receptors, two critical neurotransmitter systems. Dysfunction in these systems is at the core of a multitude of neurological disorders.
Notably, the company has achieved significant milestones with the development of the only two FDA-approved treatments for postpartum depression. The Zulresso (brexanolone) injection, administered intravenously, and Zurzuvae (quinolone), the first oral medication for PPD, have been pivotal in addressing this critical unmet need in adult women. The company has several significant partnerships, including collaborations with Shionogi and Biogen.
8. Adaptive Biotechnologies
Adaptive Biotechnologies, founded in 2009 by brothers Harlan and Chad Robins, is a Seattle-based biotechnology company that has emerged as a pioneering force in immunosequencing technology. Their breakthrough in understanding the adaptive immune system opened new avenues in medicine. The company's mission revolves around harnessing the potential of the immune system to enhance human health.
With a decade of innovation, Adaptive has developed a proprietary immune medicine platform that enables the comprehensive analysis of the adaptive immune system with precision and scalability. Their immune profiling technology facilitates the translation of insights gleaned from the immune system into actionable solutions. In 2018, the first immunosequencing-based diagnostic developed by the company received FDA clearance.
Recent collaborations, such as the FDA acceptance of Genentech's Investigational New Drug Application and a translational partnership with Takeda, underscore Adaptive's commitment to advancing the field of immune-driven medicine. As they continue to pioneer advancements, Adaptive Biotechnologies remains dedicated to improving lives through the power of the adaptive immune system.
9. BillionToOne
BillionToOne, headquartered in Menlo Park, California, and co-founded by Oguzhan Atay, Ph.D., and David Tsao, Ph.D. is a next-generation molecular diagnostics company with a mission to make molecular diagnostics more accurate, efficient, and accessible for all. The company developed a DNA molecular counter that increases the cell-free DNA diagnostics resolution by over 1,000 fold, unlocking unprecedented improvements in liquid biopsy for cancers, and prenatal screenings. Their proprietary technology, Quantitative Counting Templates, QCTs, enables counting DNA molecules at the single-count level with single base-pair precision, improving disease detection.
Unity Screen, BilliontoOne’s commercially available non-invasive prenatal test is the only test that can determine fetal risk for recessive conditions and was made possible by the company’s molecular counting technology. Recent milestones include the publication of single-gene NIPT sickle cell disease data in the American Journal of Hematology, and the publication of the first clinical outcome study in Genetics in Medicine, showing high accuracy of single-gene NIPT. In March 2022, the company closed a $125 million Series C funding and announced an additional $48.5 million capital raise in December of 2022.
10. Bluebird Bio
Founded in 2010 and headquartered in Somerville, Massachusetts, Bluebird has been setting the standard for gene therapy for more than a decade, first as a scientific pioneer and now as a commercial leader. Bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, the company is proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers. It custom-designs each investigational therapy to address the underlying causes of the disease at the genetic level. Over 375 people work at the company, with more than 170 patients having received therapies across 8 clinical trials.
With a dedicated focus on severe genetic diseases, Bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy. The company custom designs each therapy to address the underlying cause of disease and has developed in-depth and effective analytical methods to drive the field of gene therapy forward.
11. Rocket Pharmaceuticals
Rocket Pharmaceuticals is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of rare and life-threatening cardiac and cardiac and hematologic diseases. The company’s strategic approach to gene therapy has positioned it to launch a sustainable, best-in-class multi-platform pipeline of treatments that directly target the genetic mutation in the affected cells for rare, life-threatening disorders, including Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Danon Disease, Pyruvate Kinase Deficiency, PKP2 arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy. Each program is intended to be transformative, enabling not only reversal of the disorder at molecular and cellular levels, but also providing sustained relief from potentially life-threatening symptoms.
Rocket’s R&D group in the Cranbury, NJ facility features 20,000 square feet of state-of-the-art labs, and the company also has a facility for its manufacturing needs. In December 2022, Rocket Pharmaceuticals acquired Renovacor, a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases.
12. Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. is a global mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine (Kostaive®) in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics.
Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase deficiency and cystic fibrosis, along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications in the U.S., Europe, Japan, China, and other countries).
13. Recursion
Recursion Pharmaceuticals was founded in 2013 by Blake Borgeson, Christopher Gibson, and Dean Li, and is headquartered in Salt Lake City. They are a clinical-stage techbio company, deciphering biology to revolutionize drug discovery. At the company’s core is the Recursion OS, a platform integrating various technologies to cultivate one of the largest proprietary biological and chemical datasets globally. Employing advanced machine-learning algorithms, Recursion extracts trillions of searchable relationships from its dataset, devoid of human bias. With an unparalleled experimental scale, conducting millions of wet lab experiments weekly, and computational prowess, boasting one of the world's most powerful supercomputers, Recursion merges technology, biology, and chemistry to propel medicine forward.
