Biotech & Pharma Stocks: Big Pharma, Biotech & Gene Editing

AstraZeneca is a UK-based big pharma company with portfolios in the oncology, cardiovascular, renal, metabolic, respiratory, immunology, and rare-disease areas. Its oncology business includes targeted therapies and antibody-drug conjugates developed with partners. Pipeline areas span cell therapy, metabolic candidates, and additional oncology and rare-disease programs.

Novartis is a Swiss big pharma company focused on patented prescription medicines after divesting its generics and eye-care units. Therapeutic areas include cardiovascular, immunology, neuroscience, oncology, and hematology. Its pipeline includes radioligand therapies, gene therapies, and RNA-targeted programs in development across several disease areas.

Novo Nordisk is a Danish big pharma company concentrated in the diabetes-care, obesity, and rare-disease categories. Its incretin portfolio includes semaglutide-based GLP-1 medicines marketed in the type 2 diabetes and chronic weight-management categories. Pipeline areas include next-generation obesity and metabolic candidates and hemophilia and growth-disorder programs.

Amgen is a large biotechnology company with products in the inflammation, oncology, hematology, bone-health, cardiovascular, and rare-disease categories. Its portfolio includes biologics and biosimilars. Pipeline areas include the obesity candidate MariTide, an injectable GIPR-GLP-1 program in development, alongside oncology and inflammation assets.

Gilead Sciences is a large biotechnology company focused on antiviral medicines in the HIV and viral-hepatitis areas, plus oncology and inflammation. Its portfolio includes long-acting HIV products and cell-therapy assets. Pipeline areas span HIV, oncology including cell therapy, and liver-disease programs in development.

Pfizer is a big pharma company with products across the vaccines, oncology, internal-medicine, inflammation, and rare-disease categories. Its portfolio includes vaccines and oncology medicines, several added through acquisitions. Pipeline areas include oncology antibody-drug conjugates, obesity and metabolic candidates, and additional vaccine and rare-disease programs.

Bristol Myers Squibb is a big pharma company with portfolios in the oncology, hematology, immunology, cardiovascular, and neuroscience areas. Its products include checkpoint-inhibitor immunotherapies, cell therapies, and a neuroscience medicine. Pipeline areas cover oncology, immunology, cardiovascular, and neuroscience programs across several mechanisms.

Vertex is a large biotechnology company historically focused on the cystic fibrosis area. Its broader portfolio includes a non-opioid acute-pain medicine, Journavx, and a co-developed gene-edited cell therapy, Casgevy, in the inherited blood-disorder category. Pipeline areas include pain, kidney disease, type 1 diabetes cell therapy, and additional rare-disease programs.

Sanofi is a French big pharma company with portfolios in the immunology, vaccines, rare-disease, oncology, and neurology areas. Its products include an immunology biologic and a broad vaccine franchise. Pipeline areas span immunology and inflammation, vaccines, neurology, and rare-disease programs across multiple mechanisms.

GSK is a UK-based big pharma company organized around specialty medicines, general medicines, and vaccines. Therapeutic areas include HIV, respiratory and immunology, oncology, and infectious disease. Pipeline areas span vaccines, HIV long-acting options, oncology, and respiratory programs across several disease categories.

Regeneron is a large biotechnology company built on antibody discovery. Its products span the ophthalmology, immunology and inflammation, and oncology areas, several developed with Sanofi or Bayer. Pipeline areas include oncology bispecific antibodies, immunology, hematology, and genetic-medicine programs using its antibody and genetics platforms.

argenx is a Netherlands-based mid-cap biotechnology company focused on antibody therapies in the autoimmune-disease area. Its lead product targets the neonatal Fc receptor and is approved across several autoimmune indications. Pipeline areas include additional autoimmune and immunology programs derived from its antibody-engineering platform.

Alnylam is a mid-cap biotechnology company that pioneered RNA interference therapeutics. Its approved products use small interfering RNA to target genes associated with rare and cardiometabolic conditions, including hereditary and wild-type forms of amyloidosis. Pipeline areas include cardiometabolic, central nervous system, and additional rare-disease RNAi programs.

Biogen is a large biotechnology company focused on the neurology and neuroscience areas, including multiple sclerosis, spinal muscular atrophy, and Alzheimer's disease, with a rare-disease portfolio. Its products include neurology biologics and antisense medicines. Pipeline areas span neurology, neuropsychiatry, immunology, and rare-disease programs.

BioNTech is a Germany-based biotechnology company known for messenger RNA technology and its COVID-19 vaccine developed with Pfizer. Its pipeline emphasizes oncology, including individualized mRNA cancer immunotherapy candidates, bispecific antibodies, and cell therapies, alongside infectious-disease vaccine programs built on its mRNA and immunotherapy platforms.

Incyte is a mid-cap biopharmaceutical company with products in the oncology, hematology, and dermatology categories. Its portfolio includes a JAK inhibitor franchise spanning oral and topical formulations. Pipeline areas include oncology, myeloproliferative neoplasms, and inflammation and autoimmunity programs across small molecules and biologics.

