4D Molecular Therapeutics
NASDAQ:FDMT4D 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.
Gracell Biotechnologies
NASDAQ:GRCLGracell Biotechnologies Inc., a clinical-stage biopharmaceutical company, primarily discovers and develops cell therapies for the treatment of cancer in the People's Republic of China. Its lead product candidates include GC012F, a FasTCAR-enabled dual BCMA- and CD19-directed autologous CAR-T product candidate that is in Phase I trial for the treatment of multiple myeloma; GC019F, a FasTCAR-enabled CD19-directed autologous CAR-T product candidate, which is in Phase I clinical trial for the treatment of adult B cell acute lymphoblastic leukemia (B-ALL), as well as in phase 1 clinical trial for the treatment of relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B-ALL) in adult; and GC027, a TruUCAR-enabled CD7-directed allogeneic CAR-T product candidate, which is in Phase I clinical trial for the treatment of adult T cell acute lymphoblastic leukemia. The company's product candidates also comprise GC007g, a donor-derived CD19-directed allogeneic CAR-T cell therapy that is in Phase 2 clinical trial for the treatment of r/r B-ALL; and GC502, a TruUCAR-enabled dual CD19- and CD7 -directed, off-the-shelf allogeneic CAR-T product candidate, which is in Phase I trial for the treatment of B-cell malignancies. In addition, it has a portfolio of earlier stage product candidates targeting various cancer indications, such as hematologic cell malignancies and solid tumors. Gracell Biotechnologies Inc. was incorporated in 2017 and is headquartered in Suzhou, China. As of February 22, 2024, Gracell Biotechnologies Inc. operates as a subsidiary of AstraZeneca Treasury Limited.
Pharming Group
NASDAQ:PHARPharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company's commercialized product is RUCONEST, a plasma-free rhC1INH protein replacement therapy for the treatment acute hereditary angioedema (HAE) attacks. It also engages in the development of leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome; OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE; and alpha-glucosidase therapy for the treatment of Pompe and fabry diseases. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. is headquartered in Leiden, the Netherlands.
Pharvaris
NASDAQ:PHVSPharvaris N.V., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for rare diseases. The company develops PHA121, a small molecule bradykinin B2-receptor antagonist that is in Phase II clinical trial for the treatment of hereditary angioedema (HAE). It also develops PHVS416, an on-demand, rapid exposure soft capsule for patients suffering from acute HAE attacks which is under Phase 2 clinical trial; and PHVS719, a prophylactic extended-release tablet for HAE patients which is under Phase 1 clinical trial. It operates in the Netherlands, Switzerland, and the United States. Pharvaris N.V. was incorporated in 2015 and is based in Leiden, the Netherlands.