U.S. Exchanges
list of publicly traded biotech companies
Comprehensive directory of 500+ publicly traded biotech companies on the NASDAQ and NYSE. Track 2026 market cap leaders, clinical pipelines, and AI-driven precision medicine innovators.
This page is for informational and educational purposes only and does
not constitute investment advice. Always consult a qualified financial
professional before making investment decisions.
Navigating a list of publicly traded biotech companies is essential for investors seeking exposure to high-growth medical breakthroughs and genomic revolutions. The sector is currently dominated by a Large-Cap Biotech List of powerhouses like Vertex and Regeneron, while hundreds of smaller firms populate the Mid-Cap Biotech and small-cap tiers. In 2026, the industry is increasingly defined by AI scenario modeling and precision oncology, driving significant volatility and opportunity. This index serves as a central hub to compare these innovators against the Complete Healthcare Companies directory, tracking the $5.6 trillion global market projection for 2035.
Key Takeaways
01
Mega-Cap Consolidation
The Top 25 biotech companies now command a $1.8 trillion market cap, led by cystic fibrosis and oncology specialists. Compare these giants in our Large-Cap Biotech List.
02
AI and Precision Medicine
Over 66% of large sponsors now use AI scenario modeling to accelerate drug discovery, making tech-integrated pipelines a primary 2026 investment catalyst.
03
ETF Performance Divergence
The XBI ETF has outperformed the broader market with a +23% gain in 2026, signaling a recovery in clinical-stage sentiment.
04
Small-Cap Volatility
While nano-caps dominate the company count, they remain high-risk acquisition targets. See more in our Small-Cap Biotech index.
Top list of publicly traded biotech companies by Market Cap (2026)
The following table captures the dominant biotech market leaders and top 2026 performers, highlighting the scale of cystic fibrosis and oncology pipelines.
| Rank | Ticker | Company | Focus | Market Cap | Gross Margin | P/E Ratio | Zen Score |
|---|---|---|---|---|---|---|---|
| 1 | VRTX | Vertex Pharmaceuticals | Cystic Fibrosis | $112.9B | -- | -- | High |
| 2 | REGN | Regeneron Pharma | Oncology/Eye | $79.9B | -- | -- | High |
| 3 | BNTX | BioNTech SE | Immunotherapy | $24.7B | 77.82% | -- | Med |
| 4 | AXSM | Axsome Therapeutics | CNS/Neuro | $9.4B | 91.16% | -- | High |
| 5 | BCRX | BioCryst Pharma | Rare Disease | $2.4B | -- | -- | High |
| 6 | XERS | Xeris Biopharma | Endocrinology | $1.1B | -- | -- | High |
| 7 | TXG | 10x Genomics | Life Sciences | -- | -- | -- | Med |
| 8 | FDMT | 4D Molecular | Gene Therapy | -- | -- | -- | Med |
| 9 | VEEV | Veeva Systems | Cloud Health | -- | -- | -- | High |
| 10 | ZTS | Zoetis | Animal Health | -- | -- | -- | High |
Market data is approximate and for informational purposes only. Data reflects early Q2 2026 figures. Not a recommendation to buy or sell.
list of publicly traded biotech companies — Complete Company List
Biotech Stocks
A list of publicly traded biotech companies can be found by scrolling down or you can access a list of the companies in each group through the industry links on this page.
Large, Mid, Small, Micro and Nano-Cap Biotech Sections
- List of Large-Cap Biotech
- List of Mid-Cap Biotech
- List of Small-Cap Biotech
- List of Micro-Cap Biotech
Specific/Classes of Diseases
Areas of the Body
- Blood
- Central Nervous System
- Dental
- Ears
- ENT
- Eye Care
- Gastrointestinal
- Heart
- Kidney
- Liver
- Respiratory Health
- Skin Care
- Spine
- Urology
- Vascular
Specific Demographics
Industry Links: Biotech
Healthcare Areas of Focus
- Aesthetics
- Animal Health
- Blood
- Cancer
- Central Nervous System
- Dental
- Diabetes
- Ears and Hearing Loss
- Ear Nose and Throat
- Eye Care
- Gastrointestinal
- Heart
- Infectious Diseases
- Kidney
- Liver
- Multiple Sclerosis
- Neurological
- Pain Management
- Prenatal and Children
- Orthopedics
- Rare Diseases
- Respiratory Health
- Skin Care
- Sleep
- Spine
- Urology
- Vascular
- Weight Loss
- Women’s Health
- Wound Care
- AbbVie Inc. (ABBV) (Biopharmaceutical company: immunology, kidney disease, liver disease, neuroscience, oncology, women’s health)
- Abeona Therapeutics (ABEO) (Gene therapy and plasma-based products)
- AC Immune SA (ACIU) (IPO September 23: biopharmaceutical company focused on diseases that are associated with protein misfolding)
- ACADIA Pharmaceuticals Inc. (ACAD) (Biopharmaceutical company: central nervous system disorders)
- Acasti Pharma, Inc. (ACST) (Canada, biopharmaceutical company: active pharmaceutical ingredients (API)
- Acceleron Pharma Inc. (XLRN) (Cancer and orphan diseases)
- Achaogen, Inc. (AKAO) (Biopharmaceutical company: antibacterials to treat multi-drug resistant gram-negative infections)
- Achillion Pharmaceuticals, Inc. (ACHN) (Complement-related diseases and infectious diseases)
- Acorda Therapeutics, Inc. (ACOR) (Biopharmaceutical company: neurological disorders)
- Actinium Pharmaceuticals, Inc. (ATNM) (Biopharmaceutical company: immunotherapeutics for the treatment of advanced cancers)
- Adaptimmune Therapeutics plc (ADAP) (IPO in May 2015, United Kingdom, biopharmaceutical company: cancer immunotherapy products)
- ADMA Biologics Inc. (ADMA) (Biopharmaceutical company: Plasma-based biologics for the treatment and prevention of certain infectious diseases)
- Aduro BioTech, Inc. (ADRO) (IPO in April 2015: Cancer immunotherapy; pancreatic cancer)
- Advaxis, Inc. (ADXS) (Clinical-stage biotechnology company: immunotherapies to treat cancer)
- Adverum Biotechnologies, Inc. (ADVM) (Biopharmaceutical company: serious eye diseases)
- Aeglea BioTherapeutics, Inc. (AGLE) (IPO April 7, 2016: Biotech company focused therapeutics for the treatment of inborn errors of metabolism (IEM)
