Remethylation Disorders Research Map
In 2021, HCU Network America undertook an initiative to identify the therapeutic approaches recently studied or under investigation for classical homocystinuria and characterize clinical and preclinical stage technologies in development. The Research Map aims to give a snapshot in time for the treatment approaches being studied by academia and industry covering pre-clinical and clinical stage technologies.
The Research Map was developed based on information available in the public domain and provided directly by academic and industry researchers. Interviews were held with Global Experts including leading researchers and clinicians to expand and validate the Research Map and to provide expert input on the Research Strategy.
Expert input included
- Sean Froese, MD, PhD - Principal Investigator; University of Children’s Hospital Zurich, Switzerland
- David Rosenblatt, MD - McGill University, Canada
- Manuel Schiff, MD - Clinician; Paris, France
- Shawn McCandless, MD - Clinical Geneticist; Colorado Children’s Hospital
- Peter Baker, MD - Clinical Geneticist/Metabolic; Colorado Children’s Hospital
- Nicola Longo, MD, PhD - Professor, and Chief of Medical Genetics/Pediatrics; University of Utah
Industry input included:
•Pfizer Pharmaceuticals*
Investigational Therapies Research Map
Our Research Map focuses on one of our key priorities: Advance New Treatment Modalities. The Research Map is not intended to be an exhaustive list of research activities around the world and does not include an analysis of existing and emerging tools for diagnosis and disease monitoring.
Click here for a description of Therapeutic Avenues further describing the different types of investigational therapies for remethylation disorders.
Click here to see a video update on these programs - view the Global Research Map: Remethylation Disorders presentation by Brittany Parke, HCU Network America Board Member and chair of Cobalamin Steering Committee
Note: the evaluation of each program is based on a compilation of input from expert interviewees and does not represent a consensus. Also, while the program timelines for industry programs and based on the latest information from each company’s website, future projections on timelines are not provided by industry and are based on standard industry assumptions and HCU NA’s estimates.
