Allogene Therapeutics
NASDAQ:ALLOAllogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. It develops, manufactures, and commercializes UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate for the treatment of pediatric and adult patients with R/R CD19 positive B-cell acute lymphoblastic leukemia (ALL). The company also develops cemacabtagene ansegedleucel, an engineered allogeneic CAR T cell product candidate that targets CD19 for the treatment of large B-cell lymphoma; and is in Phase 1b clinical trial for the treatment of chronic lymphocytic leukemia. In addition, it is developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-605, an allogeneic CAR T cell product candidate that is in a Phase I clinical trial for the treatment of multiple myeloma; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; ALLO-316, an allogeneic CAR T cell product candidate that is in Phase 1 clinical trial for the treatment of advanced or metastatic RCC; ALLO-329 for the treatment of certain autoimmune diseases; DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors; and Claudin 18.2 for the treatment of gastric and pancreatic cancer. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc. It also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of allogeneic CAR T cell product candidates; and a strategic partnership with Foresight Diagnostics to develop MRD-based In-Vitro Diagnostic for use in ALPHA3. The company was incorporated in 2017 and is headquartered in South San Francisco, California.
BioCryst Pharmaceuticals
NASDAQ:BCRXBioCryst 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.
Krystal Biotech
NASDAQ:KRYSKrystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.
Relay Therapeutics
NASDAQ:RLAYRelay Therapeutics, Inc. operates as a clinical-stage precision medicines company. It engages in transforming the drug discovery process with an initial focus on enhancing small molecule therapeutic discovery in targeted oncology and genetic disease indications. The company's lead product candidates include RLY-4008, an oral small molecule inhibitor of fibroblast growth factor receptor 2 (FGFR2), which is in a first-in-human clinical trial for patients with advanced or metastatic FGFR2-altered solid tumors; RLY-2608, a lead mutant-PI3Ka inhibitor program that targets phosphoinostide 3 kinase alpha; and Migoprotafib (GDC-1971), an oral, small molecule, potent and selective inhibitor of the protein tyrosine phosphatase SHP2 that binds and stabilizes Src homology region 2 domain-containing phosphatase-2 (SHP2) as a monotherapy in patients with advanced or metastatic solid tumors. In addition, it has collaboration and license agreements with D. E. Shaw Research, LLC to research certain biological targets through the use of D. E. Shaw Research computational modeling capabilities focused on analysis of protein motion to develop and commercialize compounds and products directed to such targets; and Genentech, Inc. for the development and commercialization of GDC-1971. The company was formerly known as Allostery, Inc. and changed its name to Relay Therapeutics, Inc. in December 2015. Relay Therapeutics, Inc. was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Twist Bioscience
NASDAQ:TWSTTwist Bioscience Corporation engages in the manufacture and sale of synthetic DNA-based products. The company offers synthetic genes and gene fragments used in product development for therapeutics, diagnostics, chemicals/materials, food/agriculture, data storage, and various applications within academic research by biotech, pharma, industrial chemical, and agricultural companies, as well as academic labs; Oligo pools used in targeted NGS, CRISPR gene editing, mutagenesis experiments, DNA origami, DNA computing, data storage in DNA, and other applications; and immunoglobulin G proteins for customers focused on the pursuit of drug discovery and development. It also provides NGS tools comprising library preparation kits, human exome kits, and fixed and custom panels, as well as alliance panels, primarily used within diagnostic tests for various indications, population genetics research and biomarker discovery, translational research, microbiology, and applied markets research; and methylation detection kits for cancer, and rare and inherited disease study, as well as fast hybridization and full RNA sequencing workflow solution. In addition, the company provides synthetic SARS-CoV-2 RNA reference sequences as positive controls for the development of both NGS and reverse transcription-polymerase chain reaction assays; synthetic monkeypox controls; and various respiratory viral controls, including for influenzas, respiratory syncytial virus, rhinoviruses, SARS, MERS, and coronaviruses. Further, it offers SARS-CoV-2 Research Panels, such as the Twist Respiratory Virus Panel and the Pan-Viral Research Panel, for the detection of disease in a research setting; precision DNA libraries used for antibody engineering, affinity maturation, and humanization; and antibody optimization solution to enable simultaneous optimization of multiple characteristics of a given antibody. The company was incorporated in 2013 and is headquartered in South San Francisco, California.