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Phase 2/3 Clinical Trial to Evaluate Potential COVID-19 Treatment

June 17, 2020

  1. Phase 2/3 Clinical Trial to Evaluate Potential COVID-19 Treatment

    The U.S. Food and Drug Administration has authorized the initiation of a Phase 2/3 trial to investigate PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, as a potential treatment for COVID-19. PTC299 is an oral investigational drug with a novel dual-mechanism of action that has the potential to address the two critical elements of COVID-19: High viral…

  2. Expanding Areas of Therapeutic Focus – Phenylketonuria (PKU)

    PTC has successfully completed acquisition of Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic rare diseases, including phenylketonuria (PKU) and other rare diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. “This acquisition adds a well-established late-stage PKU clinical…

  3. Expanding Gene Therapy Development in a State-of-the-Art Biologics Facility

    A long-term lease agreement with Bristol-Myers Squibb Company provides us more than 185,000 square feet of space, including an existing state-of-the-art biologics production facility and supporting research and operations buildings on the BMS Hopewell, New Jersey campus. We plan to further develop the biologics facility to support gene therapy production and foster innovation and employment…

  4. A Scientist’s Story

    PTC and Pfizer had formed a collaboration using PTC’s GEMS platform to target multiple oncology and cardiovascular targets. The research collaboration came to an end, but that doesn’t mean a scientist stops thinking about it. PTC scientist Anu Bhattacharyya, senior director, biology / nucleotide repeat disorders, worked on the collaboration and couldn’t stop thinking about…

  5. PTC Awards Grants for Global Duchenne Muscular Dystrophy Research

    In 2019, we launched PRIORITY, a global innovative research funding program to support pioneering clinical research initiatives that address the unmet medical needs of patients with the rare genetic disorder Duchenne muscular dystrophy. The goal is to advance the diagnosis and care of patients and support unique research programs that are focused on increasing the number…