Recent milestones include collaborations with Tempus to leverage data synergies, and a partnership with NVIDIA to propel into the top 50 supercomputing ranks. Additionally, Recursion redefines its collaboration with Bayer toward precision oncology. Furthering its commitment to innovation, Recursion unveiled Valence Labs, a hub for open science and academic research in machine learning. With a pledge of $1 million in academic scholarships, Valence Labs aims to nurture the next generation of machine learning scholars, underscoring Recursion's dedication to advancing scientific exploration.
14. Scientist.com
Scientist.com is a network of public and private e-commerce marketplaces that connect buyers and sellers of scientific research services. The company’s founders, Kevin Lustig, Chris Petersen, and Andrew Martin foresaw that future drug research endeavors would rely heavily on external laboratories, ultimately founding Scientist.com as the first public research marketplace. Scientist.com facilitates the connection between researchers and service providers, offering a centralized platform where researchers can explore different services, request quotes, and initiate collaborations to leverage the expertise of external service providers.
In 2010, Scientist.com worked with a pharmaceutical company wanting to use the platform to give its researchers private access to both internal and external research capabilities, resulting in the launch of a new enterprise software product that helped pharmaceutical researchers more effectively interact with internal core labs and external research suppliers. Between 2011 and 2014, Scientist.com built the world’s most extensive database of global supplier capabilities, ultimately pivoting back to its original model in 2015 and creating a digital marketplace to improve drug research efficiency. Collaborating closely with prominent pharmaceutical firms, Scientist.com pioneered a distinctive industry-wide legal framework to support the externalization of scientific research. Presently, Scientist.com partners with major global pharmaceutical corporations and has achieved impressive growth, reporting 31 consecutive quarters of increased sales and accumulating over $1 billion in sales. Recently, the Scientist.com marketplace expanded to include a broad selection of ready-to-use life science reagents and lab supplies. Between 2021-2022, Scientist.com acquired HealthEconomics.com, InsideScientific, Notch8, and BioPharmCatalyst.
15. Iovance Biotherapeutics
Iovance Biotherapeutics is a biopharmaceutical startup, headquartered in San Carlos, CA. Iovance aims to lead in innovating, developing, and delivering tumor-infiltrating lymphocyte (TIL) therapy for cancer patients globally. They pioneer a transformative approach to cancer treatment, harnessing the natural human immune system to recognize and attack diverse cancer cells with tumor-infiltrating lymphocytes (TIL), the naturally occurring immune cells that fight cancer. Their TIL platform demonstrates promising clinical data across solid tumors, with a commitment to advancing cell therapy, including gene-edited options.
The company’s investigational TIL therapy is manufactured in their state-of-the-art, 136,000-square-foot Cell Therapy Center in Philadelphia, PA. Recently, Iovance Biotherapeutics, Inc. announced the pricing of a $211 million underwritten offering of common stock. Furthermore, their AMTAGVI™ received accelerated approval from the U.S. FDA for advanced melanoma, and is the first FDA-approved T-Cell therapy for solid tumor cancer, and the first treatment option for advanced melanoma after anti-PD-1 and targeted therapy.
16. Freenome
Freenome, a leading company in early cancer detection, is transforming cancer diagnosis and treatment. Freenome adopts a multimodal approach combining molecular biology, computational biology, machine learning, and diverse data types to detect cancer at its earliest stages. Securing over $1.1 billion in funding from investors like Perceptive Advisors, RA Capital Management, and Roche Venture Fund, Freenome aims to make cancer screenings accessible to everyone. With a standard blood draw, Freenome empowers individuals to undergo recommended cancer screenings conveniently.
The company’s first blood test is for early detection of colorectal cancer since detecting and treating this cancer early before it spreads boasts a survival rate of over 90 percent. The company is currently conducting the largest clinical study validating a blood-based colorectal screening test, enrolling more than 40,000 participants. Partnering with healthcare organizations and population health decision-makers, Freenome integrates its technology and software platform to streamline cancer detection processes. Recent partnerships with Walgreens and acquisitions like Oncimmune Ltd demonstrate Freenome's commitment to advancing cancer research and improving early detection methods.
17. Elegen
Elegen is a pioneering biotech company with an innovative cell-free technology to manufacture made-to-order DNA, delivering an industry-leading combination of sequence length, complexity, accuracy, and speed. The ability to receive custom DNA in 7 days without compromising any of these factors enables companies to shave months off the development of mRNA vaccines, cell and gene therapies, and individualized medicines.