- AEterna Zentaris Inc. (AEZS) (Canada, biopharmaceutical company: oncology, endocrinology and women’s health)
- Affimed N.V. (AFMD) (Germany, biopharmaceutical company: cancer immunotherapies)
- Agenus Inc. (AGEN) (Immunology: cancer, infectious diseases)
- Agios Pharmaceuticals, Inc. (AGIO) (Biopharmaceutical company: cancer and rare genetic disorders of metabolism)
- Aileron Therapeutics, Inc. (ALRN) (IPO June 29, 2017; biopharmaceutical company: cancer)
- Aimmune Therapeutics, Inc. (AIMT) (IPO in August 2015: Biopharmaceutical company: allergies)
- Akari Therapeutics, Plc (AKTX) (United Kingdom, biopharmaceutical company: therapeutics to treat orphan autoimmune and inflammatory diseases)
- Akcea Therapeutics, Inc. (AKCA) (IPO July 14, 2017; biopharmaceutical company: cardiometabolic lipid disorders)
- Akebia Therapeutics, Inc. (AKBA) (Biopharmaceutical company: anemia secondary to chronic kidney disease)
- Alder BioPharmaceuticals, Inc. (ALDR) (Biopharmaceutical company: migraine, autoimmune diseases and inflammatory diseases)
- Aldeyra Therapeutics, Inc. (ALDX) (Treatments for diseases thought to be related to endogenous free aldehydes)
- Alexion Pharmaceuticals, Inc. (ALXN) (Biopharmaceutical company: nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, hypophosphatasia, Lysosomal Acid Lipase Deficiency)
- Alkermes Plc. (ALKS) (Ireland, biopharmaceutical company: medicines for the treatment of central nervous system diseases)
- Allena Pharmaceuticals, Inc. (ALNA) (IPO November 2, 2017; biopharmaceutical company: metabolic and kidney disorders)
- Alnylam Pharmaceuticals, Inc. (ALNY) (Biopharmaceutical company: RNAi therapeutics as genetic medicines; serious, life-threatening diseases)
- Altimmune, Inc. (ALT) (Immunotherapeutics and vaccines)
- Amarin Corporation Plc. (AMRN) (Ireland, biopharmaceutical company: cardiovascular health)
- Amgen Inc. (AMGN) (Therapeutics: cardiovascular, inflammation, oncology, biosimilars)
- Amicus Therapeutics, Inc. (FOLD) (Biopharmaceutical company: lysosomal storage diseases)
- Ampio Pharmaceuticals, Inc. (AMPE) (Biopharmaceutical company: inflammatory conditions)
- AmpliPhi Biosciences Corporation (APHB) (Biopharmaceutical company: bacterial infections)
- AnaptysBio, Inc. (ANAB) (IPO January 26, 2017; biotech company; inflammation)
- Anavex Life Sciences Corp. (AVXL) (Biopharmaceutical company: Alzheimer’s, other CNS diseases; cancer)
- ANI Pharmaceuticals, Inc. (ANIP) (Branded and generic prescription pharmaceuticals)
- Anika Therapeutics Inc. (ANIK) (Therapeutic products for tissue protection, healing and repair; animal health, ophthalmic, joint health and orthopedic products, surgical products to treat ENT disorders, wound care products)
- Apellis Pharmaceuticals, Inc. (APLS) (IPO November 9, 2017; biopharmaceutical company: complement-dependent autoimmune and inflammatory diseases)
- Applied Genetic Technologies Corporation (AGTC) (Treatments for rare eye diseases caused by mutated genes)
- Apricus Biosciences, Inc. (APRI) (Biopharmaceutical company: sexual health therapies, urology and rheumatology)
- Aptevo Therapeutics Inc. (APVO) (Biosciences: oncology and hematology therapies)
- Aptose Biosciences, Inc. (APTO) (Canada: therapeutics to treat life-threatening cancers; acute myeloid leukemia, high-risk myelodysplastic syndromes)
- Arbutus Biopharma Corporation (ABUS) (Canada, biopharmaceutical company: chronic hepatitis B infection)
- ARCA Biopharma, Inc. (ABIO) (Biopharmaceutical company: therapies for cardiovascular diseases)
- Arcturus Therapeutics (ARCT) (RNA medicines and technologies)
- Arcus Biosciences, Inc. (RCUS) (IPO March 15, 2018; biopharmaceutical company: cancer immunotherapies)
- Ardelyx, Inc. (ARDX) (Molecule therapeutics: gastrointestinal)
- Arena Pharmaceuticals, Inc. (ARNA) (Biopharmaceutical company: drugs that target G protein-coupled receptors; weight management)
- argenx SE (ARGX) (IPO May 18, 2017; biotech company: autoimmune diseases and cancer)
- Ariad Pharmaceuticals, Inc. (ARIA) (Oncology company: chronic and acute leukemia, lung cancer and other difficult-to-treat cancers)
- ARMO BioSciences, Inc. (ARMO) (IPO January 26, 2018; immuno-oncology company)
- ArQule, Inc. (ARQL) (Small molecule cancer therapeutics)
- Array BioPharma Inc. (ARRY) (Biopharmaceutical company: targeted small molecule drugs used to treat patients afflicted with cancer)
- Arrowhead Research Corporation (ARWR) (Biopharmaceutical company: RNAi therapeutics, chronic hepatitis B infection)
- Arsanis, Inc. (ASNS) (IPO November 16, 2017; biopharmaceutical company: infectious diseases)
- Ascendis Pharma (ASND) (IPO in January 2015: Denmark, biopharmaceutical company: growth hormone deficiency, pulmonary arterial hypertension)
- Assembly Biosciences, Inc. (ASMB) (Biopharmaceutical company: treatments for hepatitis B virus infection and C. difficile-associated diarrhea)
- Asterias Biotherapeutics, Inc. (AST) (Regenerative medicine: therapeutics developed from platforms based on pluripotent stem cells and dendritic cell cancer immunotherapy)
- AstraZeneca Plc. (AZN) (United Kingdom, biopharmaceutical company: autoimmune, cardiovascular, infection, inflammation, metabolic, neuroscience, oncology, respiratory)
- Atara Biotherapeutics, Inc. (ATRA) (Molecularly-targeted product candidates and T-cell product candidates; cancer, kidney disease and other illnesses)
- Athenex, Inc. (ANTX) (IPO June 14, 2017; biopharmaceutical company: cancer)
- Athersys, Inc. (ATHX) (Biopharmaceutical company: most advanced programs in area of regenerative medicine; stem cell products)
- aTyr Pharma Inc. (LIFE) (IPO in May 2015: Medicines used in the treatment of severe rare diseases)
