Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases. Its marketed products include ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria (AHP); and OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1). In addition, the company is developing givosiran for the treatment of adolescent patients with AHP; patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; ALN-AAT02 for the treatment of AAT deficiency-associated liver disease; ALN-HBV02 to treat chronic HBV infection; Zilebesiran to treat hypertension; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia and rare bleeding disorders, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1 and recurrent renal stones, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Sanofi Genzyme to discover, develop, and commercialize RNAi therapeutics. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

Alnylam Pharmaceuticals, Inc.

Biogen Inc. discovers, develops, manufactures, and delivers therapies for treating neurological and neurodegenerative diseases. The company offers TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI, and FAMPYRA for multiple sclerosis (MS); SPINRAZA for spinal muscular atrophy; and FUMADERM to treat plaque psoriasis. It also provides BENEPALI, an etanercept biosimilar referencing ENBREL; ADUHELM for the treatment of Alzheimer's disease; IMRALDI, an adalimumab biosimilar referencing HUMIRA; and FLIXABI, an infliximab biosimilar referencing REMICADE. In addition, the company offers RITUXAN for treating non-Hodgkin's lymphoma, chronic lymphocytic leukemia (CLL), rheumatoid arthritis, two forms of ANCA-associated vasculitis, and pemphigus vulgaris; RITUXAN HYCELA for non-Hodgkin's lymphoma and CLL; GAZYVA to treat CLL and follicular lymphoma; and OCREVUS for treating relapsing MS and primary progressive MS; and other anti-CD20 therapies. Further, it develops BIIB135, BIIB061, BIIB091, and BIIB107 for MS and neuroimmunology; Aducanumab, Lecanemab, BIIB076, and BIIB080 to treat Alzheimer's disease and dementia; BIIB067, BIIB078, BIIB105, BIIB100, and BIIB110 to treat neuromuscular disorders; BIIB124, BIIB094, BIIB118, BIIB101, and BIIB122 for treating Parkinson's disease and movement disorders; BIIB125 and BIIB104 for treating neuropsychiatry; Dapirolizumab pegol and BIIB059 to treat immunology related diseases; BIIB093 and BIIB131 to treat acute neurology; BIIB074 for neuropathic pain; and BYOOVIZ, BIIB800, and SB15 biosimilars, which are under various stages of development. The company has collaboration and license agreements with Acorda Therapeutics, Inc.; Alkermes Pharma Ireland Limited; Denali Therapeutics Inc.; Eisai Co., Ltd.; Genentech, Inc.; Neurimmune SubOne AG; Ionis Pharmaceuticals, Inc.; Samsung Bioepis Co., Ltd.; Sangamo Therapeutics, Inc.; and Sage Therapeutics, Inc. Biogen Inc. was founded in 1978 and is headquartered in Cambridge, Massachusetts.

Biogen Inc.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of TTR amyloidosis; Olezarsen for patients with severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Tofersen to inhibit the production of superoxide dismutase 1. In addition, the company develops medicines for metabolic diseases, infectious diseases, renal diseases, ophthalmic diseases, and cancer. It has a strategic collaboration with Biogen Inc.; and collaboration and license agreement with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Ionis Pharmaceuticals, Inc.

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 6 years of age or older who have at least one F508del mutation. Its pipeline includes VX-864 for the treatment of AAT deficiency, which is in Phase 2 clinical trial; VX-147 for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases which is in Phase 2 clinical trial; VX- 880, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-548, a NaV1.8 inhibitor for treatments of acute, neuropathic, musculoskeletal pain which is in Phase 2 clinical trial; and CTX001 for the treatment severe SCD and TDT which is in Phase 3 clinical trial. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. It has collaborations with Affinia Therapeutics, Inc.; Arbor Biotechnologies, Inc.; CRISPR Therapeutics AG.; Kymera Therapeutics, Inc.; Mammoth Biosciences, Inc.; Moderna, Inc.; Obsidian Therapeutics, Inc.; and Skyhawk Therapeutics, Inc.; as well as Ribometrix, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany, and X-Chem, Inc. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

Vertex Pharmaceuticals Incorporated

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes ARO-AAT, a RNA interference (RNAi) therapeutic candidate that is in Phase II clinical trial for the treatment of liver diseases associated with alpha-1 antitrypsin deficiency; ARO-APOC3, which is in phase 2b and one phase 3 clinical trial to treat hypertriglyceridemia; ARO-ANG3 that is in Phase 2b clinical trial to reduce production of angiopoietin-like protein 3; ARO-HSD, which is in Phase 1/2a clinical trial to treat liver diseases; ARO-ENaC, which is in a Phase 1/2a clinical trial to reduce production of the epithelial sodium channel alpha subunit in the airways of the lung; ARO-C3 for the treatment of complement-mediated disease that is in Phase 1/2a clinical trial; ARO-Lung2 for the treatment of chronic obstructive pulmonary disorder; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-XDH to treat uncontrolled gout; ARO-COV for the treatment of COVID-19 and other pulmonary-borne pathogens; and ARO-HIF2, which is in phase 1b clinical trial to treat clear cell renal cell carcinoma. It is also involved in the development of JNJ-3989, a subcutaneously administered RNAi therapeutic candidate to treat chronic hepatitis B virus infection; Olpasiran to reduce the production of apolipoprotein A; and ARO-AMG1 for treating genetically validated cardiovascular targets. Arrowhead Pharmaceuticals, Inc. has a license and research collaboration agreement with Janssen Pharmaceuticals, Inc. to develop ARO-JNJ1, ARO-JNJ2, and ARO-JNJ3 RNAi therapeutics for liver-expressed targets; and license and research collaboration agreement with Takeda Pharmaceuticals U.S.A., Inc. to develop RNAi therapeutic candidate as a treatment for liver disease. Arrowhead Pharmaceuticals, Inc. was incorporated in 1989 and is headquartered in Pasadena, California.