Elegen commercially launched ENFINIA™ DNA in March 2023. At 7,000 base pairs, ENFINIA DNA is 2x longer than linear DNA provided by any other DNA manufacturer, with an accuracy 20x higher. The company continues to push the boundaries, providing early access partners with DNA up to 20,000 base pairs and a higher sequence complexity that other manufacturers reject. Earlier this year, Elegen and GSK announced a multi-year collaboration and licensing agreement to use the Elegen’s cell-free DNA manufacturing technology in the development of GSK’s medicines and vaccines, including mRNA vaccines.
18. ElevateBio
ElevateBio® is a technology-driven company powering the creation of life-transforming cell and gene therapies. They were founded in 2017 by David Hallal, Mitchell Finer, and Vikas Sinha, raising $150 million in Series A financing in 2019. The company’s integrated model, combining R&D platforms and cGMP manufacturing, unifies groundbreaking technologies forging the missing link between discovery and widespread delivery of cell and gene therapies, enabling an unprecedented breadth of advanced therapies. Their technologies include gene editing, cell therapy, and cell, protein, and vector engineering, helping their partners scale to new heights in cell and gene therapies by providing a full spectrum of viral vector and cell therapy capabilities.
ElevateBio has significant partnerships with Massachusetts General Hospital, Boston Children’s Hospital, California Institute for Regenerative Medicine, Moderna Therapeutics, and Novo Nordisk. Working with leading biopharma and academic partners, ElevateBio’s team of scientists, drug developers, and company builders are transforming the cell and gene therapy industry, changing the future of medicine. In 2021, they acquired Life Edit Therapeutics, Recently, ElevateBio announced a significant milestone, a $401 million Series D financing round. This substantial investment will further accelerate the company's mission to revolutionize medicine, making life-changing therapies more accessible to patients globally.
19. Quanterix
Quanterix, headquartered in Billerica, Massachusetts, is revolutionizing biomarker analysis, driving forward the realm of precision health. Their groundbreaking technology, Simoa, delivers ultra-sensitive detection capabilities, poised to reshape healthcare provision. By empowering researchers to scrutinize biomarkers across the health-disease spectrum, Quanterix aims to usher in earlier disease detection, refined prognoses, and tailored treatments, ultimately enhancing global health and longevity.
Established in 2007, Quanterix's technology spans diverse therapeutic domains, from oncology to neurology, cardiology, inflammation, and infectious diseases, facilitating cutting-edge research applications. Recent milestones underscore Quanterix's impact, with the FDA's accelerated approval of Tofersen, showing the significance of blood neurofilament light chain as a surrogate endpoint in neurology therapeutic trials. Additionally, a new agreement with Janssen Sciences Ireland UC signals Quanterix's dedication to advancing blood-based Alzheimer's disease detection. As Quanterix continues to push the boundaries of biomarker analysis, its contributions promise to shape the future of healthcare.
20. Poseida Therapeutics
Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. The company’s pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors as well as in vivo gene therapy product candidates that address patient populations with highly unmet medical needs.
Poseida’s approach to cell and gene therapies is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™️ Site-Specific Gene Editing System, and nanoparticle and hybrid gene delivery technologies as well as in-house GMP cell therapy manufacturing. They boldly develop new solutions, redefining treatment paradigms and giving new hope to the patients they serve.
The company has formed a global strategic collaboration with Roche to unlock the promise of cell therapies for patients with hematological malignancies. In August 2023, they announced a $50 million strategic investment by Astellas and granted Astellas certain strategic rights, further validating Poseida's suite of genetic engineering technologies and its allogeneic T-cell platform.
21. AmplifyBio
AmplifyBio is a drug development and commercialization partner for advanced therapies, to advance the quality of human health and save lives through next-generation platforms, tools, and services that accelerate the development of therapies and vaccines. Launched out of Battelle in 2021, they provide industry-leading preclinical toxicology, safety, and pharmacology testing in an agile environment with analytics capabilities that serve the dynamic safety requirements of modern modalities. AmplifyBio further expanded in 2022 to offer R&D services with a particular focus on drug characterization services that de-risk scale-up and manufacturing.
In 2023, they opened their first fully customizable manufacturing suites for lease in staffing models that range from full "hoteling" to more traditional outsourcing partnerships. In any service program, AmplifyBio clients harness decades of experience in drug development, safety testing, and manufacturing support services to reduce risks, timelines, and costs of bringing life-saving cures to patients. They also have the Amplify Safety, Efficacy, and Toxicology headquarters in West Jefferson, Ohio, the location of all preclinical CRO safety studies, and the Amplify Discovery, Optimization, and Characterization R&D services headquartered in San Francisco.
Recently, AmplifyBio chose Apprentice.io’s cutting-edge Tempo Manufacturing Execution System (MES) to expand production capabilities and meet the increasing demand for modern modality commercialization on shorter de-risked timelines.
