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Aptose Biosciences Inc., a clinical-stage biotechnology company, discovers and develops personalized therapies addressing unmet medical needs in oncology in Canada. Its lead clinical program is APTO-253, which is a Phase I clinical trial for the treatment of patients with relapsed or refractory hematologic malignancies. The company has an agreement with CrystalGenomics, Inc. to research, develop, and commercialize CG026806, a non-covalent small molecule therapeutic agent, which is in preclinical stage for the treatment of acute myeloid leukemia and chronic lymphocytic leukemia/mantle cell lymphoma. The company was formerly known as Lorus Therapeutics Inc. and changed its name to Aptose Biosciences Inc. in August 2014. Aptose Biosciences Inc. was founded in 1986 and is headquartered in Mississauga, Canada.

Fennec Pharmaceuticals Inc., a biopharmaceutical company, develops product candidates for use in the treatment of cancer in the United States. Its lead product candidate is the Sodium Thiosulfate, which has completed the Phase III clinical trial for the prevention of cisplatin induced hearing loss or ototoxicity in children. The company was formerly known as Adherex Technologies Inc. and changed its name to Fennec Pharmaceuticals Inc. in September 2014. Fennec Pharmaceuticals Inc. was founded in 1996 and is based in Research Triangle Park, North Carolina.

Orchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..

Renovacor, Inc., a biotechnology company, focuses on delivering various precision therapies to enhance the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. It primarily focuses on the treatment of BCL2-associated athanogene 3 (BAG3) mutation-associated dilated cardiomyopathy (DCM). The company's lead product candidate is REN-001, a recombinant adeno-associated virus 9-based gene therapy designed to deliver a functional BAG3 gene to augment BAG3 protein levels in cardiomyocytes, and slow or halt progression of BAG3 DCM. It is also developing a pipeline of BAG3-associated gene therapies for diseases with high unmet medical need associated with mutations in the BAG3 gene and mechanistically linked to BAG3's expression and function. The company was founded in 2013 and is based in Greenwich, Connecticut.

Voyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.