- Audentes Therapeutics, Inc. (BOLD) (IPO July 20, 2016: biotech company focused on therapy treatments for rare diseases)
- Aurinia Pharmaceuticals Inc. (AUPH) (Canada, biopharmaceutical company: lupus nephritis)
- Auris Medical Holding AG (EARS) (Switzerland: inner ear therapies)
- AVEO Pharmaceuticals, Inc. (AVEO) (Biopharmaceutical company: therapies through biomarker-driven insights)
- Axovant Sciences Ltd. (AXON) (Therapeutics designed to treat dementia)
- Axsome Therapeutics, Inc. (AXSM) (IPO in November 2015: Biopharmaceutical company: therapies for pan management and central nervous system disorders)
- AzurRx BioPharma, Inc. (AZRX) (IPO October 11, 2016: Development stage biopharmaceutical company focused on gastrointestinal disorders)
- Beigene, Ltd. (BGNE) (IPO February 3, 2016: Biopharmaceutical company with therapeutics designed to treat cancer)
- Bellerophon Therapeutics, Inc. (BLPH) (Cardiopulmonary and cardiac diseases)
- Bellicum Pharmaceuticals, Inc. (BLCM) (Biopharmaceutical company: cellular immunotherapies for various forms of cancer; hematological and solid tumors, orphan inherited blood disorders)
- Benitec Biopharma LTD (BNTC) (IPO in August 2015: Australia, biopharmaceutical company: gene silencing technology)
- BeyondSpring Inc. (BYSI) (IPO March 9, 2017; cancer therapies)
- Bio Blast Pharma Ltd. (ORPN) (Therapies for patients with rare and ultra-rare genetic diseases)
- BioCryst Pharmaceuticals, Inc. (BCRX) (Infectious and inflammatory diseases)
- Biofrontera AG (BFRA) (IPO February 14, 2018; biopharmaceutical company: dermatology)
- Biogen Inc. (BIIB) (Biopharmaceutical company: therapies for immunology, hematology, multiple sclerosis, neurology)
- Biohaven Pharmaceutical Holding Company Ltd. (BHVN) (IPO May 4, 2017; biopharmaceutical company: neurological diseases)
- BioLife Solutions, Inc. (BLFS) (Hypothermic storage and cryopreservation products; thermal shipping products for cells, tissues, and organs)
- BioLineRx Ltd. (BLRX) (Israel, biopharmaceutical company: Cardiac device, acute myeloid leukemia, celiac disease, infectious diseases, inflammatory bowel disease, skin lesions)
- BioMarin Pharmaceutical Inc. (BMRN) (Therapies for serious and life-threatening rare and ultra-rare genetic diseases)
- BiondVax Pharmaceuticals Ltd. (BVXV) (IPO in May 2015: Israel, biopharmaceutical company: flu vaccine)
- Bio-Path Holdings, Inc. (BPTH) (Therapeutic products utilizing its proprietary liposomal delivery technology; blood cancer, triple negative and inflammatory breast cancers)
- BioSpecifics Technologies Corp (BSTC) (Biopharmaceutical company: injectable collagenase used for treatment of Dupuytren’s contracture and Peyronie’s disease)
- Bio-Techne Corp (TECH) (Bioactive tools and resources used in the research and clinical diagnostic markets)
- BioTime, Inc. (BTX) (Regenerative medicine to patients with serious diseases and degenerative conditions)
- Bioverativ Inc. (BIVV) (Biotech company: hemophilia and other rare blood disorders)
- BioXcel Therapeutics, Inc. (BTAI) (IPO March 8, 2018; biopharmaceutical company: neuroscience and immuno-oncology)
- Bluebird Bio, Inc. (BLUE) (Gene therapy)
- Blueprint Medicines Corporation (BPMC) (IPO in April 2015: Biopharmaceutical company: kinase inhibitors for genomically defined cancers)
- Brainstorm Cell Therapeutics Inc. (BCLI) (Stem cell therapies for neurodegenerative diseases)
- Bristol-Myers Squibb Company (BMY) (Biopharmaceutical company: cancer, cardiovascular disease, hepatitis B and hepatitis C, HIV/AIDS, rheumatoid arthritis)
- Caladrius Biosciences, Inc. (CLBS) (Immunotherapy company: cell process optimization, development, and manufacturing)
- Calithera Biosciences, Inc. (CALA) (Immunotherapy company: cell process optimization, development, and manufacturing)
- Can-Fite Biopharma Ltd (CANF) (Israel: biopharmaceutical company: autoimmune-inflammatory and cancer diseases)
- Capricor Therapeutics, Inc. (CAPR) (Treatment of heart failure and heart attacks)
- Cara Therapeutics, Inc. (CARA) (Chemical entities designed to alleviate pain and pruritus)
- CASI Pharmaceuticals, Inc. (CASI) (Biopharmaceutical company: therapeutics addressing cancer and other unmet medical needs; focus on China)
- Catabasis Pharmaceuticals Inc. (CATB) (Biopharmaceutical company: rare diseases, serious lipid disorders)
- Catalyst Biosciences, Inc. (CBIO) (Biopharmaceutical company: hemostasis)
- Catalyst Pharmaceutical Partners, Inc. (CPRX) (Biopharmaceutical company: rare neuromuscular and neurological diseases)
- Celcuity Inc. (CELC) (IPO September 20, 2017; diagnostic tests for new cancer subtypes)
- Celgene Corporation (CELG) (Biopharmaceutical company: cancer and immune-inflammatory related diseases)
- Cellect Biotechnology Ltd. (APOP) (IPO July 29, 2016: biotech company with functional stem cells selection platform)
- Cellectis SA (CLLS) (IPO in March 2015, France: Gene-editing company; cancer therapies based on engineered T cells)
- Celldex Therapeutics, Inc. (CLDX) (Therapeutic antibodies, antibody drug conjugates, immune system modulators and vaccines; brain cancer, triple negative breast cancer)
- Cellectar Biosciences, Inc. (CLRB) (Agents to detect, treat and monitor a broad spectrum of cancers)
- Cellular Biomedicine Group, Inc. (CBMG) (IPO in November 2014; China: clinical-stage biopharmaceutical firm focused on developing immunotherapies for cancer and stem cell therapies)
- Celsion Corporation (CLSN) (Oncology drug development company)
- Celyad SA (CYAD) (Belgium: Immuno-oncology company: allogeneic CAR-T cell therapy)
- Cerus Corporation (CERS) (Biomedical products company: pathogen reduction systems for blood components)
- ChemoCentryx, Inc. (CCXI) (Biopharmaceutical company: autoimmune diseases, inflammatory disorders, cancer)
- CTI BioPharma Corp. (CTIC) (Oncology company)
