Update on Gene Therapy
The Gauchers Association is pleased to provide an update on the progress of a potential Gene Therapy targeting treatment for all types of Gaucher disease being undertaken by Professor Simon Waddington and Dr Ahad Rahim. We are delighted to announce that Apollo Therapeutics teamed up with Professor Simon Waddington and Dr Ahad Rahim in late 2017 to progress the pre-clinical work previously undertaken and reported in the Gauchers News. The UK Gauchers Association continue to be a valued and active member of this team to bring a potential treatment to Gaucher patients.
Apollo Therapeutics provide committed translational funding and drug discovery expertise for novel therapeutics, sourced from the best of British academic research, accelerating them towards the clinic.
The Gauchers Association gave a small grant to both Professor Waddington and Dr Rahim back in 2009 to purchase some viral vectors for use in gene therapy research thanks to the family of Ellie Carter who died from Type 2 Gaucher disease.
The Association alongside Professor Waddington, Dr Rahim and Apollo Therapeutics will continue to keep our members updated on progress.