Atai Life Sciences
NASDAQ:ATAIATAI Life Sciences N.V., a clinical-stage biopharmaceutical company, engages in acquiring, incubating, and developing various therapeutics to treat depression, anxiety, addiction, and other mental health disorders. Its principal clinical programs include RL-007, a pro-cognitive neuromodulator for cognitive impairment associated with schizophrenia; GRX-917, a deuterated etifoxine for anxiety disorders; VLS-01 a N,N-Dimethyltryptamine for treatment resistant depression (TRD); DMX-1002, an oral formulation of ibogaine, a cholinergic, glutamatergic and monoaminergic receptor modulator that is a naturally occurring psychedelic product isolated from a West African shrub for the treatment of opioid use disorders (OUD); and EMP-01, an oral formulation of an MDMA derivative being developed for the treatment of post-traumatic stress disorder. The company's other clinical programs comprise PCN-101, a subcutaneous formulation of R-ketamine, as a therapy for psychiatric indications initially focused on TRD; and KUR-101, a formulation of deuterated mitragynine for the treatment of OUD. ATAI Life Sciences N.V. was founded in 2018 and is headquartered in Berlin, Germany with offices in New York and London.
Design Therapeutics
NASDAQ:DSGNDesign Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.
PMV Pharmaceuticals
NASDAQ:PMVPPMV Pharmaceuticals, Inc., a precision oncology company, engages in the discovery and development of small molecule and tumor-agnostic therapies for p53 mutations in cancer. It's lead product candidate is PC14586, a small molecule that corrects mutant p53 protein containing the Y220C mutation and restores wild-type p53 function. The company was formerly known as PJ Pharmaceuticals, Inc. and changed its name to PMV Pharmaceuticals, Inc. in July 2013. PMV Pharmaceuticals, Inc. was incorporated in 2013 and is based in Princeton, New Jersey.
uniQure
NASDAQ:QUREuniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Avidity Biosciences
NASDAQ:RNAAvidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.