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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.

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..

Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company's lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.

Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.

Vaxart, Inc., a clinical-stage biotechnology company, discovers and develops oral recombinant protein vaccines based on its proprietary oral vaccine platform. The company's product pipeline includes norovirus vaccine, a bivalent oral tablet vaccine in Phase 2 clinical trial for the GI.1 and GII.4 norovirus strains; coronavirus vaccine, which is in Phase 2 clinical trial, for the treatment of SARS-CoV-2 infection; seasonal influenza vaccine, which is in Phase 2 clinical trial, to treat H1 influenza infection; and human papillomavirus therapeutic vaccine, which is in preclinical stage, that targets HPV-16 and HPV-18 for cervical cancers and precancerous cervical dysplasia. It has a license agreement with Altesa Biosciences, Inc. to develop and commercialize Vapendavir, a capsid-binding broad-spectrum antiviral. Vaxart, Inc. is headquartered in South San Francisco, California.