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Codiak BioSciences, Inc., a clinical-stage biopharmaceutical company, focuses on developing exosome-based therapeutics for the treatment of oncology, neuro-oncology, neurology, neuromuscular diseases, infectious diseases, and other diseases. Its lead product candidates are exoSTING and exoIL-12 exosome therapeutic candidates for the treatment of various solid tumors. The company is also developing exoASO-STAT6, an antisense oligonucleotide targeting the STAT6 transcription factor; and exoVACC, a modular platform for constructing precision vaccines. It has a collaboration agreement with the Jazz Pharmaceuticals Ireland Limited, Sarepta Therapeutics, Inc., Kayla Therapeutics S.A.S., Washington University, Ragon Institute of MGH, MIT, and Harvard. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Precision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia. Its programs in clinical development include Phase I/II clinical stage programs in achromatopsia, X-linked retinitis pigmentosa, and RPE65-deficiency; Phase I clinical trials for radiation-induced xerostomia; and Parkinson's program that has completed a Phase II trial. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease. The company was incorporated in 2015 and is based in New York, New York.

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..

Osiris Therapeutics, Inc. researches, develops, manufactures, markets, and distributes regenerative medicine products in the United States. Its products include Grafix and Stravix for treating chronic wounds of diabetic foot ulcers, venous leg ulcers, pressure ulcers, arterial ulcers, and severe burns, as well as surgical and trauma wounds; BIO4 for bone repair and regeneration in spine, trauma, extremity, cranial, and foot and ankle surgeries; and Cartiform for treating articular cartilage lesions in the knee and other joints. The company also develops and markets Menvivo for the repair of meniscus; and TruSkin for treating chronic wounds. It markets and distributes its products directly to physicians, hospitals, and other healthcare providers, as well as through agents and distributors. Osiris Therapeutics, Inc. was founded in 1992 and is headquartered in Columbia, Maryland.