Arrowhead Pharmaceuticals, Inc.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; and VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. The company is also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

Sarepta Therapeutics, Inc.

BioNTech SE, a biotechnology company, develops and commercializes immunotherapies for cancer and other infectious diseases. The company is developing FixVac product candidates, including BNT111, which is in Phase II clinical trial for advance melanoma; BNT112 that is in Phase I/IIa clinical trial for prostate cancer; BNT113, which is in Phase II clinical trial to treat HPV+ head and neck cancers; BNT114 that is in Phase I clinical trial for triple negative breast cancer; BNT115, which is in Phase I clinical trial in ovarian cancer; and BNT116, a preclinical stage product for non-small cell lung cancer. It also develops neoantigen specific immunotherapies, such as Autogene cevumeran (BNT122), which is in Phase II clinical trial for first-line melanoma, as well as in Phase 1a/1b clinical trial to treat multiple solid tumors; mRNA intratumoral immunotherapy comprising SAR441000 that is in Phase I clinical trial for solid tumors; and BNT141 and BNT142 that are in Phase I clinical trial to treat multiple solid tumors. In addition, the company develops RiboCytokines, which include BNT151, BNT152, and BNT153 to treat solid tumors; chimeric antigen receptor T cell immunotherapies, such as BNT211 to treat multiple solid tumors, and BNT221 for other cancers; and checkpoint immunomodulators consisting of GEN1046 and GEN1042, which are in Phase I/II clinical trial to treat solid tumors. Further, it develops BNT321, an IgG1 monoclonal antibody in Phase II clinical trial for pancreatic cancer; BNT411, a small molecule immunomodulator product candidate for solid tumors; prophylactic vaccine for COVID-19 and Influenza; and infectious disease immunotherapies and rare disease protein replacement therapies. The company has collaborations with Genentech, Inc.; Sanofi S.A.; Genmab A/S; Pfizer Inc.; Shanghai Fosun Pharmaceutical (Group) Co., Ltd.; and Regeneron Pharmaceuticals, Inc. BioNTech SE was incorporated in 2008 and is headquartered in Mainz, Germany.

BioNTech SE

Moderna, Inc., a biotechnology company, discovers, develops, and commercializes messenger RNA therapeutics and vaccines for the treatment of infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases, and auto-immune diseases in the United States, Europe, and internationally. Its respiratory vaccines include COVID-19, flu, respiratory syncytial virus, Endemic HCoV, and hMPV+PIV3 vaccines; latent vaccines comprise cytomegalovirus, epstein-barr virus, human immunodeficiency virus, herpes simplex virus, and varicella-zoster virus vaccines; and public health vaccines consists of Zika and Nipah vaccines. The company also offers systemic secreted and cell surface therapeutics; cancer vaccines, such as personalized cancer, KRAS, and checkpoint vaccines; intratumoral immuno-oncology products; localized regenerative, systemic intracellular, and inhaled pulmonary therapeutics. It has strategic alliances with AstraZeneca PLC; Merck & Co., Inc.; Vertex Pharmaceuticals Incorporated; Vertex Pharmaceuticals (Europe) Limited; Carisma Therapeutics, Inc.; Metagenomi, Inc.; the Defense Advanced Research Projects Agency; Biomedical Advanced Research and Development Authority; Institute for Life Changing Medicines; and The Bill & Melinda Gates Foundation, as well as a collaboration and license agreement with Chiesi Farmaceutici S.P.A. The company was formerly known as Moderna Therapeutics, Inc. and changed its name to Moderna, Inc. in August 2018. Moderna, Inc. was founded in 2010 and is headquartered in Cambridge, Massachusetts.

Moderna, Inc.

Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 for the treatment of hereditary angioedema, as well as other liver-focused programs comprising hemophilia A and hemophilia B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo pipeline includes NTLA-5001 for the treatment of acute myeloid leukemia; and proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune disorders. In addition, it offers tools comprising of Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for the treatment of sickle cell disease; Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; Ospedale San Raffaele; and a strategic collaboration with SparingVision SAS to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. The company was formerly known as AZRN, Inc. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

Intellia Therapeutics, Inc.

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Ticker	Market Cap	Gross Profit Margin	Forward P/E
ALNY	$61,088.11M	83.9%	66.2
BIIB	$26,764.10M	76.3%	10.4
IONS	$12,908.12M	5.3%	-22.1
VRTX	$118,292.77M	52.7%	21.2
ARWR	$8,634.18M	100.0%	-22.1
SRPT	$2,218.83M	18.4%	70.9
BNTX	$23,129.41M	82.5%	-19.5
MRNA	$10,290.17M	-71.4%	-2.8
NTLA	$1,004.89M	-612.5%	-4.8