Immunovant
NASDAQ:IMVTImmunovant, Inc., a clinical-stage biopharmaceutical company, develops monoclonal antibodies for the treatment of autoimmune diseases. It develops batoclimab, a novel fully human monoclonal antibody that target the neonatal fragment crystallizable receptor for the treatment of myasthenia gravis, thyroid eye disease, chronic inflammatory demyelinating polyneuropathy, and Graves diseases, as well as warm autoimmune hemolytic anemia. The company is headquartered in New York, New York. Immunovant, Inc. operates as a subsidiary of Roivant Sciences Ltd.
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.
Ocugen
NASDAQ:OCGNOcugen, Inc., a clinical-stage biopharmaceutical company, focuses on the developing gene therapies to cure blindness diseases. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, such as retinitis pigmentosa and leber congenital amaurosis; OCU410 and OCU410ST for the treatment of dry age-related macular degeneration (AMD); and OCU200, a novel fusion protein that is in preclinical development stage for the treatment of diabetic macular edema, diabetic retinopathy, and wet AMD. It has a strategic partnership with CanSino Biologics Inc. for gene therapy co-development and manufacturing; and Bharat Biotech for the commercialization of COVAXIN in the United States market. The company is headquartered in Malvern, Pennsylvania.
REGENXBIO
NASDAQ:RGNXREGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.
Telesis Bio
NASDAQ:TBIOTelesis Bio, Inc., a synthetic biology company, manufactures and sells synthetic biology instruments, reagents, and associated products and related services, primarily to pharmaceutical and academic laboratories worldwide. The company offers BioXp 3250 system and BioXp 9600 system, that empower researchers to go from a digital DNA sequence to endpoint-ready synthetic deoxyribonucleic acid (DNA) and messenger ribonucleic acid (mRNA) with onboard next generation sequencing (NGS) library preparation; BioXp portal, an online portal that offers an intuitive guided workflow and design tools for building new DNA sequences and assembling them into vectors of choice, as well as mRNA constructs; and BioXp De Novo kits which contains the necessary building blocks and reagents, including proprietary gibson assembly branded reagents, for specific synthetic biology workflow applications. It also provides BioXp Select kits which offers to use non-telesis bio DNA while using the BioXp system to perform synthetic biology workflow applications such as cloning, mRNA generation from plasmid and cell free amplification; BioXp Next Generation Sequencing kits which contains the necessary reagents to go from DNA or RNA to a sequencer-ready library; Benchtop reagents that contains the reagents necessary to proceed specific synthetic biology workflow on the benchtop using products generated on the BioXp system; and custom gibson short oligo ligation assembly enzymatic DNA synthesis solutions, designed reduce timelines for constructing synthetic DNA, RNA, and proteins for development of mRNA-based vaccines, diagnostics, therapeutics, and personalized medicines. The company serves its products to government institutions, contract research organizations, and synthetic biology companies. The company was formerly known as Codex DNA, Inc. and changed its name to Telesis Bio Inc. in November 2022. Telesis Bio, Inc. was incorporated in 2011 and is headquartered in San Diego, California.