Investigational Therapies Research Map
In 2017, HCU Network America and HCU Network Australia 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 with support from a rare disease drug development consultant 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
•Viktor Kozich – Project Advisor - MD, Ph.D., General University Hospital in Prague
•Kimberley Chapman - MD, Ph.D., Children’s National, U.S.
•Bart deGeest – MD, Ph.D, Katholieke Universiteit Leuven, Belgium
•Henk Blom – Ph.D., Erasmus University Medical Center, Rotterdam, Netherlands
•Jan Kraus (deceased) - Ph.D., University of Colorado
•Warren Kruger - Ph.D., Fox Chase Cancer Center, US
•Andrew Morris - BM, BCh, Ph.D., Manchester University Hospitals NHS Trust, UK
•Bridget Wilcken – Children’s Hospital at Westmead, Sydney, Australia
•Ken Maclean*- Ph.D. - University of Colorado at Denver
•Johan Van Hove – MD, PhD, the University of Colorado at Denver
•Cynthia Freehauf* - Genetic Counselor, the University of Colorado at Denver
Industry input included:
•Aeglea Biotech*
•Erytech*
•Orphan Technologies*
*Provided input on specific program
Classical HCU 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 the map to view it in pdf form.
Click here for a description of Therapeutic Avenues further describing the different types of investigational therapies for Classical Homocystinuria.
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 are 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.
