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

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.

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.

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 span cardiometabolic and obesity candidates, oncology bispecific antibodies, and additional inflammation and rare-disease programs in development.

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.

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.

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.

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.

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.

Moderna is a biotechnology company built on messenger RNA technology, known for its COVID-19 vaccine. Its approved portfolio also includes respiratory vaccines in the RSV and influenza categories. Pipeline areas span latent and pandemic infectious-disease vaccines, individualized cancer programs, and rare-disease and therapeutic candidates developed on its mRNA platform.

Revolution Medicines is an oncology company developing targeted therapies aimed at RAS-driven cancers, a family of genetic mutations common in difficult-to-treat tumors. Its lead candidate completed a pivotal trial in metastatic pancreatic cancer and is under review by the FDA. Pipeline areas include additional RAS-directed inhibitors in trials across several tumor types.

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. It has expanded into the ophthalmology and immunology areas through acquisition. Pipeline areas span neurology, neuropsychiatry, immunology, and rare-disease programs across biologics and antisense medicines.

Insmed is a biotechnology company focused on serious and rare respiratory and inflammatory diseases. Its marketed products cover a refractory lung infection and bronchiectasis, a chronic airway condition. Pipeline areas span pulmonary hypertension, additional respiratory candidates, gene therapy, and inflammation programs across several mechanisms.

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.

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.

Neurocrine Biosciences is a biotechnology company focused on the neuroscience and endocrinology areas. Its marketed products cover movement disorders, endocrine conditions, and a rare genetic obesity syndrome added through acquisition. 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.

BioMarin Pharmaceutical is a biotechnology company focused on rare genetic diseases. Its products include enzyme therapies and treatments in the skeletal, metabolic, and lysosomal-storage categories, broadened through its acquisition of Amicus Therapeutics. Pipeline areas span skeletal, metabolic, and other rare-disease programs across enzyme, small-molecule, and gene-therapy approaches.

Arrowhead Pharmaceuticals is a biotechnology company developing RNA interference medicines using its targeted delivery platform. Its first approved product reduces triglycerides in adults with familial chylomicronemia syndrome and is cleared in the United States, Europe, and other markets. Pipeline and partnered programs span the cardiometabolic, pulmonary, liver, and rare-disease areas.

Ionis Pharmaceuticals is a biotechnology company specializing in antisense oligonucleotide medicines that target RNA. Its independently marketed and partnered products span the cardiometabolic, neurological, and rare-disease categories, including severe hypertriglyceridemia, amyloidosis, and hereditary angioedema. Pipeline areas span neurology, cardiometabolic, and additional rare-disease antisense programs.

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.

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.

Roivant Sciences is a biopharmaceutical company that builds subsidiary companies to develop and commercialize drug candidates across multiple therapeutic areas, with a concentration in immunology and inflammation. Its lead medicine is preparing for commercial launch in a rare autoimmune muscle disease, and additional late-stage studies are underway in other inflammatory conditions.

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.

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.

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

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.

BridgeBio Pharma is a biotechnology company that develops medicines for genetic diseases through a network of focused programs. Its marketed product is a transthyretin stabilizer in the cardiomyopathy category, and it has applications under FDA review in a rare calcium disorder and a form of limb-girdle muscular dystrophy. Pipeline areas include achondroplasia and additional genetic conditions.

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.

Madrigal Pharmaceuticals is a biotechnology company focused on the liver and metabolic-disease areas. Its marketed product is a thyroid hormone receptor-beta agonist in the metabolic dysfunction-associated steatohepatitis category. Pipeline areas include a licensed oral DGAT-2 inhibitor and small interfering RNA programs in development for the same disease area.

Corcept Therapeutics is a biopharmaceutical company that develops medicines regulating the effects of cortisol. Its marketed products include a treatment in the endogenous hypercortisolism category and a selective glucocorticoid receptor antagonist approved with chemotherapy for platinum-resistant ovarian cancer. Pipeline areas span additional oncology, metabolic, and endocrine programs built on its cortisol-modulator platform.

Axsome Therapeutics is a biopharmaceutical company focused on the central nervous system area. Its marketed products cover the major depressive disorder, narcolepsy, and Alzheimer's disease agitation categories. Pipeline areas include late-stage programs in attention deficit hyperactivity disorder and fibromyalgia, alongside additional neuropsychiatric and neurological candidates 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.

