Praxis Precision Medicines, Inc.

NeumoraBiotechUnited States

72% match

Neumora Therapeutics is a clinical-stage biopharmaceutical company developing precision medicines for brain diseases, including neuropsychiatric and neurodegenerative disorders. It uses neuroscience, data science, and biomarkers to identify and advance treatments for patients with these conditions.

Stoke Therapeutics, Inc. is an emerging biopharmaceutical company dedicated to developing innovative antisense oligonucleotide (ASO) therapies. Its primary focus is to target and address the fundamental causes of severe genetic disorders, particularly within the United States. The company employs its exclusive Targeted Augmentation of Nuclear Gene Output (TANGO) platform to engineer ASOs that precisely enhance protein expression. Stoke's most advanced candidate, STK-001, is currently progressing through Phase I/IIa clinical trials for Dravet syndrome, a severe and debilitating genetic epilepsy. Additionally, STK-002 is in the preclinical development stage for treating autosomal dominant optic atrophy. The company has also established a licensing and collaborative agreement with Acadia Pharmaceuticals Inc. to identify, develop, and market new RNA-based medicines for critical and rare genetic neurodevelopmental conditions impacting the central nervous system. Formerly known as ASOthera Pharmaceuticals, Inc., the company rebranded to Stoke Therapeutics, Inc. in May 2016. Founded in 2014, Stoke Therapeutics, Inc. maintains its headquarters in Bedford, Massachusetts.

Alto Neuroscience, Inc. operates as a biopharmaceutical entity in the clinical stage, specializing in the creation of novel psychiatric medications. Its development pipeline includes ALTO-100, an investigational therapy targeting individuals affected by major depressive disorder (MDD) and post-traumatic stress disorder. Also in its portfolio is ALTO-300, a small molecule designed to function as both a melatonergic agonist and serotonergic antagonist, exhibiting antidepressant properties for MDD patients. For addressing the cognitive impairments linked with schizophrenia, Alto Neuroscience is advancing ALTO-101, an innovative small molecule phosphodiesterase 4 inhibitor. The company's therapeutic candidates further extend to ALTO-203, a pioneering small-molecule histamine H3 receptor inverse agonist intended for MDD patients experiencing heightened anhedonia, and ALTO-202, an orally available antagonist of the GluN2B subunit of the NMDA receptor, also under investigation for MDD. Beyond these individual drug candidates, the firm is engaged in developing novel drug combinations that demonstrate synergistic pharmacodynamic effects. Crucially, Alto Neuroscience leverages an advanced AI-enabled biomarker platform that synthesizes data concerning patients' brain activity and behavioral patterns. This platform's purpose is to accurately identify which individuals are most likely to respond positively to its cutting-edge product candidates. Alto Neuroscience, Inc., established in 2019, maintains its headquarters in Los Altos, California.

Passage Bio, Inc. is a biopharmaceutical company dedicated to pioneering genetic therapies for conditions affecting the central nervous system. Their advanced pipeline includes PBGM01, designed to treat infantile GM1 gangliosidosis by utilizing a proprietary AAVhu68 capsid to deliver a functional GLB1 gene, which codes for the lysosomal acid beta-galactosidase enzyme, to both the brain and peripheral tissues. Another key program, PBFT02, employs an AAV1 capsid to introduce a functional granulin (GRN) gene, encoding progranulin (PGRN), to the brain as a treatment for FTD-GRN. Additionally, PBKR03 is under development for infantile Krabbe disease, leveraging a proprietary AAVhu68 capsid to deliver a functional GALC gene, responsible for producing the hydrolytic enzyme galactosylceramidase, to the brain and surrounding tissues. The company's portfolio also features PBML04 for metachromatic leukodystrophy, PBAL05 for amyotrophic lateral sclerosis, and PBCM06 for Charcot-Marie-Tooth Type 2A. Passage Bio maintains a significant research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program, alongside a development services and clinical supply agreement with Catalent Maryland, Inc. Established in 2017, the company's headquarters are located in Philadelphia, Pennsylvania.

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

Precision BioSciences, Inc. is a clinical-stage biotechnology firm based in the United States, specializing in the creation of both in vivo gene editing solutions and ex vivo allogeneic CAR T-cell therapies. At its core is ARCUS, a proprietary genome editing platform designed to address and potentially cure genetic diseases. The company's portfolio also encompasses Ex vivo Allogeneic CAR T Immunotherapy, an innovative approach where specific immune cells, known as T-cells, are genetically modified outside the body to precisely identify and eliminate cancer cells. Among its prominent therapeutic candidates are: PBCAR0191, currently undergoing Phase 1/2a clinical trials for adult patients battling relapsed/refractory (R/R) non-Hodgkin lymphoma or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). PBCAR19B, an anti-CD19 CAR T candidate leveraging a "stealth cell" platform through a single-step gene edit, engineered to reduce the likelihood of chromosomal abnormalities. PBCAR269A, an investigational allogeneic CAR T immunotherapy formulated to target BCMA, intended for the treatment of R/R multiple myeloma. Precision BioSciences has strategically formed several collaborative alliances. These include an agreement with Les Laboratoires Servier for the development and commercialization of allogeneic chimeric antigen receptor T-cell therapies, aimed at various antigen targets, including hematological cancers beyond CD19, and solid tumors. The company is also partnering with Tiziana Life Sciences to assess foralumab, a fully human anti-CD3 monoclonal antibody, for its utility as a lymphodepleting agent in potential cancer treatments. Furthermore, a collaboration with iECURE, Inc. focuses on advancing ARCUS-based gene editing therapies. Established in 2006, Precision BioSciences, Inc. maintains its corporate headquarters in Durham, North Carolina.