- CytomX Therapeutics Inc. (CTMX) (Probody™ therapeutics for the treatment of cancer)
- CytRx Corporation (CYTR) (Biopharmaceutical company: research and development company specializing in oncology)
- Dare Bioscience, Inc. (DARE) (Women’s reproductive health)
- DBV Technologies S.A. (DBVT) (France: therapies for food and pediatric allergy patient)
- Deciphera Pharmaceuticals, Inc. (DCPH) (IPO September 28, 2017; biopharmaceutical company: cancer)
- DelMar Pharmaceuticals, Inc. (DMPI) (Biopharmaceutical company focused on cancer therapies)
- Dermira, Inc. (DERM) (Biopharmaceutical company: products for dermatologists and their patients)
- Denali Therapeutics Inc. (DNLI) (IPO December 8, 2017; biotech company: neurodegenerative diseases)
- Dicerna Pharmaceuticals, Inc. (DRNA) (Biopharmaceutical company: inherited diseases of the liver and genetically defined cancers)
- Durect Corporation (DRRX) (Biopharmaceutical company: pain management, CNS disorders, acute organ injury and metabolic diseases)
- Dynavax Technologies Corporation (DVAX) (Biopharmaceutical company; products to prevent and treat infectious diseases)
- Edge Therapeutics Inc. (EDGE) (IPO in October 2015: Neurological conditions)
- Editas Medicine, Inc. (EDIT) (IPO February 3, 2016: Genome editing company)
- Eiger BioPharmaceuticals, Inc. (EIGR) (Biopharmaceutical company: Hepatitis Delta, Hypoglycemia, Pulmonary Arterial Hypertension, Lymphedema)
- Emergent Biosolutions, Inc. (EBS) (Biopharmaceutical company: therapeutics and vaccines in hematology/oncology; transplantation and infectious disease; biological defense products)
- Enanta Pharmaceuticals, Inc. (ENTA) (Small molecule drugs for viral infections and liver diseases)
- Endocyte, Inc. (ECYT) (Biopharmaceutical company: therapies for the treatment of cancer and inflammatory diseases)
- Epizyme, Inc. (EPZM) (Biopharmaceutical company: therapeutics for patients with genetically defined cancers)
- Eyenovia Inc. (EYEN) (IPO January 25, 2018; biopharmaceutical company: ophthalmology)
- Evolus, Inc. (EOLS) (IPO February 8, 2018; medical aesthetics)
- Exelixis, Inc. (EXEL) (Biopharmaceutical company: small molecule therapies used in the treatment of cancer)
- Fate Therapeutics, Inc. (FATE) (Biopharmaceutical company: cellular immunotherapeutics; cancer, rare genetic diseases and immune disorders)
- Fibrocell Science Inc. (FCSC) (Cell and gene therapy company: rare and serious skin and connective tissue diseases)
- FibroGen, Inc. (FGEN) (Biopharmaceutical company: anemia in chronic kidney disease, liver fibrosis, pancreatic cancer, corneal blindness)
- Five Prime Therapeutics, Inc. (FPRX) (Protein therapeutics)
- Flex Pharma, Inc. (FLKS) (Neuromuscular disorders including muscle cramps)
- Fortress Biotech (FBIO) (Biopharmaceutical company: cancer, additional products in pipeline)
- Forward Pharma (FWP) (Denmark: immunomodulatory compound dimethyl fumarate and derivatives used to treat immune disorders including multiple sclerosis and psoriasis)
- G1 Therapeutics, Inc. (GTHX) (IPO May 17, 2017; biopharmaceutical company: cancer)
- Galapagos NV (GLPG) (IPO in May 2015: Belgium: small molecule medicines; rheumatoid arthritis)
- Galectin Therapeutics Inc. (GALT) (Therapies for fibrotic diseases and cancer)
- Galmed Pharmaceuticals Ltd. (GLMD) (Israel, biopharmaceutical company: oral therapy for treating liver diseases and cholesterol gallstones)
- Gemphire Therapeutics Inc. (GEMP) (IPO August 5, 2016: biopharmaceutical company focused on therapies to treat dyslipidemia)
- Genocea Biosciences, Inc. (GNCA) (Vaccines and immunotherapies: infectious diseases, cancers and autoimmune diseases)
- Genprex, Inc. (GNPX) (IPO March 29, 2018; gene therapy: cancer)
- Geron Corporation (GERN) (Biopharmaceutical company: telomerase inhibitor in hematologic myeloid malignancies)
- Gilead Sciences, Inc. (GILD) (Biopharmaceutical company: cardiovascular, human immunodeficiency virus (HIV)
- Global Blood Therapeutics Inc. (GBT) (Therapeutics for patients with grievous blood-based disorders)
- GlycoMimetics, Inc. (GLYC) (Vaso-occlusive crisis of sickle cell disease; treatment for AML in combination with chemotherapy)
- Grifols, S.A. (GRFS) (Spain: Bioscience (treatment of medical conditions using blood or blood components)
- GTX, Inc. (GTXI) (Small molecules for the treatment of cancer and serious medical conditions)
- Halozyme Therapeutics, Inc. (HALO) (Oncology therapies that target the tumor microenvironment)
- Heat Biologics, Inc. (HTBX) (Therapeutic vaccines to combat a wide range of cancers and infectious diseases)
- Hemispherx BioPharma, Inc. (HEB) (Biopharmaceutical company: drug entities used in the treatment of viral and immune-based disorders)
- Homology Medicines, Inc. (FIXX) (IPO March 28, 2018; genetic medicines company)
- Horizon Pharma plc (HZNP) (Ireland, biopharmaceutical company: arthritis pain, rare diseases)
- Hutchison China MediTech Limited (HCM) (IPO March 17, 2016: Biopharmaceutical company focused on immunological diseases and oncology in addition to consumer health products)
- iBio, Inc. (IBIO) (Develops pharmaceutical product applications through its iBioLaunch platform)
- Idera Pharmaceuticals, Inc. (IDRA) (B-cell Lymphoma, autoimmune, rare)
- IDEXX Laboratories, Inc. (IDXX) (Diagnostic and information technology-based solutions)
- ImmuCell Corporation (ICCC) (Biotechnology company focused on improving animal health and productivity in the dairy and beef industries)
- Immune Design Corp. (IMDZ) (Immunotherapy company)
- ImmunoCellular Therapeutics, Ltd. (IMUC) (Immune-based therapies for the treatment of brain cancers and other cancers)
- ImmunoGen, Inc. (IMGN) (Tumor-targeting antibodies to deliver a cell-killing agent specifically to cancer cells)
