Decibel Therapeutics
NASDAQ:DBTXDecibel Therapeutics, Inc., a clinical-stage biotechnology company, engages in discovering and developing transformative treatments for hearing and balance disorders. The company's product candidates and programs focuses on three areas, such as Gene Therapies for Congenital, Monogenic Hearing Loss designed to restore functional cells within the cochlea to address hearing disorders caused by single gene mutations; Gene Therapies for Hair Cell Regeneration designed to replace lost hair cells within the inner ear to address acquired hearing loss and balance disorders; and Otoprotection Therapeutic in clinical development to prevent hearing loss in cancer patients undergoing chemotherapy with cisplatin. Its lead gene therapy product candidate is DB-OTO to provide hearing to individuals born with profound hearing loss due to mutation of the otoferlin gene. The company is also developing DB-ATO and AAV.201, a gene therapy program designed to restore balance in patients with bilateral vestibulopathy by regenerating lost hair cells within the vestibule; DB-020 for the prevention of cisplatin-induced hearing loss, which is in Phase 1b clinical trial; AAV.103 to restore hearing in individuals with a gap junction beta-2 (GJB2) deficiency; AAV.104 to restore hearing in individuals with stereocilin (STRC) deficiency; and cochlear hair cell regeneration program, an AAV-based gene therapy that utilizes cell-selective expression of reprogramming factors to convert supporting cells into outer hair cells. Decibel Therapeutics, Inc. has a strategic collaboration with Regeneron Pharmaceuticals, Inc. to develop gene therapies for monogenic forms of congenital hearing loss. The company was formerly known as Hearing Inc. and changed its name to Decibel Therapeutics, Inc. in April 2014. Decibel Therapeutics, Inc. was incorporated in 2013 and is headquartered in Boston, Massachusetts. As of September 25, 2023, Decibel Therapeutics, Inc. operates as a subsidiary of Regeneron Pharmaceuticals, Inc.
Hepion Pharmaceuticals
NASDAQ:HEPAHepion Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of drug therapy treatment for chronic liver diseases in the United States. It develops Rencofilstat, a cyclophilin inhibitor that has completed Phase 2a clinical trials to target multiple pathologic pathways involved in the progression of liver disease; and is in phase 2 clinical development for the treatment of non-alcoholic steatohepatitis (NASH), as well as preclinical studies of Rencofilstat shows reduction in liver fibrosis, liver inflammation, liver tumor burden, and titers of HBV, HCV, HDV, and HIV-1, and has demonstrated antiviral activities for hepatitis B, C, and D viruses through several mechanisms. The company was formerly known as ContraVir Pharmaceuticals, Inc. and changed its name to Hepion Pharmaceuticals, Inc. in July 2019. Hepion Pharmaceuticals, Inc. was incorporated in 2013 and is headquartered in Edison, New Jersey.
4D pharma
NASDAQ:LBPS4D pharma plc, together with its subsidiaries, develops live biotherapeutic products (LBPs) in the United Kingdom. The company develops therapeutic candidates, including MRx0518 for the treatment of cancer and immune-oncology diseases; MRx-4DP0004 for the treatment of asthma; MRx0029 and MRx0005 the treatment of central nervous system disorders; Blautix for irritable bowel syndrome; and Thetanix for pediatric crohn's disease. It also develops products candidates, such as MRx1299 for solid tumors, MRx0006 for rheumatoid arthritis, and MRx0002 for multiple sclerosis. In addition, the company develops MicroRx platform to discover new LBP candidates for major diseases. 4D pharma plc has a collaboration agreement with Merck KGaA, Darmstadt, Germany and Pfizer to evaluate MRx0518; strategic collaboration agreement with University of Texas MD Anderson for discovery and development of LBPs in oncology; and Merck & Co., Inc. to conduct a clinical trial evaluating the combination of Keytruda, MRx0518 in patients with solid tumors, and to discover and develop LBPs for vaccines. The company was formerly known as Schosween 18 Limited. 4D pharma plc was incorporated in 2014 and is headquartered in Leeds, the United Kingdom.
Reneo Pharmaceuticals
NASDAQ:RPHMReneo Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases. The company's lead product candidate is REN001, a potent and selective agonist of the peroxisome proliferator-activated receptor delta that is in clinical development for genetic mitochondrial diseases comprising primary mitochondrial myopathies and long-chain fatty acid oxidation disorders. Reneo Pharmaceuticals, Inc. was incorporated in 2014 and is headquartered in Irvine, California.
Societal CDMO
NASDAQ:SCTLSocietal CDMO, Inc., a contract development and manufacturing organization, engages in the research and development, manufacturing, and packaging for various therapeutic dosage forms primarily in the small molecules in the United States and internationally. The company provides end-to-end services, such as formulation development focusing on complex formulations, reformulation, physical characterization, and excipient compatibility; analytical methods development which offers a range of analytical testing capabilities, including product testing, ICH stability, method development and validation, chromatography and spectroscopy equipment, stability chambers, and microbial testing; and pharmaceutical manufacturing that provides milling, blending, compression, spray and rotary granulation, particle and bead coating, encapsulation, liquids, lyophilization, and sterile fill and finish services. It also offers regulatory support, including handling communications with the food and drug administration (FDA) and seek consultation and guidance for client FDA meetings and responses; and pharmaceutical packaging and logistics, as well as smaller-scale primary and secondary packaging, labeling and kitting options suited for clinical trial materials and development packaging needs across dosage forms. The company was formerly known as Recro Pharma, Inc. and changed its name to Societal CDMO, Inc. in March 2022. Societal CDMO, Inc. was incorporated in 2007 and is based in Exton, Pennsylvania.