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Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.

Ironwood Pharmaceuticals, Inc., a healthcare company, focuses on the development and commercialization of gastrointestinal (GI) products. It markets linaclotide, a guanylate cyclase type-C (GC-C) agonist for the treatment of adults suffering from irritable bowel syndrome with constipation or chronic idiopathic constipation under the LINZESS name in the United States, Mexico, Japan, Saudi Arabia, and China, as well as under the CONSTELLA name in the Canada and European countries. The company is also developing IW-3300, a GC-C agonist for the treatment of visceral pain conditions, including interstitial cystitis/bladder pain syndrome and endometriosis; Apraglutide, a next-generation, long-acting synthetic peptide analog of glucagon-like peptide-2, as a differentiated therapeutic for rare diseases, including short bowel syndrome dependent on parenteral support and acute graft versus host disease; and CNP-104, an immune nanoparticle for the treatment of primary biliary cholangitis. The company has strategic partnerships with AbbVie Inc., AstraZeneca AB, and Astellas Pharma Inc. for the development and commercialization of linaclotide. The company was formerly known as Microbia, Inc. and changed its name to Ironwood Pharmaceuticals, Inc. in April 2008. Ironwood Pharmaceuticals, Inc. was incorporated in 1998 and is headquartered in Boston, Massachusetts.

Intra-Cellular Therapies, Inc., a biopharmaceutical company, focuses on the discovery, clinical development, and commercialization of small molecule drugs that address medical needs primarily in neuropsychiatric and neurological disorders by targeting intracellular signaling mechanisms in the central nervous system (CNS) in the United States. The company offers CAPLYTA for the treatment of schizophrenia and bipolar depression in adults. It is also involved in developing Lumateperone, which is in Phase 3 clinical trial for the treatment of various depressive disorders, as well as additional neuropsychiatric indications. In addition, the company is developing Lenrispodun (ITI-214) for the treatment of Parkinson's disease, CNS, and other disorders; ITI-1284 for the treatment of neuropsychiatric disorders and behavioral disturbances in dementia; and ITI-333 for substance use disorders, pain, and psychiatric comorbidities, including depression, anxiety, and sleep disorders. Intra-Cellular Therapies, Inc. was founded in 2002 and is headquartered in New York, New York.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

Zentalis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on discovering and developing small molecule therapeutics for the treatment of various cancers. Its products candidatures include the ZN-c3, an inhibitor of WEE1, a protein tyrosine kinase for the treatment of inhibitor for advanced solid tumors and hematological malignancies; in Phase 2 clinical trial as a monotherapy for the treatment of uterine serous carcinoma indications; Phase 2 clinical trial in Cyclin E1 driven high-grade serous ovarian cancer, fallopian tube, or primary peritoneal cancer; Phase 1/2 clinical trial in combination with PARPi for platinum-resistant ovarian cancer; Phase 1b clinical trial in combination with chemotherapy in platinum-resistant ovarian, and peritoneal or fallopian tube cancer; Phase 1/2 clinical trial in combination with chemotherapy in relapsed or refractory osteosarcoma; Phase 1/2 clinical trial with encorafenib and cetuximab for mutant metastatic colorectal cancer; and Phase 1/2 clinical trial in combination with chemotherapy in pancreatic cancer. The company's products pipeline also includes ZN-d5, an oral small molecule inhibitor of B-cell lymphoma 2, for hematological malignancies and related disorders; and BCL-xL heterobifunctional degrader, a member of the anti-apoptotic BCL-2 proteins, for solid tumors and hematological malignancies. Zentalis Pharmaceuticals, Inc. has licensing agreements and strategic collaborations with Recurium IP Holdings, LLC; Pfizer, Inc.; Zentera Therapeutics; Dana-Farber; and GlaxoSmithKline plc. The company was incorporated in 2014 and is headquartered in New York, New York.