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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.

CASI Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes therapeutics and pharmaceutical products in the People's Republic of China, the United States, and internationally. It offers EVOMELA, an intravenous formulation of melphalan for use as a conditioning treatment prior to stem cell transplantation, and as a palliative treatment for patients with multiple myeloma. The company's other hematology/oncology assets in pipeline include CNCT 19, an autologous CD19 CAR-T investigative product; BI-1206, novel anti-Fc?RIIB antibody, for the treatment of solid tumors and relapsed/refractory non-hodgkin lymphoma; CB-5339, a novel VCP/p97 inhibitor that focuses on valosin-containing protein (VCP)/p97 as a novel target in protein homeostasis, DNA damage response, and other cellular stress pathways for therapeutic use in the treatment of patients with various malignancies; and CID-103, a human IgG1 anti-CD38 monoclonal antibody. The company has licensing agreements with Juventas Biotechnology (Tianjin) Co., Ltd.; BioInvent International AB; Black Belt Therapeutics Limited; Cleave Therapeutics, Inc.; and Acrotech Biopharma L.L.C. to develop and commercialize its commercial product EVOMELA. It also has distribution agreements with China Resources Pharmaceutical Commercial Group International Trading Co., Ltd; Pharmathen Global BV; and Riemser Pharma GmbH. CASI Pharmaceuticals, Inc. was incorporated in 1991 and is based in Beijing, the People's Republic of China.

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.

Harpoon Therapeutics, Inc., a clinical-stage immunotherapy company, engages in the development of a novel class of T cell engagers that harness the power of the body's immune system to treat patients suffering from cancer and other diseases in the United States. The company develops tri-specific T cell activating construct (TriTAC) product candidate, including HPN328, which is in Phase I/II clinical trials for the treatment of small cell lung cancer and other Delta-like canonical Notch ligand 3-expressing tumors; and HPN217 that is in Phase I clinical trials for the treatment of multiple myeloma. Its preclinical stage product is HPN601 for the treatment of multiple solid tumor indications. It has a discovery collaboration and license agreement with AbbVie Biotechnology Ltd. to develop and commercialize products that incorporate its proprietary TriTAC platform technology together with soluble T cell receptors. The company was incorporated in 2015 and is headquartered in South San Francisco, California. As of March 11, 2024, Harpoon Therapeutics, Inc. operates as a subsidiary of Merck Sharp & Dohme LLC.

TCR2 Therapeutics, Inc. is a clinical-stage cell therapy company, which engages in the development of biological drugs and engineering T-cells for cancer therapy. It is also involved in the research and collaboration with academic laboratories and industry partners in the field of T-cell immunology, cell therapy, gene editing, and process development. The company was founded by Patrick A. Baeuerle on May 29, 2015 and is headquartered in Cambridge, MA.