Next-Gen Biopharma: The Startups Leading the Charge

From drug discovery and development through manufacturing, analytics, and patient diagnosis and treatment administration, small and emerging biotechs are dramatically influencing the shape of the biopharmaceutical industry and enabling new, important medicines to reach the market. Whether they are finding new strategies for leveraging more traditional modalities, creating novel drug classes, or introducing enabling technologies, the continued success of these organizations — both those that exist today and those that will be born tomorrow — is crucial to the continued success of the pharmaceutical sector.

Big Biotech is Big, but Emerging Biotechs Drive Innovation

The biggest biotech companies by market capitalization at the end of 2024 included Amgen, Gilead Sciences, Samsung Biologics, and Celltrion, among others.¹ Despite their size, these companies do not contribute to innovation in the biopharma industry as much as the hundreds of small and emerging biotechs, many of which don’t even have marketed therapies.² In 2022, emerging biotechs accounted for 65% of pipeline candidates with no large biopharma company involvement. In addition, the number of drug products approved by the U.S. Food and Drug Administration (FDA) in 2022 represented slightly more than 40% of all approved products. Furthermore, in August 2025, the NASDAQ Biotechnology Index (INDEXNASDAQ:NBI) was trading at a three-year high due to the continued innovation within small and emerging biotechs.³

What Makes an Emerging Biotech Promising?

There are endless lists of promising biotech companies available for review. Each one considers widely varying sets of factors when making their choices. Top of the lists are usually financing (type, quantity, and timing), technology and its level of novelty, pipeline and clinical data, marketed products, and collaborations. Some lists consider geographic reach, others the modality and/or targeted indication(s). Some only focus on companies developing therapeutics, while others include technology companies enabling advanced manufacturing or analytics.

Below are some small and emerging biotechs chosen from various lists of “companies to watch” published in the last year.^3–10

Potentially Exciting Gene Therapy Companies

Gene therapy remains a nascent sector of the biopharma industry. It is possible, therefore, to consider most small biotechs developing gene therapies as emerging companies. If this approach is taken, several companies appear repeatedly on lists of top gene therapy companies: Krystal Biotech, CRISPR Therapeutics, Beam Therapeutics, Intellia Therapeutics, and Verve Therapeutics are prime examples.¹¹

CRISPR, Beam, Intellia, and Verve are all developing gene-editing therapies. Others are setting themselves apart by developing proprietary technologies. Aro Biotherapeutics is focused on its novel, targeted delivery platform, while Neurogene has developed a proprietary gene regulation technology. nChromBio is combining epigenetic editing and next-generation in vivo delivery technologies to develop treatments for chronic hepatitis. Rampart Bioscience is hoping its proprietary nonviral gene therapy platform will enhance safety and enable redosing of its gene therapies.

Some companies are differentiating themselves by addressing specific indications. Castle Creek Biosciences is focused on rare dermatologic and metabolic diseases, while Life Biosciences is advancing an innovative cellular rejuvenation platform to reverse diseases of aging and injury, and Latus Bio is leveraging novel AAV capsids to treat central nervous system disorders.

A Few Promising Cell Therapy Companies

Narrowing down the top cell therapy companies is challenging given the numerous types of cell therapies under development. They can be divided, for instance, into organizations focusing on autologous versus allogeneic therapies or on firms developing stem-cell therapies versus adoptive cell therapies. Within those categories, there are companies focused on induced pluripotent stem cells (iPSCs) and mesenchymal stem cells (MECs) and various types of immune cells (e.g., T, natural killer), and so on.

Some trends can be observed when looking at the activities of emerging biotechs involved in the development of cell therapies. The majority are targeting chronic diseases, such as cancer, autoimmune diseases, and neurodegenerative disorders; they often are leveraging novel technologies; and they typically form partnerships to facilitate commercialization.12

Interesting emerging biotechs focused on iPSC-based therapies include Lineage Cell Therapeutics with its OpRegen^® technology for treatment of dry age-related macular degeneration (AMD); Aspen Neuroscience with its focus on patient-specific treatments for Parkinson’s disease and its use of AI-driven genomic analysis; Kenai Therapeutics. previously known as Ryne Bio, with its off-the-shelf dopamine neuron replacement cell therapies for treatment of neurodegenerative diseases; GC Therapeutics with its focus on off-the-shelf therapies developed using synthetic biology, gene-editing, cell engineering, and machine learning; Garuda Therapeutics with its allogenic stem cells that do not require any donor matching; and Sana Biotechnology with its use of advanced delivery systems for allogeneic therapies. Fate Therapeutics and Century Therapeutics, meanwhile, are developing iPSC-derived CAR-T and NK cell therapies.