- Immunomedics, Inc. (IMMU) (Biopharmaceutical company: antibody-based products for the targeted treatment of cancer, autoimmune and other serious diseases)
- Immutep Limited (IMMP) (Australia: immunotherapeutic products for the treatment of cancer)
- Imprimis Pharmaceuticals, Inc. (IMMY) (Ophthalmology and urology drug therapies)
- Incyte Corporation (INCY) (Biopharmaceutical company: small molecule drugs; oncology and inflammation)
- Infinity Pharmaceuticals, Inc. (INFI) (Biopharmaceutical company: Blood cancers, indolent non-Hodgkin lymphoma, chronic lymphocytic leukemia)
- InflaRx N.V. (IFRX) (IPO November 8, 2017; biopharmaceutical company: C5a inhibitors)
- Innoviva, Inc. (INVA) (Royalty: bio-pharmaceuticals, respiratory assets)
- Inovio Pharmaceuticals, Inc. (INO) (Cancer, infectious diseases, vaccines)
- Insmed, Inc. (INSM) (Biopharmaceutical company: lung disease and pulmonary arterial hypertension)
- Intec Pharma Ltd. (NTEC) (IPO in August 2015: Israel, biopharmaceutical company: oral drug delivery system, Parkinson’s disease)
- Intellia Therapeutics, Inc. (NTLA) (IPO May 6, 2016: gene editing company)
- Intercept Pharmaceuticals, Inc. (ICPT) (Biopharmaceutical company: orphan and more prevalent liver diseases)
- Intra-Cellular Therapies Inc. (ITCI) (Biopharmaceutical company: drugs for the treatment of diseases and disorders of the Central Nervous System)
- Intrexon Corporation (XON) (Products for the consumer, energy, food, health and environmental industries)
- Ionis Pharmaceuticals, Inc. (IONS) (RNA-targeted drug discovery and development: cancer, cardiovascular, metabolic, neurological disorders, rare diseases)
- Iovance Biotherapeutics, Inc. (IOVA) (Immunotherapies based on tumor infiltrating lymphocytes)
- Jaguar Animal Health (JAGX) (IPO in May 2015: Animal health: gastrointestinal products)
- Jazz Pharmaceuticals Plc. (JAZZ) (Ireland, biopharmaceutical company: narcolepsy, lymphoblastic leukemia, hepatic veno-occlusive disease)
- Jounce Therapeutics, Inc. (JNCE) (IPO January 27, 2017; cancer immunotherapies)
- Kadmon Holdings, Inc. (KDMN) (IPO July 27, 2016: biopharmaceutical company developing product candidates within autoimmune and fibrotic diseases, oncology and genetic diseases)
- Kala Pharmaceuticals, Inc. (KALA) (IPO July 20, 2017; biopharmaceutical company: eye diseases)
- Kamada Ltd. (KMDA) (Israel, biopharmaceutical company: specialty therapeutics developed through a protein purification technology)
- Kazia Therapeutics (KZIA) (Biotech company: oncology)
- Keryx Biopharmaceuticals, Inc. (KERX) (Biopharmaceutical company: renal disease)
- Kindred Biosciences, Inc. (KIN) (Biotechnology company that develops medicines for pets)
- Kitov Pharamceuticals Holdings Ltd. (KTOV) (Israel: anti-inflammatory drugs that treat pain without raising blood pressure)
- Krystal Biotech, Inc. (KRYS) (IPO September 20, 2017; biotech company: treatments for dermatological diseases utilizing gene therapy)
- Kura Oncology, Inc. (KURA) (IPO in November 2015: Biopharmaceutical company; therapeutics for the treatment of solid tumors and blood cancers)
- La Jolla Pharmaceutical Company (LJPC) (Biopharmaceutical company: therapies for patients suffering from life-threatening diseases)
- Leap Therapeutics, Inc. (LPTX) (Cancer therapies)
- Lexicon Pharmaceuticals, Inc. (LXRX) (Biopharmaceutical company: treatment of type 1 and type 2 diabetes, treatment of carcinoid syndrome)
- Ligand Pharmaceuticals Incorporated (LGND) (Biopharmaceutical company: licensing, royalty-generating assets)
- Loxo Oncology, Inc. (LOXO) (Cancer therapies)
- Lpath, Inc. (LPTN) (Monoclonal antibodies that target bioactive lipids)
- MabVax Therapeutics Holdings, Inc. (MBVX) (Monoclonal antibody-based therapeutics and vaccines for cancer)
- MacroGenics, Inc. (MGNX) (Biopharmaceutical company: monoclonal antibody-based therapeutics for the treatment of cancer and autoimmune diseases)
- Mallinckrodt Plc. (MNK) (Ireland, biopharmaceutical company: ADHD, autoimmune, central nervous system disorders, pain, rare diseases; medical imaging: contrast media and delivery systems, radiopharmaceuticals)
- MannKind Corporation (MNKD) (Biopharmaceutical company: therapeutic products for diseases which include diabetes)
- Marinus Pharmaceuticals, Inc. (MRNS) (Biopharmaceutical company: neuropsychiatric therapeutics)
- Mateon Therapeutics, Inc. (MATN) (Biopharmaceutical company: vascular disrupting agents for orphan oncology indications)
- Matinas Biopharma Holdings, Inc. (MTNB) (Anti-infectives for orphan indications)
- MediciNova, Inc. (MNOV) (Biopharmaceutical company: neurological disorders, treatment of acute exacerbations of asthma)
- MediWound Ltd. (MDWD) (Israel, Biopharmaceutical company: severe burns, chronic and other hard-to-heal wounds)
- MEI Pharma, Inc. (MEIP) (Therapies for the treatment of cancer)
- Menlo Therapeutics Inc. (MNLO) (IPO January 25, 2018; biopharmaceutical company: pruritus associated with certain dermatologic conditions)
- Merrimack Pharmaceuticals, Inc. (MACK) (Biopharmaceutical company: cancer)
- Mersana Therapeutics, Inc. (MRSN) (IPO June 28, 2017; biopharmaceutical company: cancer)
- Merus N.V. (MRUS) (IPO May 19, 2016, Netherlands: immuno-oncology company)
- Mesoblast Ltd. (MESO) (IPO in November 2015, Australia: regenerative medicine company; areas of focus include inflammation, cardiovascular disease and back pain)
- Minerva Neurosciences, Inc. (NERV) (Biopharmaceutical company: central nervous system diseases)
- Miragen Therapeutics, Inc. (MGEN) (RNA-targeted therapeutics)
- Mirati Therapeutics, Inc. (MRTX) (Oncology company: genetically-targeted oncology therapeutics)
- Momenta Pharmaceuticals, Inc. (MNTA) (Therapeutics for oncology and autoimmune indications)
- MyoKardia Inc. (MYOK) (IPO in October 2015: Biopharmaceutical company; therapies for serious and neglected rare cardiovascular diseases)