Moderna is a biotechnology company built on messenger RNA technology, known for its COVID-19 vaccine. Its pipeline spans respiratory vaccines including RSV and influenza, latent and other infectious-disease vaccines, individualized cancer programs, and rare-disease and therapeutic candidates developed on its mRNA platform across multiple areas.

Neurocrine Biosciences is a mid-cap biotechnology company focused on the neuroscience and endocrinology areas. Its marketed products are in the movement-disorder and endocrine categories. Pipeline areas span neuropsychiatry, including schizophrenia, mood and movement disorders, and additional neurological and endocrine programs across small molecules.

Exelixis is a mid-cap oncology-focused biotechnology company. Its marketed franchise centers on a tyrosine kinase inhibitor used across several cancer categories, including kidney and liver cancer. Pipeline areas include next-generation small molecules and biologics targeting additional solid tumors developed through internal discovery and partnerships.

Ionis Pharmaceuticals is a mid-cap biotechnology company specializing in antisense oligonucleotide medicines that target RNA. Its products and partnered programs are in the neurological, cardiometabolic, and rare-disease categories, including amyloidosis and a rare lipid disorder. Pipeline areas span neurology, cardiometabolic, and rare-disease antisense programs.

BioMarin Pharmaceutical is a mid-cap biotechnology company focused on rare genetic diseases. Its products include enzyme therapies, a product in the growth-condition category, and a gene therapy in the bleeding-disorder category. Pipeline areas span skeletal, metabolic, and other rare-disease programs across enzyme, small-molecule, and gene-therapy approaches.

CRISPR Therapeutics is a gene-editing biotechnology company using CRISPR/Cas9 technology. With Vertex it co-developed Casgevy, a gene-edited cell therapy approved in the inherited blood-disorder category. Pipeline areas include in vivo and ex vivo editing programs across cardiovascular, immuno-oncology, autoimmune, and diabetes settings using its editing platform.

Beam Therapeutics is a gene-editing biotechnology company developing base-editing medicines that aim to make single-letter changes to DNA without double-strand breaks. Its pipeline includes ex vivo and in vivo programs in development for blood disorders, liver disease, and other genetic conditions on its base-editing platform.

Intellia Therapeutics is a gene-editing biotechnology company developing CRISPR-based medicines, including in vivo editing programs delivered systemically. Its lead candidates are in development for a form of amyloidosis and a chronic swelling condition. Pipeline areas include additional in vivo and ex vivo editing programs across genetic and immunological diseases.

Johnson & Johnson is a diversified healthcare company whose Innovative Medicine segment develops and markets drugs across the oncology, immunology, neuroscience, pulmonary hypertension, and infectious-disease areas. It also operates a large MedTech medical-device business. Pipeline areas include cell therapy, bispecific antibodies, and targeted oncology programs.

Takeda is a Japanese big pharma company with portfolios in the gastroenterology, rare-disease, plasma-derived therapies, oncology, and neuroscience areas. Its products include inflammatory-bowel-disease and rare-disease medicines and plasma-derived immunoglobulins. Pipeline areas span gastroenterology, rare genetic and hematologic disease, neuroscience, and oncology programs.

Teva is an Israel-based pharmaceutical company that is one of the largest generic-drug makers, complemented by a branded portfolio. Branded areas include neuroscience and respiratory, with a migraine medicine and a movement-disorder product. Pipeline areas include long-acting branded programs in neuroscience and immunology alongside biosimilars.

Revolution Medicines is a clinical-stage oncology company developing targeted therapies aimed at RAS-driven cancers, a family of genetic mutations common in difficult-to-treat tumors. Its pipeline focuses on small-molecule inhibitors directed at RAS and related pathways, and it is advancing multiple candidates through clinical trials.

United Therapeutics is a mid-cap biotechnology company focused on the pulmonary arterial hypertension area and organ-related products. Its portfolio includes several prostacyclin-based products delivered through different routes. Pipeline areas include additional pulmonary and cardiopulmonary programs and organ-manufacturing and xenotransplantation research aimed at expanding transplant supply.

Insmed is a mid-cap biotechnology company focused on rare and serious diseases. Its marketed product is in the refractory lung-infection category, and its pipeline includes a program in the bronchiectasis area and additional respiratory and rare-disease candidates. Pipeline areas span pulmonary disease, gene therapy, and inflammation across several mechanisms.

Genmab is an antibody-focused biotechnology company that discovers and develops therapeutic antibodies, several of which are marketed by partners. It uses proprietary antibody technology platforms to create cancer and other treatments, earning revenue from royalties and collaborations while advancing its own clinical pipeline.

Jazz Pharmaceuticals is a biopharmaceutical company with portfolios in the neuroscience and oncology areas. Its neuroscience products are in the sleep-disorder and epilepsy categories, including a cannabidiol medicine, and its oncology products are in certain blood and solid-tumor categories. Pipeline areas include additional neuroscience and oncology programs across several mechanisms.