Several companies are leveraging other types of cells. Orchard Therapeutics is developing hematopoietic stem cells for delivery of gene therapies to treat rare genetic disorders. Vericel Corporation’s MACI comprises expanded, patient-derived chondrocytes on a porcine collagen membrane for treatment of damaged cartilage. Rubius Therapeutics’ proprietary Red Cell Therapeutics™ platform consists of engineered red blood cells that deliver therapeutic payloads for treatment of a variety of diseases. The encapsulated PEC-Encap cell therapy from ViaCyte uses pluripotent stem cell-derived pancreatic cells to restore insulin production as a treatment for type 1 diabetes. The MultiStem^® allogeneic stem cell therapy from Athersys is derived from adult bone marrow or other non-embryonic tissues and is designed to modulate immune responses, reduce inflammation, and promote tissue repair.

Companies focused on CAR therapies include:

Allogene Therapeutics: allogenic CAR-T cell therapies.
Anixa Biosciences: autologous chimeric endocrine receptor T cell therapies for treatment of ovarian cancer.
Autolus Therapeutics: cell therapies leveraging proprietary target binders that minimize excessive activation of the programmed T cells. Its technology has been licensed by BioNTech.
Cartesion Therapeutics: (DESCARTES-08 therapy comprising killer T cells modified using mRNA and designed to attack pathogenic plasma cells that secrete auto-antibodies leading to autoimmune disease.
CARGO Therapeutics: lead candidate induces costimulatory signaling and is designed for patients who have become resistant to CD19-targeting CAR-T therapy

Companies focused on allogeneic cell therapies of one type or another also include A2 Biotherapeutics, Adicet Bio, Caribou Biosciences, Coeptis Therapeutics, Cytovia Therapeutics, IN8Bio, Poseida Therapeutics, Shinobi Therapeutics, TC BioPharm, and Tr1X.¹³

A few emerging biotechs are developing enabling technologies rather than therapeutics. Examples include Cellino, which offers an iPSC manufacturing platform leveraging robotics, machine learning, and laser-guided automation. Rooster Bio, meanwhile, offers a large-scale manufacturing platform technology for MSCs. StemoniX and Curi Bio offer solutions to facilitate cell therapy drug discovery. The microHeart® and microBrain^® technologies from StemoniX replicate human heart and brain cells, respectively, for drug toxicity and efficacy screening. Curi Bio’s Mantarray platform mimics human cardiac and skeletal muscle tissue. Exodigm Biosciences’ sensors enable sampling of RNA and other genomic material from live cells for sorting and other functional downstream applications. MagGenix’ magnetogenetic cell therapy platform that enables the magnetic activation of engineered cells within clinical, industrial, and research settings to support highly targeted delivery.

Interesting RNA Startups

With the success of the mRNA COVID-19 vaccines, interest in mRNA therapeutics has blossomed, and countless biotechs have been formed to targeted a wide array of diseases. Several companies are also exploring other RNA-based therapies.

Leading the mRNA pack are Moderna Therapeutics and BioNTech, the developers of the approved COVID-19 vaccines, CureVac (also active during the pandemic), and Translate Bio (now part of Sanofi).¹⁴ Other companies making progress in the field of mRNA therapeutics and vaccines (e.g. for cancer and infectious diseases) include Abogen Biosciences, Arctutus Therapeutics, Averna Therapeutics (previously Exsilio Therapeutics), Cartesion Therapeutics, Etherna, Nutcracker Therapeutics, Orbital Therapeutics (also circular RNA), and Radar Therapeutics.

Companies developing anti-sense oligonucleotide (ASO) and RNA interference (RNAi) therapeutics include Atalanta Therapeutics, Dicerna Pharmaceuticals, Sirnaomics, Sand toke Therapeutics. Those leveraging RNA-editing technologies include ADARx, Korro Bio, and Wave Life Sciences. Alltrna, meanwhile, is focused on developing transfer RNA (tRNA) therapies, and Orna Therapeutics specializes in fully engineered circular RNA therapeutics. NanoVation Therapeutics™ is an example of a company developing proprietary lipid nanoparticle technology for the delivery of RNA (and DNA) therapeutics and vaccines.

Highlighting Select Next-Gen Antibody Developers

Despite the hype surrounding cell and gene therapies, many emerging biotechs continue to focus on antibody drug development. Candid Therapeutics, Clasp Therapeutics, and EvolveImmune Therapies are developing T cell engagers immunotherapies comprising multi-functional antibodies that combine cancer cell targeting with the stimulation of T cells to enhance the body’s immune response.

Other types of bispecific candidates are progressing in the clinic as well. Larimar Therapeutics is developing a recombinant fusion protein designed to deliver a deficient protein to patient cells suffering from Friedreich’s Ataxia. Immunocore is using its ImmTAC^® technology to develop T cell receptor (TCR) bispecific immunotherapies including the first approved TCR therapeutic (KIMMTRAK^®, tebentafusp) for treatment of metastatic uveal melanoma.