Cytokinetics is a biopharmaceutical company focused on muscle biology, developing therapies in the cardiovascular and neuromuscular areas. Its cardiac myosin inhibitor is approved for obstructive hypertrophic cardiomyopathy in the United States, Europe, and other markets. Pipeline areas include non-obstructive hypertrophic cardiomyopathy and additional muscle-directed candidates across several mechanisms.

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.

Alkermes is a biopharmaceutical company focused on neuroscience. Its marketed products cover the schizophrenia, bipolar disorder, addiction, and narcolepsy categories. Pipeline areas include an orexin-2 receptor agonist in late-stage trials for narcolepsy and an extended-release medicine studied in idiopathic hypersomnia, alongside additional sleep and psychiatry programs.

Rhythm Pharmaceuticals is a biotechnology company focused on rare neuroendocrine and metabolic diseases. Its marketed melanocortin-4 receptor agonist is approved in the rare genetic obesity category and for obesity caused by injury to the hypothalamus. Pipeline areas include additional weight-regulation candidates and programs in other rare metabolic and obesity syndromes.

Mirum Pharmaceuticals is a biopharmaceutical company focused on rare liver diseases. Its marketed products are in the cholestatic liver disease category, covering conditions that impair bile flow in children and adults. Pipeline areas include late-stage programs in additional cholestatic conditions and other rare hepatic and metabolic disorders.

Travere Therapeutics is a biopharmaceutical company focused on rare kidney and metabolic diseases. Its marketed product is approved in the immunoglobulin A nephropathy and focal segmental glomerulosclerosis categories, two rare conditions affecting the filtering units of the kidney. Pipeline areas include a licensed BTK inhibitor and additional rare kidney programs in clinical development.

PTC Therapeutics is a biopharmaceutical company focused on rare disorders. Its approved products include a treatment in the phenylketonuria category and a gene therapy for a rare neurotransmitter disorder. Pipeline areas include Huntington's disease, Fabry disease, and additional rare metabolic and neurological programs across small molecules and gene therapies.

Acadia Pharmaceuticals is a biopharmaceutical company focused on central nervous system disorders and rare diseases. Its marketed products are in the Parkinson's disease psychosis category and in a rare neurodevelopmental disorder category. Pipeline areas include additional neuropsychiatric and rare-disease candidates in clinical development across several mechanisms.

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.

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.

Harmony Biosciences is a biopharmaceutical company focused on rare neurological disorders. Its marketed product is a histamine-3 receptor medicine in the narcolepsy category for adult and pediatric patients. Pipeline areas include an orexin-2 receptor agonist and additional late-stage candidates for sleep disorders and other rare neurological conditions.

Sarepta Therapeutics is a 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 small interfering RNA programs for myotonic dystrophy, facioscapulohumeral dystrophy, and Huntington's disease, alongside additional genetic-medicine approaches.

ADMA Biologics is a biopharmaceutical company that develops and manufactures plasma-derived biologics. Its approved products are immune globulin therapies in the immune-deficiency category, and it runs its own plasma collection and manufacturing operations. Pipeline areas include additional plasma-derived products and label expansions for its existing therapies.

Intellia Therapeutics is a gene-editing biotechnology company developing CRISPR-based medicines, including in vivo editing programs delivered systemically. Its lead candidate completed Phase 3 testing in hereditary angioedema and is the subject of a marketing application to the FDA. Pipeline areas include amyloidosis and additional in vivo and ex vivo editing programs across genetic and immunological diseases.

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.

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.

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.

Elanco Animal Health is an animal-health pharmaceutical company that develops medicines, vaccines, and parasiticides for livestock and companion animals. Its portfolio includes products in the dermatology, parasite-control, and vaccine categories for pets and farm animals. Pipeline areas include monoclonal antibodies and additional companion-animal and livestock products.

Amneal Pharmaceuticals is a pharmaceutical company that develops and manufactures generic medicines alongside a branded specialty portfolio. Its branded products include treatments in the Parkinson's disease category, and it markets injectable, inhalation, and ophthalmic products. Pipeline areas include biosimilars, additional complex generics, and further specialty candidates.

Organon is a pharmaceutical company formed through a spinoff, with a portfolio spanning women's health, dermatology, established brands, and biosimilars. Its products include a non-steroidal cream in the inflammatory skin-condition category and biosimilar versions of several biologic medicines. Pipeline areas include additional biosimilars and women's health programs.