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Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.

Bavarian Nordic A/S develops, manufactures, and commercializes life-saving vaccines. The company offers non-replicating smallpox and monkeypox vaccines under the IMVAMUNE, IMVANEX, and JYNNEOS names; rabies vaccine for human use under the Rabipur/RabAvert name; tick-borne encephalitis vaccine under the Encepur name; and Ebola vaccine under the MVABEA name. It is also developing MVA-BN for the treatment of smallpox; MVA-BN RSV, which is in Phase III clinical trials for the treatment of respiratory syncytial virus; ABNCoV2 that has completed Phase III clinical trial for the treatment of SARS-CoV-2. The company has license and collaboration agreement with AdaptVac; and license agreements with National Cancer Institute and Public Health Service. It operates in the United States, Canada, France, Germany, Spain, Australia, Switzerland, Sweden, Chile, Taiwan, the United Kingdom, Hong Kong, Saudi Arabia, Belgium, and internationally. Bavarian Nordic A/S was incorporated in 1992 and is headquartered in Hellerup, Denmark.

Krystal 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.

Nightstar Therapeutics plc, a clinical-stage gene therapy company, focuses on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases in the United Kingdom. The company's lead product candidate is NSR-REP1, a candidate that is in phase 3 clinical development for the treatment of patients with choroideremia. It is also developing NSR-RPGR, a candidate that is in Phase 1/2 clinical trials for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease; and NSR-BEST1, a candidate that is in preclinical development stage for the treatment of best vitelliform macular dystrophy. In addition, the company is developing NSR-ABCA4, a candidate that is in preclinical development stage for the treatment of Stargardt disease. The company was founded in 2013 and is headquartered in London, the United Kingdom.

Vericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in North America. The company markets autologous cell therapy products comprising MACI, an autologous cultured chondrocytes on porcine collagen membrane for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns; and NexoBrid, a biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, Massachusetts.