22. Gritstone Bio
Headquartered in Emeryville, California, with key operations in Cambridge, Massachusetts, and Pleasanton, California, Gritstone Bio is a clinical-stage biotech company, founded in August 2015. The company is developing next-generation vaccines for cancer and infectious diseases, committed to advancing medical science and leading the way in innovative immunotherapy, shaping the future of healthcare. Leveraging insights into the immune system's ability to recognize and destroy diseased cells, the company targets specific antigens to generate a therapeutic immune response.
Gritstone EDGE, the company’s proprietary artificial intelligence-based platform, aids in their understanding of antigens and neoantigens, and specifically which ones will be transcribed, translated, processed, and presented on a cell surface by human leukocyte antigen (HLA) molecules, therefore being visible to T cells. Initially focused on tumor-specific neoantigens, they have several programs, including their first Oncology program, GRANITE, and their second Oncology program, SLATE. They’ve since expanded to include viral antigens on infected cell surfaces. This approach taps into the common biology of anti-tumor and anti-viral immunity, extending the benefits of immunotherapy to more patients.
23. Andelyn Biosciences
Andelyn Biosciences is a cell and gene therapy development and contract manufacturing organization born out of Nationwide Children’s Hospital, where the first FDA-approved systemic gene therapy was discovered. They bring over 20 years of expertise in manufacturing viral vectors. With a track record of supporting over 75 worldwide clinical trials, over 160 employees, more than 16 customizable production suites, and 450 cGMP clinical batches, Andelyn is a trusted partner for companies developing gene therapies for both rare and prevalent diseases.
Offering plasmid manufacturing, process development, and GMP manufacturing capacity up to 2,000L for adherent and suspension processes, Andelyn accelerates the journey from development to commercialization. Their advanced digital model, robust quality system, and full regulatory support ensure efficient and compliant manufacturing processes.
The recent partnership with Armatus Bio to produce suspension-based AAV gene therapy for Charcot-Marie-Tooth Type 1A underscores Andelyn's commitment to advancing therapies for neurological diseases. Additionally, their selection by Ultragenyx for late-stage Process Performance Qualification manufacturing of a novel gene therapy for Sanfilippo Syndrome highlights their role in supporting cutting-edge treatments.
24. Vizgen
Vizgen is a biotechnology company founded in 2019 and headquartered in Cambridge, MA, dedicated to pioneering the next generation of genomics, providing tools that demonstrate the possibilities of in situ single-cell spatial genomics. The company has established the benchmark in spatial genomics and continues to drive industry innovation, delivering the highest sensitivity as well as excellent specificity and accuracy in cell segmentation. The company’s tools enable researchers to gain new insight into the biological systems that govern human health and disease with spatial context.
Vizgen’s MERSCOPE® Platform enables massively multiplexed, genome-scale nucleic acid imaging with high accuracy and unrivaled detection efficiency at subcellular resolution. MERSCOPE® provides transformative insight into a wide range of tissue-scale basic research and translational medicine in oncology, immunology, neuroscience, infectious disease, developmental biology, cell and gene therapy, and is an essential tool for accelerating drug discovery and development.
Vitzgen recently announced its partnership with Scale Biosciences to combine their single-cell and spatial technologies for enhanced data analysis.
25. Precigen
Precigen, headquartered in Germantown, MD with subsidiary locations in Iowa and Belgium, is a leading biopharmaceutical company dedicated to pioneering gene and cell therapies with precision technology, targeting critical diseases across immuno-oncology, autoimmune disorders, and infectious diseases. Their innovative approach focuses on developing affordable biotherapeutics through controlled methods, driving advancements in preclinical and clinical pipelines. Precigen ActoBio™, one of Precigen’s two subsidiaries, is pioneering a new class of therapeutic agents that locally deliver potential disease-modifying therapeutics at disease sites including the intestine, mouth, and nasopharynx. The second subsidiary, Precigen Exemplar, founded in 2008, enables discovery by providing the vehicle to break new ground on the medical research frontier, using specialized miniature swine models that are genetically engineered to exhibit a wide variety of human disease states and to provide a more accurate platform to test the efficacy of new medications and devices.
The company's recent achievements include FDA confirmation of the ongoing Phase 1/2 study of PRGN-2012 AdenoVerse Immunotherapy as pivotal for accelerated approval. Additionally, Precigen announced promising Phase 1 data for PRGN-3005 Autologous UltraCAR-T® Cells, demonstrating potential benefits for advanced-stage platinum-resistant ovarian cancer patients. As an innovation engine, Precigen continues to progress unique therapies towards clinical proof-of-concept and commercialization, exemplifying its commitment to addressing urgent medical needs with cutting-edge solutions.