Some emerging biotechs are developing antibodies with novel mechanisms of action. One example is Diagonal Therapeutics, whose agonist antibodies for rare diseases activate a broken signaling cascade in the cells to go after the underlying cause of disease instead of inhibiting specific cellular functions. COUR Pharmaceuticals uses its proprietary encapsulated antigen technology to reprogram the immune system, and is focusing on primary biliary cholangitis. The company has signed deals with both Roche and Takeda.

Skye Bioscience and Marea Therapeutics are taking different approaches to obesity and metabolic disease treatment than the currently popular GLP-1 agonist therapies. Skye’s lead antibody candidate nimacimab inhibits CB1, which is expressed strongly in tissues involved in metabolism and metabolic disorders. Marea uses genetics information to identify unaddressed drivers of cardiometabolic disease risk. Its lead candidate is an antibody that inhibits ANGPTL4 to augment lipoprotein lipase activity.

TG Therapeutics appeared at the top of the Therapeutics Deloitte Technology Fast 500 in November 2024. Its antibody drug BRIUMVI (ublituximab) was approved in 2022 for patients suffering from relapsing forms of multiple sclerosis (MS) and because of its attractive administration protocol and pricing has done very well. Tizania Life Sciences has an antibody drug in development for treatment of MS, amyotrophic lateral sclerosis (ALS), and potentially Alzheimer’s disease (foralumab), leveraging its proprietary intranasal delivery technology. Nabla Bio, meanwhile, is combining generative drug design and massively parallel physical experimentation to design antibodies effective against targets previously thought to be undruggable.

Emerging Biotechs Pursuing Other Modalities

Antibody–Drug Conjugate (ADC) Companies

ADCs offer another approach to realizing more targeted delivery of antibody therapeutics to achieve greater efficacy with fewer side effects. Several emerging biotechs are involved in the field.15 OnCusp Therapeutics has a lead ADC candidate for platinum-resistant ovarian cancer and other solid tumors. ADC Therapeutics’ ADC leverage pyrrolobenzodiazepine (PBD)-based class of payloads. Ascendo is focused on increasing the targeting ability of its ADCs by choosing receptors on cancer cells known to internalize molecules that bind to them. Tubulis Technologies has developed new conjugation approaches that help prevent early payload release and allow a wider choice of drug-to-antibody ratios. Pheon Therapeutics, meanwhile, has focused on developing a novel payload platform. Firefly Bio’s degrader antibody conjugates comprise ADCs with protein degraders to achieve more precise delivery with minimal off-target effects.

Peptide Therapeutic Developers

Peptide therapies have received significant attention recently due to the tremendous success of GLP-1 agonists for weight loss. Emerging biotechs have, not surprisingly, entered this field with the hopes of bringing novel and improved solutions to the market. Metsera, for instance, is developing a long-acting GLP-1 drug that has shown positive results in early clinical trials.

Other biotechs are investigating the potential of peptide therapeutics for treatment of other diseases. One such company is Protagonist Therapeutics, which has several candidates in development for the treatment of hematology and blood disorders involving excessive iron levels and inflammatory and immunomodulatory disorders and diseases such as psoriasis and other skin conditions. Red Queen Therapeutics is another. It has developed a proprietary lipopeptide platform technology for rapidly designing new antivirals and has rapidly been contracted by the Biomedical Advanced Research and Development Authority (BARDA) to develop a pan-influenza treatment.

A Few Novel Technology Developers

Many of the therapeutic candidates being advanced by emerging biotech companies are enabled by novel technologies introduced by other small biotechs looking to improve analytics, manufacturing, and most other aspects of accelerated and innovative drug development, manufacturing, and administration.

Syncelll has developed a technology that enables accelerated spatial proteomics discovery, while 10x Genomics has introduced a more efficient method for single-cell analysis to support accelerated cell-line engineering and selection. TMRW Life Sciences’ automated robotic cryo-storage platform eliminates the potential for sample contamination in biological research and diagnostic applications. Emmulate is focused on organ-on-chip technology for pharmaceutical testing applications during discovery and development (e.g., drug screening, disease modeling).

Companies including DNA Script, Touchlight, 4basebio, Elegen, GenScript, and Gxstrands are producing synthetic single-stranded and double-stranded DNA as key raw materials for genetic therapies. Halozyme Therapeutics’ Enhanze drug delivery technology helps convert intravenous formulations into products that can be delivered subcutaneously. Strata Oncology, Inc. uses genomic profiling to sequence tumors and match patients with the most suitable clinical trials involving personalized therapies. PathAI, meanwhile, is using its AI-based pathology platform to analyze digital images of tissue samples to accelerate and increase the accuracy of disease diagnosis.

Advancing Future Medicines

From drug discovery and development through manufacturing, analytics, and patient diagnosis and treatment administration, small and emerging biotechs are dramatically influencing the shape of the biopharmaceutical industry and enabling new, important medicines to reach the market. Whether they are finding new strategies for leveraging more traditional modalities, creating novel drug classes, or introducing enabling technologies, the continued success of these organizations, both those that exist today and those that will be born tomorrow, is crucial to the continued success of the pharmaceutical sector.