- MYOS Corporation (MYOS) (BioTherapeutics and BioNutrition company: muscle health and function)
- Myovant Sciences Ltd. (MYOV) (IPO October 27, 2016: biopharmaceutical company, women’s health and other endocrine-related disorders)
- Nabriva Therapeutics AG (NBRV) (IPO in September 2015: Austria, biopharmaceutical company: anti-infectives designed to treat bacterial infections)
- NanoViricides, Inc. (NNVC) (Nanotechnology-based biomimetic anti-viral medicines)
- Nantkwest Inc. (NK) (IPO in June 2015: Immunotherapy company: cancer)
- Nektar Therapeutics (NKTR) (Biopharmaceutical company: anti-infective, anti-viral, immunology, oncology, pain)
- Neptune Technologies & Bioresources Inc. (NEPT) (Canada: omega-3 dietary supplements)
- Neuralstem, Inc. (CUR) (Production of central nervous system stem cells used in the treatment of central nervous system diseases)
- Neurocrine Biosciences, Inc. (NBIX) (Neurological and endocrine based diseases and disorders)
- NewLink Genetics Corporation (NLNK) (Biopharmaceutical company: immunotherapy products, pancreatic cancer)
- Nightstar Therapeutics plc (NITE) (IPO September 28, 2017; gene therapy company: rare inherited retinal diseases)
- Northwest Biotherapeutics, Inc. (NWBO) (Cancer vaccines designed to treat a broad range of solid tumor cancers)
- NovaBay Pharmaceuticals, Inc. (Biopharmaceutical company: anti-infective treatments, ophthalmology)
- Novavax, Inc. (NVAX) (Vaccine company; products to prevent infectious diseases)
- NovoCure Ltd. (NVCR) (IPO in October 2015, oncology company: treatment of solid tumor cancers)
- NuCana plc (NCNA) (IPO September 28, 2017; biopharmaceutical company: medicines designed to overcome key cancer resistance mechanisms using proprietary ProTide technology)
- Nymox Pharmaceutical Corporation (NYMX) (Drugs and diagnostic products for the aging population; treatment for enlarged prostate or benign prostatic hyperplasia)
- ObsEva SA (OBSV) (IPO January 26, 2017; women’s reproductive health and pregnancy)
- Ocular Therapeutix, Inc. (OCUL) (Biopharmaceutical company: therapies for diseases and conditions of the eye through its hydrogel platform technology)
- Ohr Pharmaceuticals, Inc. (OHRP) (Ocular diseases)
- Omeros Corporation (OMER) (Biopharmaceutical company: small-molecule and protein therapeutics targeting inflammation, coagulopathies and disorders of the central nervous system)
- Oncobiologics, Inc. (ONS) (IPO May 13, 2016, biopharmaceutical company: biosimilar therapeutics)
- OncoMed Pharmaceuticals, Inc. (OMED) (Biopharmaceutical company: antibody therapeutics targeting cancer stem cells)
- Onconova Therapeutics, Inc. (ONTX) (Biopharmaceutical company: small molecule drug candidates used in the treatment of cancer)
- OncoSec Medical Inc. (ONCS) (Technologies designed to stimulate the body’s immune system; cancer)
- Ophthotech Corporation (OPHT) (Biopharmaceutical company: back of the eye diseases)
- Oragenics, Inc. (OGEN) (Biopharmaceutical company: oral probiotics products and antibiotics for humans and companion pets)
- Organovo Holdings, Inc. (ONVO) (3D bioprinting technology)
- Otonomy, Inc. (OTIC) (Biopharmaceutical company: therapeutics for the treatment of diseases and disorders of the ear)
- Ovascience Inc. (OVAS) (Life sciences company: treatments for infertility)
- Ovid Therapeutics Inc. (OVID) (IPO May 5, 2017; biopharmaceutical company: rare neurological disorders)
- Pacific Biosciences of California, Inc. (PACB) (Systems for gene sequencing; platform that enables real-time analysis of biomolecules with single molecule resolution)
- Pain Therapeutics (PTIE) (Biopharmaceutical company; painkillers)
- Palatin Technologies, Inc. (PTN) (Biopharmaceutical company: female sexual dysfunction; development programs or drug candidates for other areas)
- Paratek Pharmaceuticals, Inc. (PRTK) (Antibiotics)
- PDL BioPharma, Inc. (PDLI) (Patents and royalty assets: antibody humanization patents)
- Pfenex Inc. (PFNX) (Non-glycosylated biosimilars)
- Pieris Pharmaceuticals, Inc. (PIRS) (Biotechnology company: areas of focus include cancer, asthma and anemia)
- Pluristem Therapeutics, Inc. (PSTI) (Israel: Cell therapies for a broad spectrum of diseases)
- Portola Pharmaceuticals, Inc. (PTLA) (Biopharmaceutical company: thrombosis and hematological diseases)
- Prana Biotechnology Ltd. (PRAN) (Australia: neurodegenerative diseases, Alzheimer’s and Huntington diseases)
- Progenics Pharmaceuticals Inc. (PGNX) (Oncology company: diagnostic and therapeutic needs of prostate cancer patients)
- ProQR Therapeutics N.V. (PRQR) (Netherlands: Cystic fibrosis and severe genetic disorders)
- Protagonist Therapeutics, Inc. (PTGX) (IPO August 11, 2016: biopharmaceutical company with a platform focused on discovering and developing peptide-based new chemical entities)
- Protalix BioTherapeutics, Inc. (PLX) (Israel, biopharmaceutical company: therapeutic proteins used in the treatment of genetic disorders including Gaucher disease and Fabry disease)
- Prothena Corporation plc (PRTA) (Ireland: therapeutic monoclonal antibodies directed specifically to disease-causing proteins)
- Proteon Therapeutics, Inc. (PRTO) (Biopharmaceutical company: kidney and vascular diseases)
- Proteostasis Therapeutics, Inc. (PTI) (IPO February 11, 2016: Biopharmaceutical company with an initial focus on cystic fibrosis)
- PTC Therapeutics, Inc. (PTCT) (Biopharmaceutical company: oral treatments)
- Pulmatrix Inc. (PULM) (Biopharmaceutical company: therapies to address pulmonary disease)
- Puma Biotechnology Inc. (PBYI) (Biopharmaceutical company: products for the treatment of various forms of cancer)
- Qiagen N.V. (QGEN) (Netherlands: Sample & assay technologies: automated systems and consumable products; bioinformatics)
