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AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Innate Pharma S.A., a biotechnology company, develops immunotherapies for cancer patients in France and internationally. The company's products include Lacutamab (IPH4102), an anti-KIR3DL2 antibody, which is in Phase II clinical trials for the treatment of cutaneous T-cell and peripheral T-cell lymphoma, as well as in Phase II clinical trials to treat refractory sézary syndrome; Monalizumab, an immune checkpoint inhibitor that is in Phase III clinical trial to treat advanced solid tumors comprising colorectal and lung cancer, as well as head and neck cancer; IPH5201, a blocking antibody that is in Phase II clinical trials targeting the CD39 immunosuppressive pathway; IPH5301, an anti-CD73 antibody targeting the immunosuppressive adenosine pathway to promote antitumor immunity; IPH6401, an BCMA-targeting NK cell engager; and IPH6101, a NKp46-based NK cell engager that targets CD123 proprietary multi-specific antibody format; and IPH62, an B7-H3-targeting NK cell engager. Its products in preclinical trials are IPH43, an anti-MICA/B ADC and IPH45, an antibody drug conjugates (ADC); and IPH67, an undisclosed, multi-specific ANKET. The company has licensing and collaboration agreements with AstraZeneca, Novo Nordisk A/S, Sanofi, and Orega Biotech; and co-development and license agreement with MedImmune Limited. Innate Pharma S.A. was incorporated in 1999 and is headquartered in Marseille, France.

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..

Proteon Therapeutics, Inc., a late-stage biopharmaceutical company, focuses on the development of pharmaceuticals to address the needs of patients with renal and vascular disease. Its lead product candidate is vonapanitase, an investigational drug intended to enhance hemodialysis vascular access outcomes. The company develops vonapanitase, a recombinant human elastase, which has completed Phase II and Phase III clinical trials for patients with chronic kidney disease. It is also evaluating vonapanitase in a Phase I clinical trial in patients with peripheral artery disease. The company was founded in 2001 and is based in Waltham, Massachusetts.