Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF). It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years of age or older; SYMDEKO/SYMKEVI for people with CF for 6 years of age or older; ORKAMBI for CF patients 1 year or older; and KALYDECO for the treatment of patients with 1 year or older who have CF with ivacaftor. The company's pipeline includes VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment of acute and neuropathic pain which is in Phase 3 clinical trial; Exa-cel, for the treatment of sickle cell disease and transfusion-dependent beta thalassemia which is in Phase 2/3 clinical trial. In addition, it provides inaxaplin for the treatment of APOL1-mediated focal segmental glomerulosclerosis and co-morbidities, such as hypertension which is in single Phase 2/3; VX- 880 and VX-264, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-970, which is in Phase 2 clinical trial for the treatment of cancer; and VX-803 and VX-984 for treatment of cancer in Phase 1 clinical trial. Further, it sells the products to specialty pharmacy and specialty distributors in the United States, as well as retail pharmacies, hospitals, and clinics. Additionally, the company has collaborations with CRISPR Therapeutics AG.; Moderna, Inc.; Entrada Therapeutics, Inc.; Arbor Biotechnologies, Inc.; Mammoth Biosciences, Inc.; and Verve Therapeutics., as well as collaborations with Tevard Biosciences to develop novel tRNA-based therapies for duchenne muscular dystrophy. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

BioCryst Pharmaceuticals, Inc., a biotechnology company, develops oral small-molecule and protein therapeutics to treat rare diseases. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names; and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema. It is also developing BCX10013, an oral factor D inhibitor for complement-mediated diseases. The company has collaborations and in-license relationships with the Torii Pharmaceutical Co., Ltd.; Seqirus UK Limited; Shionogi & Co., Ltd.; Green Cross Corporation; National Institute of Allergy and Infectious Diseases; Biomedical Advanced Research and Development Authority; the U.S. Department of Health and Human Services; and The University of Alabama at Birmingham, as well as Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd. BioCryst Pharmaceuticals, Inc. was founded in 1986 and is headquartered in Durham, North Carolina.

Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. It develops BEAM-101 for the treatment of sickle cell disease or beta-thalassemia; and BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; BEAM-201, an anti-CD7 CAR-T product candidate, which is in Phase 1/2 clinical trials for the treatment of refractory T-cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. The company has research collaboration agreement with Pfizer Inc., focus on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Apellis Pharmaceuticals to conduct preclinical research on target specific genes within the complement system in various organs, including the eye, liver, and brain; Verve Therapeutics, Inc., for cardiovascular disease treatments; Sana Biotechnology, Inc., to research, develop, and commercialize rights to CRISPR Cas12b for cell therapy programs; and Orbital Therapeutics design RNA for the prevention, treatment or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.

Korro Bio, Inc., a biopharmaceutical company, engages in the discovery, development, and commercialization of genetic medicines based on editing RNA for the treatment of rare and highly prevalent diseases primarily in the United States. Its lead product candidate is KRRO-110 which is in preclinical trials for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). It uses its RNA editing platform, OPERA to generate differentiated RNA editing product candidates. The company is also developing programs for Parkinson's disease, Amyotrophic lateral sclerosis, severe alcohol-associated hepatitis, and other subsets of pain. Korro Bio, Inc. has a collaboration agreement with Novo Nordisk A/S for the discovery and development of new genetic medicines to treat cardiometabolic diseases. Korro Bio, Inc. was founded in 2018 and is based in Cambridge, Massachusetts.

4D Molecular Therapeutics, Inc., a clinical-stage biotherapeutics company, develops genetic medicines using its therapeutic vector evolution platform in the Netherland and the United States. The company develops a portfolio of genetic medicine product candidates focuses in three therapeutic areas for ophthalmology, cardiology, and pulmonology. Its product pipeline includes 4D-150, which is in phase 1/2 clinical trial for the treatment of wet age-related macular degeneration; and is in phase 1/2 clinical trial for the treatment of diabetic macular edema. The company develops 4D-125, which is in phase 1/2 clinical trial for the treatment of x-linked retinitis pigmentosa; 4D-110, which is in phase 1/2 clinical trial for the treatment of choroideremia; 4D-710, which is in phase 1/2 clinical trial for the treatment cystic fibrosis lung disease; and 4D-310 to treat fabry disease cardiomyopathy and is in phase 1/2 clinical trial. In addition, its product candidates comprises 4D-175, which is in pre-clinical development for the treatment for geographic trophy; and 4D-725 to treat alpha-1 antitrypsin deficiency lung disease and is in pre-clinical development. The company has collaboration and licensing agreements with Arbor Biotechnologies, Inc.; Astellas Gene Therapies, Inc; uniQure biopharma B.V.; and Cystic Fibrosis Foundation. 4D Molecular Therapeutics, Inc. was founded in 2013 and is headquartered in Emeryville, California.

KALA BIO, Inc., a clinical-stage biopharmaceutical company, engages in the research, development, and commercialization of innovative therapies for rare and severe eye diseases in the United States. The company's product candidates include KPI-012, which is in Phase 2b clinical trial for the treatment of persistent corneal epithelial defects. Its preclinical development product, including KPI-014 for the treatment of rare inherited retinal diseases. The company was formerly known as Kala Pharmaceuticals, Inc. and changed its name to KALA BIO, Inc. in August 2023. KALA BIO, Inc. was incorporated in 2009 and is headquartered in Arlington, Massachusetts.

NeuBase Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company's proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington's disease (HD) and myotonic dystrophy, as well as other genetic disorders, and cancer and oncology applications. Its pipeline includes NT0100 for Huntington's disease; NT0200 for myotonic dystrophy type 1; and NT0300 treatment targets the mutated KRAS gene. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.