- Ra Pharmaceuticals, Inc. (RARX) (IPO October 26, 2016: biopharmaceutical company, therapeutics for complement-mediated diseases)
- Radius Health, Inc. (RDUS) (Biopharmaceutical company: osteoporosis, endocrine-mediated diseases)
- Redhill Biopharma Ltd. (RDHL) (Israel, biopharmaceutical company: inflammatory and gastrointestinal diseases, gastrointestinal cancers)
- Regeneron Pharmaceuticals, Inc. (REGN) (Biopharmaceutical company: Antibody technologies; cardiovascular, diabetes, eye diseases, infectious diseases, inflammation, oncology, pain)
- Regenxbio Inc. (RGNX) (IPO in September 2015: In vivo gene therapy products designed to treat various diseases)
- Regulus Therapeutics Inc. (RGLS) (Biopharmaceutical company: microRNA therapeutics for oncology indications and orphan diseases)
- resTORbio, Inc. (TORC) (IPO January 26, 2018; biopharmaceutical company: aging-related diseases)
- Retrophin, Inc. (RTRX) (Biopharmaceutical company: catastrophic diseases affecting children)
- Revance Therapeutics, Inc. (RVNC) (Biopharmaceutical company: botulinum toxin products used in aesthetic and therapeutic applications)
- Rexahn Pharmaceuticals, Inc. (RNN) (Biopharmaceutical company: therapeutics for the treatment of cancer)
- Rhythm Pharmaceuticals, Inc. (RYTM) (IPO October 5, 2017; biopharmaceutical company: rare genetic deficiencies that lead to metabolic disorders)
- Rigel Pharmaceuticals, Inc. (RIGL) (Small-molecule drugs for the treatment of immune diseases and cancers)
- Rocket Pharmaceuticals, Inc. (RCKT) (Biotech company: gene therapies for rare diseases)
- Rockwell Medical, Inc. (RMTI) (Biopharmaceutical company: end-stage renal disease and chronic kidney disease)
- RXI Pharmaceuticals Corporation (RXII) (Therapeutics in the areas of dermatology and ophthalmology)
- Sangamo BioSciences, Inc. (SGMO) (Biopharmaceutical company: infectious diseases, monogenic diseases, hemoglobinopathies)
- Sage Therapeutics, Inc. (SAGE) (Biopharmaceutical company: rare central nervous system disorders)
- Sarepta Therapeutics, Inc. (SRPT) (Biopharmaceutical company: RNA-based therapeutics; rare and infectious diseases)
- Seattle Genetics, Inc. (SGEN) (Antibody-based therapies for the treatment of cancer)
- Selecta Biosciences, Inc. (SELB) (IPO June 22, 2016: clinical-stage biopharmaceutical company focused on products designed to modulate the immune system)
- SELLAS Life Sciences Group, Inc. (SLS) (Biopharmaceutical company: cancer immunotherapies)
- Sesen Bio, Inc. (SESN) (Antibody-drug conjugate (ADC)
- Sienna Biopharmaceuticals, Inc. (SNNA) (IPO July 27, 2017; biopharmaceutical company: dermatology and aesthetics)
- Sierra Oncology, Inc. (SRRA) (Oncology company)
- Shire Plc. (SHPG) (Ireland, biopharmaceutical company: gastrointestinal, internal medicine, rare diseases, neuroscience)
- Sinovac Biotech, Ltd. (SVA) (China: Biopharmaceutical company: vaccines that protect against human infectious diseases)
- Solid Biosciences Inc. (SLDB)
- Soleno Therapeutics, Inc. (SLNO) (Rare diseases)
- Sophiris Bio, Inc. (SPHS) (Biopharmaceutical company: urological diseases)
- Sorrento Therapeutics, Inc. (SRNE) (Oncology company: treatments for cancer and associated pain)
- Spark Therapeutics, Inc. (ONCE) (Gene therapies: treatments for debilitating genetic diseases)
- Spectrum Pharmaceuticals, Inc. (SPPI) (Hematology, oncology)
- Spero Therapeutics, Inc. (SPRO) (IPO November 2, 2017; biopharmaceutical company: multi-drug resistant bacterial infections)
- Stellar Biotechnologies, Inc. (SBOT) (Immunotherapy)
- Stemline Therapeutics, Inc. (STML) (Biopharmaceutical company: therapeutics that target cancer stem cells and tumor bulk)
- Strongbridge Biopharma plc (SBBP) (IPO in October 2015: Biopharmaceutical company; rare endocrine disorders and other rare diseases)
- Summit Therapeutics Plc. (SMMT) (IPO in March 2015: United Kingdom, biopharmaceutical company: Duchenne Muscular Dystrophy, infectious disease caused by the bacteria C. difficile)
- Sunesis Pharmaceuticals, Inc. (SNSS) (Biopharmaceutical company: oncology therapeutics for the potential treatment of solid and hematologic cancers)
- Syndax Pharmaceuticals, Inc. (SNDX) (IPO March 3, 2016: Biopharmaceutical company engaged in the development of cancer treatments)
- Synergy Pharmaceuticals, Inc. (SGYP) (Biopharmaceutical company: therapies for the treatment of gastrointestinal diseases and disorders)
- Synthetic Biologics, Inc. (SYN) (Therapeutics to protect the microbiome)
- Syros Pharmaceuticals, Inc. (SYRS) (IPO June 30, 2016: Biopharmaceutical company, gene control medicines)
- Tenax Therapeutics, Inc. (TENX) (Treatment for various critical care conditions)
- Tesaro, Inc. (TSRO) (Biopharmaceutical company: oncology therapeutics and supportive care products)
- TetraLogic Pharmaceuticals Corporation (TLOG) (Biopharmaceutical company: cancer and infectious diseases)
- TG Therapeutics, Inc. (TGTX) (Biopharmaceutical company: B-cell malignancies and autoimmune diseases)
- Theravance Biopharma, Inc. (TBPH) (Biopharmaceutical company: diseases of the lung, gastrointestinal tract, infectious diseases)
- TiGenix NV (TIG) (IPO December 15, 2016: biopharmaceutical company, allogeneic stem cell therapies)
- Titan Pharmaceuticals, Inc. (TTNP) (Biopharmaceutical company: CNS disorders)
- Tocagen Inc. (TOCA) (IPO April 13, 2017; cancer-selective gene therapies)
- Tonix Pharmaceuticals Holding Corp. (TNXP) (Medicines for common disorders of the central nervous system: fibromyalgia, post-traumatic stress disorder, episodic tension-type headache)
- Tracon Pharmaceuticals, Inc. (TCON) (IPO in January 2015: Biopharmaceutical company: therapeutics for cancer, age-related macular degeneration and fibrotic diseases)
- Trevena, Inc. (TRVN) (Biopharmaceutical company: discovery and development of GPCR biased ligands)
