PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.
Censa Pharmaceuticals Inc. is a pharmaceutical company that specializes in developing and commercializing therapeutics for ultra orphan central nervous system (CNS) disorders. Founded in 2015 and located in Wellesley, Massachusetts, the company focuses on treating extremely rare disorders related to neurotransmitter degradation and synthesis. Censa's pipeline includes innovative programs aimed at addressing orphan metabolic diseases, with the goal of helping patients overcome debilitating conditions. As of May 2020, Censa operates as a subsidiary of PTC Therapeutics, Inc.
ClearPoint Neuro
Post in 2020
ClearPoint Neuro, Inc., established in 1998 and headquartered in Irvine, California, operates as a medical device company specializing in minimally invasive neurosurgical procedures guided by direct magnetic resonance imaging (MRI). The company's primary product is the ClearPoint system, designed for precise insertion of catheters, electrodes, and laser fibers to treat neurological conditions such as deep-brain stimulation and biopsy. Additionally, ClearPoint Neuro has product candidates like the ClearTrace system for cardiac applications, aiming to expand its offerings in MRI-guided procedures. The company collaborates with renowned institutions like Johns Hopkins University and Mayo Clinic to develop innovative therapies and has a strategic focus on improving patient outcomes through precise neurological interventions.
BioElectron Technology
Acquisition in 2019
BioElectron Technology is a clinical-stage biotechnology company focused on developing innovative redox drugs aimed at addressing human health and disease. The company's research centers on identifying and treating mitochondrial diseases, which are genetic disorders linked to defects in mitochondrial proteins. By detecting these defects, BioElectron Technology seeks to provide healthcare professionals with new therapeutic options for managing mitochondrial-related conditions.
Agilis Biotherapeutics
Acquisition in 2018
Agilis Biotherapeutics, LLC is focused on developing DNA therapeutics for patients suffering from rare central nervous system disorders caused by single-gene defects. The company’s innovative technology allows for precise targeting and restoration of gene function while minimizing off-target effects. Agilis offers therapies for conditions such as AADC Deficiency, which impairs motor strength and control due to a deficiency in the enzyme aromatic L-amino acid decarboxylase, as well as Friedreich’s Ataxia, a life-shortening neurodegenerative disease linked to defects in the FXN gene. Additionally, it develops treatments for Angelman Syndrome, a neurological disorder that results in severe developmental delays. Established in 2013 and headquartered in Cambridge, Massachusetts, Agilis Biotherapeutics has formed a strategic partnership with Intrexon Corporation to enhance its therapeutic offerings.
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