Ascendis Pharma is a Denmark-based biotechnology company applying a prodrug technology that extends the release of therapeutic molecules. Its products are in the endocrinology rare-disease category, covering growth and parathyroid conditions. Pipeline areas span endocrinology rare disease and oncology programs built on its sustained-release platform.

Summit Therapeutics is a clinical-stage biotechnology company developing ivonescimab, a bispecific antibody designed to engage the PD-1 and VEGF pathways, licensed for certain regions. Its programs are in the non-small cell lung cancer and other solid-tumor categories, with multiple late-stage trials and regulatory submissions in oncology underway.

Axsome Therapeutics is a mid-cap biopharmaceutical company focused on the central nervous system area. Its marketed products are in the major depressive disorder and narcolepsy categories, and its pipeline includes programs in development for Alzheimer's disease agitation, migraine, and other neuropsychiatric and neurological conditions across several mechanisms.

Madrigal Pharmaceuticals is a mid-cap biotechnology company focused on the liver and metabolic-disease areas. Its marketed product, resmetirom, is a thyroid hormone receptor-beta agonist in the metabolic dysfunction-associated steatohepatitis category. Pipeline areas include a licensed oral GLP-1 candidate and additional metabolic and liver-disease programs in development.

Arrowhead Pharmaceuticals is a mid-cap biotechnology company developing RNA interference medicines using its targeted delivery platform. Its pipeline and partnered programs are in development for the cardiometabolic, pulmonary, liver, and rare-disease areas, including a partnered cardiovascular candidate. Pipeline areas span cardiometabolic, central nervous system, and rare-disease RNAi programs.

Cytokinetics is a biopharmaceutical company focused on muscle biology, developing therapies in the cardiovascular and neuromuscular areas. Its cardiac myosin inhibitor aficamten received FDA approval in late 2025, and the company continues to develop additional muscle-directed candidates through its clinical pipeline across several mechanisms.

Krystal Biotech is a mid-cap biotechnology company developing redosable gene therapies delivered with an engineered viral vector. Its marketed product is a topical gene therapy in the rare skin-disease category. Pipeline areas include respiratory, ophthalmology, oncology, and additional dermatology programs using its gene-delivery platform.

Vaxcyte is a clinical-stage vaccine biotechnology company using a cell-free protein synthesis platform to engineer vaccines. Its lead programs are pneumococcal conjugate vaccine candidates in development for adult and pediatric populations. Pipeline areas include additional conjugate-vaccine candidates in the bacterial-infection category developed on its protein-engineering platform.

Rhythm Pharmaceuticals is a mid-cap biotechnology company focused on rare neuroendocrine and metabolic diseases. Its marketed product is a melanocortin-4 receptor agonist in the rare genetic obesity category. Pipeline areas include additional rare obesity and hypothalamic-obesity programs and other metabolic candidates in development for weight-regulation pathways.

Viking Therapeutics is a clinical-stage biotechnology company focused on the metabolic and endocrine areas. Its lead candidate, VK2735, is a dual GLP-1 and GIP receptor agonist in development in injectable and oral formulations for the obesity category. Pipeline areas include additional metabolic, liver-disease, and endocrine programs in clinical development.

Ultragenyx Pharmaceutical is a mid-cap biotechnology company focused on rare and ultra-rare genetic diseases. Its marketed products are in several metabolic and bone categories. Pipeline areas include gene therapies and other candidates in development for rare metabolic, neuromuscular, and bone disorders developed across multiple therapeutic modalities.

Sarepta Therapeutics is a mid-cap biotechnology company focused on the rare neuromuscular-disease area. Its products include RNA-based exon-skipping therapies and a gene therapy in the Duchenne muscular dystrophy category. Pipeline areas span additional neuromuscular gene therapies and RNA programs and limb-girdle muscular dystrophy candidates across genetic-medicine approaches.

Zoetis is an animal-health pharmaceutical company that develops medicines, vaccines, and diagnostics for livestock and companion animals. Its portfolio spans parasiticides, anti-infectives, vaccines, and products in the dermatology, pain, and chronic-condition categories for pets. Pipeline areas include monoclonal antibodies and additional companion-animal and livestock products.

Roivant Sciences is a biopharmaceutical company that builds subsidiary companies to develop and commercialize drug candidates across multiple therapeutic areas, including immunology and inflammation. It in-licenses and advances programs through clinical development and also develops technology and AI tools to support drug discovery.

Viatris is a global pharmaceutical company formed from the combination of Mylan and Upjohn, with a broad portfolio of generic, branded, and biosimilar medicines. Its products span the cardiovascular, central nervous system, infectious-disease, and other categories. Pipeline areas include eye-care, dermatology, and additional complex-product and biosimilar programs.

Halozyme Therapeutics is a biopharmaceutical company whose enzyme-based drug-delivery technology enables subcutaneous delivery of injected biologics. It earns royalties and fees by licensing this platform to other drug developers and also markets proprietary products. Its business spans many partnered therapeutic areas through delivery-enabling collaborations.