- Trillium Therapeutics Inc. (TRIL) (Canada: immuno-oncology company)
- Ultragenyx Pharmaceutical Inc. (RARE) (Biopharmaceutical company: debilitating genetic diseases)
- UniQure N.V. (QURE) (Netherlands: gene therapy)
- United Therapeutics Corporation (UTHR) (Pulmonary arterial hypertension)
- Unum Therapeutics Inc. (UMRX) (IPO March 29, 2018; biopharmaceutical company: immunotherapy products)
- UroGen Pharma Ltd. (URGN) (IPO May 4, 2017; biopharmaceutical company: urological conditions)
- Vanda Pharmaceuticals Inc. (VNDA) (Biopharmaceutical company: atopic dermatitis, Non-24-Hour Disorder, schizophrenia)
- Vascular Biogenics Ltd. (VBLT) (Israel: Treatments for cancer)
- VBI Vaccines Inc. (VBIV) (Biopharmaceutical company: vaccines)
- Verastem, Inc. (VSTM) (Biopharmaceutical company: drugs to treat cancer by the targeted killing of cancer stem cells)
- Vericel Corporation (VCEL) (Cellular therapies for treatment of severe diseases and conditions)
- Versartis, Inc. (VSAR) (Biopharmaceutical company: treatment of endocrine disorders)
- Vertex Pharmaceuticals Incorporated (VRTX) (Cystic fibrosis; additional ongoing research programs aimed at other serious and life-threatening diseases)
- Verona Pharma plc (VRNA) (IPO April 27, 2017; biopharmaceutical company: respiratory diseases)
- Veru Inc. (VERU) (Biopharmaceutical company: men’s and women’s health, FC2 female condom)
- Vical Incorporated (VICL) (Biopharmaceutical company: infectious diseases through DNA delivery technologies and therapeutic approaches)
- Viking Therapeutics (VKTX) (IPO in April 2015: Therapeutics for patients suffering from metabolic and endocrine disorders)
- VistaGen Therapeutics, Inc. (VTGN) (Biopharmaceutical company focused on treatments for central nervous system disorders)
- Vital Therapies, Inc. (VTL) (Cell-based system for the treatment of acute liver failure)
- VIVUS, Inc. (VVUS) (Biopharmaceutical company: weight management, erectile dysfunction)
- Voyager Therapeutics, Inc. (VYGR) (IPO in November 2015: clinical stage gene therapy company: central nervous system)
- Wave Life Sciences Ltd. (WVE) (IPO in November 2015: Biopharmaceutical company; stereopure nucleic acid therapeutic candidates)
- Xbiotech Inc. (XBIT) (IPO in April 2015, biopharmaceutical company: cancer antibodies)
- Xenon Pharmaceuticals Inc. (XENE) (Canada, biopharmaceutical company: lipoprotein lipase deficiency)
- Xencor, Inc. (XNCR) (Antibody therapeutics: autoimmune disorders, asthma and allergic diseases, cancer)
- XOMA Corporation (XOMA)
- XTL Biopharmaceuticals Ltd. (XTLB) (Israel, biopharmaceutical company: autoimmune diseases; blood cancer)
- Zafgen, Inc. (ZFGN) (Biopharmaceutical company: obesity)
- Zai Lab Limited (ZLAB) (IPO September 20, 2017; biopharmaceutical company: autoimmune diseases, cancer, infectious diseases)
- Zealand Pharma A/S (ZEAL) (IPO August 9, 2017; biotechnology company: gastrointestinal and metabolic diseases)
- Ziopharm Oncology Inc. (ZIOP) (Biopharmaceutical company: cell-based therapies for the treatment of cancer)
- Zymeworks Inc. (ZYME) (IPO April 28, 2017; biopharmaceutical company: cancer)
- Zynerba Pharmaceuticals Inc. (ZYNE) (Synthetic cannabinoid treatments)
Risks & Considerations
Clinical Trial Failure
The majority of biotech stocks are clinical-stage, meaning their value relies on FDA approvals. A single Phase III failure can result in an overnight market cap loss of 50-90%.
Cash Burn & Dilution
Non-revenue generating biotechs often face high "burn rates" for R&D. Investors face constant risk of secondary offerings that dilute existing share value.
Regulatory & Patent Cliffs
Even for profitable biotechs, the expiration of key patents or changes in government drug pricing legislation can significantly impact long-term cash flow and valuation multiples.
Sector Concentration Risk
Biotech performance is often highly correlated. Macroeconomic shifts, such as interest rate hikes, can cause broad sell-offs across the entire sector regardless of individual pipeline success.
These risk factors are for educational purposes only and are not exhaustive. Individual investment decisions should be based on thorough due diligence.
Frequently Asked Questions
As of 2026, the largest biotech companies by market cap are Vertex Pharmaceuticals (VRTX) at $112.9B and Regeneron (REGN) at $79.9B. Together with BioNTech (BNTX), they lead a Top 25 group valued at over $1.8 trillion.
Analysts currently favor stocks with high Zen Scores and strong fundamentals, including Xeris Biopharma (XERS), BioCryst (BCRX), and Axsome Therapeutics (AXSM), especially those with oncology or AI catalysts.
There are over 500 publicly traded biotech companies listed on U.S. exchanges, with the vast majority trading on the NASDAQ. This count is dominated by small and micro-cap research firms.
Notable NASDAQ biotech leaders include 10x Genomics (TXG), 4D Molecular Therapeutics (FDMT), and AbCellera (ABCL), which represent the cutting edge of clinical-stage genomics.
Vertex remains the undisputed #1 at $112.9B. While the total sector cap decreased slightly from late 2025, mid-cap standouts like Axsome ($9.4B) continue to show relative strength.
Sector ETFs have shown strong recovery in 2026, with the XBI +23% and IBB +18% YOY. The ARK Genomic Revolution ETF (ARKG) is also up 17% tracking the genomic advancement space.
Key 2026 trends include the mainstream adoption of AI scenario modeling for drug trials, precision oncology breakthroughs, and a massive pivot toward targeted genetic therapies.
Nano and small-cap names like SXTP, AARD, and ABEO remain highly volatile but are frequently targeted for acquisition by larger pharmaceutical companies looking to bolster their pipelines.
Last updated April 2026 · Data sourced from U.S. exchange filings