Cellectis S.A. American Depositary Shares
Cellectis S.A., a clinical stage biotechnological company, develops products based on gene-editing with a portfolio of allogeneic chimeric antigen receptor T-cells product candidates in the field of immuno-oncology and gene therapy product candidates in other therapeutic indications. The company is developing ALLO-501 to treat large B-cell lymphoma; ALLO-316, a gene-edited allogeneic CAR T-cell product targeting CD70; ALLO-819 to treat acute myeloid leukemia; ALLO-213 for the treatment of small cell lung cancer; and ALLO-182 to treat gastric and pancreatic cancer. It also develops UCART22 for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia; UCART123 to treat acute myeloid leukemia; UCART 20x22 for relapsed or refractory B-Cell non-hodgkin's lymphoma; and cema-cel to treat relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma. The company was founded in 1999 and is headquartered in Paris, France.
Open CLLS dashboard ↗Ultragenyx Pharmaceutical Inc.
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, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; 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; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial 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.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
Open RARE dashboard ↗