Pre-clinical development of a novel oral therapy for hyperinsulinemic hypoglycaemia

Lead Research Organisation: University of Manchester

Abstract

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Technical Summary

Congenital Hyperinsulinism is a rare neonatal disease presenting severe hypoglycaemia associated with poor clinical outcomes and long-term neurodisability.
The support of the Technology Strategy Board will enable Heptares, in collaboration with the University of Manchester and Great Ormond Street Hospital, to carry out the pre-clinical development of a novel safe, effective and orally available treatment for Congenital Hyperinsulinsm. A successful outcome will provide a clinically ready molecule with anticipated additional benefits in other areas associated with intractable hypoglycaemia; including neonatal hypoglycaemia, hypoglycaemia occurring as a consequence of gastric bypass surgery and insulinoma-associated hypoglycaemia.

Publications

10 25 50
 
Description Million Dollar Bike Ride Fund
Amount £80,000 (GBP)
Organisation Million Dollar Bike Ride Fund 
Sector Charity/Non Profit
Country Unknown
Start 02/2016 
End 01/2017
 
Description Heptares 
Organisation Heptares Therapeutics Ltd
Country United Kingdom 
Sector Private 
PI Contribution Target validation of a potential therapeutic
Collaborator Contribution Development of novel therapeutic agents to support bioscience and translational research
Impact Abstract submitted to the Endocrine Society meeting in the USA for April 2016
Start Year 2015
 
Description International CHI Family Meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact 150 patents/parents and health care professions attended the international workshop, which sparked questions and discussion afterwards, and the CHI International charity reported increased interest in the disease and treatment.
Year(s) Of Engagement Activity 2015