Efforts to find treatment for rare condition selected for special help
The Rowett Institute’s quest to transform the lives of patients with a rare and debilitating medical condition is getting a fantastic helping hand.
Our research team seeking to develop a gene therapy to treat lipodystrophy has made some promising strides forward recently.
Now Dr George McIlroy, Prof Justin Rochford and colleagues have been selected to take part in a workshop run by Scottish Enterprise and Cell and Gene Therapy Catapult to help propel it to the next stage.
The session this spring will help them prepare a Target Product Profile (TPP), a key resource to keep innovative projects like this on track.
At present there is no cure for the debilitating condition – which reduces or removes the body’s ability to properly make crucial stores of fat.
This typically causes severe metabolic complications, including fatty liver disease and diabetes and can reduce life expectancy by more than 30 years. Treatment options remain very limited
Dr McIlroy, who leads the work, said: “Being selected to take part in Scottish Enterprise and Cell and Gene Therapy Catapult TPP workshop is an incredibly exciting opportunity. Developing a TPP is a crucial stage in shaping the future of our innovative gene therapy designed to address the underlying causes of lipodystrophy.
“Working with industry and clinical experts will help define the critical next steps in our pathway to develop a treatment with the potential to transform lives. To be part of a process that could lead to a life-changing treatment is both an honour and a responsibility. We are really excited to be advancing this research.
“Lipodystrophy is a rare and complicated condition, which is caused by fat tissue not working properly. However, the effects of this are felt throughout the body, leading to serious health complications. The groundbreaking approach of this gene therapy is to correct the root cause rather than just managing symptoms."
Prof Rochford added: “The ultimate goal is to create a therapy that restores healthy fat tissue function for people living with lipodystrophy giving them a long-lasting treatment with the exciting potential to dramatically improve their quality of life.”