AVROBIO
NASDAQ:AVROAVROBIO, Inc., a clinical-stage gene therapy company, develops ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose worldwide. 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, as well as to treat Gaucher disease type 3; AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease; AVR-RD-04, which is in phase 1/2 clinical trial for the treatment of cystinosis; and AVR-RD-05 that is in preclinical stage for the treatment of Hunter syndrome. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Cabaletta Bio
NASDAQ:CABACabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The company's lead product candidate is DSG3-CAART, which is in Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, an autoimmune blistering skin disease. Its product candidate pipeline also includes MuSK-CAART, a preclinical stage product to treat a subset of patients with myasthenia gravis; CABA-201, a 4-1BB-containing CD19-CAR T investigational therapy, for the treatment of severe autoimmune diseases; PLA2R-CAART, a discovery stage product to treat patients with PLA2R-associated membranous nephropathy; and DSG3/1-CAART, a discovery stage product for the treatment of mucocutaneous pemphigus vulgaris. It has a collaboration with the University of Pennsylvania and the Children's Hospital of Philadelphia. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.
Cognition Therapeutics
NASDAQ:CGTXCognition Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of small molecule therapeutics targeting age-related degenerative diseases and disorders of the central nervous system and retina. Its lead product candidate is CT1812, an orally delivered molecule modulator designed to penetrate the blood-brain barrier and bind selectively to the S2R complex for the treatment of Alzheimer's disease. The company is also developing COG0201 SHINE, a randomized double-blind and placebo-controlled for mild-to-moderate AD and is in phase II clinical trial. Its product pipeline also includes CT2168 for Synucleinopathies; and CT2074 for dry age-related macular degeneration. The company was incorporated in 2007 and is headquartered in Purchase, New York.
Miromatrix Medical
NASDAQ:MIROMiromatrix Medical Inc., a life sciences company, develops a novel technology for bioengineering fully transplantable organs. The company's proprietary technology is a platform that uses a two-step method of decellularization and recellularization designed to remove the porcine cells from the organs obtained from pigs and replace them with unmodified human cells. It has collaborations with Baxter, CareDx, The Mayo Clinic, Mount Sinai Hospital, and The Texas Heart Institute. The company was formerly known as TayTech, Inc. Miromatrix Medical Inc. was incorporated in 2009 and is headquartered in Eden Prairie, Minnesota.
Quantum Genomics Société Anonyme
OTCMKTS:QNNTFQuantum Genomics Société Anonyme, a biopharmaceutical company, engages in the development of drugs to treat cardiovascular diseases. The company develops its drugs based on brain inhibition mechanism of Aminopeptidase A: BAPAI, a triple-action therapeutic platform primarily to treat high blood pressure and heart failure. It is developing firibastat, a treatment for high blood pressure, and in combination with others anti-hypertensive drugs. The company also is involved in the development of drugs for the treatment of high blood pressure as monotherapy and the prevention, as well as treatment of heart failure. Quantum Genomics Société Anonyme was incorporated in 2005 and is based in Paris, France.