PTC Therapeutics
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.
Top Stages
4 past transactions
Censa Pharmaceuticals
Acquisition in 2020
Censa Pharmaceuticals Inc. is a pharmaceutical company based in Wellesley, Massachusetts, established in 2015. The company specializes in the development and commercialization of therapeutics aimed at treating ultra-orphan central nervous system (CNS) disorders, particularly those related to neurotransmitter degradation and synthesis. Censa's pipeline includes innovative solutions for extremely rare metabolic diseases that affect the CNS, with a focus on enabling patients to recover from 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 advancing human health and addressing diseases. The company's proprietary drugs are designed to detect defects in mitochondrial proteins, which play a crucial role in cellular energy production and overall health. By targeting these defects, BioElectron Technology aims to provide new treatment options for mitochondrial diseases, which are often genetic disorders that can lead to severe health complications. Through its research and development efforts, the company seeks to offer novel solutions that could significantly improve patient outcomes in the realm of mitochondrial-related conditions.
Agilis Biotherapeutics
Acquisition in 2018
Agilis Biotherapeutics, LLC is a biotechnology company focused on developing DNA therapeutics for patients with rare central nervous system diseases caused by single-gene defects. The company employs innovative technology to precisely target and restore lost gene function while minimizing off-target effects. Agilis Biotherapeutics specializes in treatments for conditions such as Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, which leads to severe motor control issues and feeding difficulties, as well as Friedreich's Ataxia, a life-threatening neurodegenerative disease resulting from a defect in the FXN gene. Additionally, the company addresses Angelman Syndrome, a rare neurological disorder characterized by significant developmental delays. Founded in 2013 and based in Cambridge, Massachusetts, Agilis Biotherapeutics operates as a subsidiary of PTC Therapeutics, Inc. and has established a strategic partnership with Intrexon Corporation to enhance its therapeutic development efforts.
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