An inhibitor compound for treatment of fibrodysplasia ossificans progressiva.
Kenneth D. Bloch, M.D., William T. G. Morton Professor of Anesthesia
Harvard Medical School, Massachusetts General Hospital, Boston.
Fibrodysplasia ossificans progressiva is a rare inherited disorder where muscle and connective tissue such as tendons and ligaments are gradually replaced by bone. The compound under development has shown efficacy in a mouse disease model.
Novel therapy for treatment of creatine transporter deficiency.
Rick Hawkins, Chief Executive Officer
Lumos Pharma, Inc., Austin, Texas.
Creatine transporter deficiency occurs from a mutation in a creatine transporter gene that prevents the transport of sufficient levels of creatine to the brain and results in cognitive function disorder. The lead compound has been evaluated in mice with creatine transporter deficiency and resulted in improved brain metabolism and cognitive function.
A compound for treatment of neonatal herpes simplex virus.
David W. Kimberlin, M.D., Professor of Pediatrics
The University of Alabama at Birmingham.
Neonatal herpes simplex virus is an infection transmitted from mother to child during childbirth. In preliminary studies, the candidate compound that will be advanced by this group shows anti-herpetic activity and can penetrate the central nervous system. This compound has received longstanding development support by National Institute of Allergy and Infectious Diseases, including funding of the current clinical trial that will collaborate closely with TRND.
Development of a deuterium-modified compound for treatment of Schistosomiasis.
Julie F. Liu, Ph.D., Director, Research Management
CoNCERT Pharmaceuticals Inc., Lexington, Mass.
Schistosomiasis is a neglected tropical disease caused by parasitic Schistosoma worms th
|Contact: Geoffrey Spencer|
NIH/National Human Genome Research Institute