Pre-clinical development of a novel oral therapy for hyperinsulinemic hypoglycaemia
Lead Research Organisation:
University of Manchester
Department Name: UNLISTED
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.
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.
People |
ORCID iD |
Mark Dunne (Principal Investigator) |
Publications
Description | Million Dollar Bike Ride Fund |
Amount | £80,000 (GBP) |
Organisation | Million Dollar Bike Ride Fund |
Sector | Charity/Non Profit |
Country | United States |
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 |