The Cell And Gene Therapy Catapult and University of Aberdeen Launch Islexa to Develop Novel Diabetes Technology

The Cell And Gene Therapy Catapult Release: New Company Created To Develop Novel Technology To Treat Diabetes

- UK technology has potential to bring islet transplant therapy to thousands of patients with type 1 diabetes

London and Aberdeen and Edinburgh, UK, May 18, 2016 – The Cell and Gene Therapy Catapult (CGT) and University of Aberdeen, UK, today announce the creation of Islexa, a new company developing a novel technology to produce laboratory grown islets, the organoids responsible for insulin production.

The technology could bring the option of an islet transplant to thousands more patients with type I diabetes. Currently in the UK, only 30-50 patients with hypoglycaemic unawareness can receive an islet transplant each year due to the low availability of suitable donor organs and the difficulty involved in extracting the islets.

Islexa technology works by reprogramming donated pancreatic tissue into fully functional islets which will significantly increase the number of patients who can receive the treatment.

An islet transplant can give patients effective, long term glucose control without the need of insulin administration.

Keith Thompson, CEO of the Cell and Gene Therapy Catapult and an Islexa director said:

“This is a really exciting technology that has the potential to bring life changing benefits to these diabetic patients. We are delighted to be forming Islexa with the partners we’ve worked with so far on this project. The collaboration has already delivered promising results and the formation of Islexa will accelerate the development of these lab grown islets and ultimately get this potential treatment to thousands of patients.”

Professor Kevin Docherty, University of Aberdeen said:

“The technology is based on converting pancreatic tissue into functional islets. This has an advantage over the use of stem cells as source material, since at the moment they generate only the insulin-producing beta cells. Islets are organoids that produce multiple hormones, including insulin, and donated islets are already effectively treating severe cases of type 1 diabetes. Having a hugely expanded supply of lab grown islets will enable us to significantly extend this established clinical treatment.”

The creation of Islexa follows successful results in pre-clinical studies on the technology and the company will hold future IP rights of the islet technology. The company will initially continue to focus on further pre-clinical development of the protocol for reprogramming the pancreas tissue into functional islets. The next stage is to take the technology into clinical trials in the next few years.

The expansion and reprogramming technology has been developed at the University of Aberdeen as part of activities led by a consortium with the support of the Cell and Gene Therapy Catapult. The consortium partners include University of Aberdeen, the Scottish Islet Transplant Programme, University of Edinburgh, and the Scottish National Blood Transfusion Service (SNBTS). The consortium partners bring unique expertise in clinical practice and manufacture, and will continue to work closely with Islexa during the development programme.

Mr John Casey, Consultant Transplant Surgeon at the Royal Infirmary of Edinburgh, and Lead Clinician for the Islet Transplantation Programme in Scotland said:

“Islet transplantation can transform the lives of patients with type I diabetes, and in some cases can result in long term freedom from insulin injections with excellent glucose control. This exciting collaboration between the Scottish Islet Programme, Aberdeen University and the Cell and Gene Therapy Catapult will allow us to rapidly develop the technology and treat more patients, more effectively.”

Notes to editors

Origin of the technology

Background information on hypoglycaemic type 1 diabetes

There are currently over 320,000 patients in the UK with type 1 diabetes and this number is predicted to rise in the UK to 652,200 by 2035 (Hex et al, 2012). (Diabetes UK, 2014). Worldwide, the total number of patients with diabetes is estimated at ~347 million (WHO, 2014), with ~21 million patients suffering from type 1 diabetes (~6 % of diabetic population).

Type 1 diabetes is normally treated by administration of insulin injections. Approximately 90% of all patients who receive insulin treatment have experienced hypoglycaemic episodes. Ten percent of T1D patients have impaired awareness of hypoglycaemia, and ~ 5% of patients experience > 7 serious hypoglycaemic events per year (corresponding to ~14,000 patients in the UK). Such episodes often require outside intervention, and can, in severe circumstances, require hospitalisation and may result in loss of consciousness and coma. There is thus a high unmet medical need for improved therapeutic strategies for this patient population.

Transplantation of islets isolated from donor pancreas to patients with type 1 diabetes have demonstrated improved hypoglycaemic awareness, decrease in severe episodes, and decreased requirement of insulin treatment.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established in 2012 as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 100 employees focusing on cell and gene therapy technologies, we work with our partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. We offer leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. We aim to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. Regenerative medicine is one of the UK government’s “eight great technologies” that support UK science strengths and business capabilities. The Cell and Gene Therapy Catapult works with Innovate UK. For more information go to ct.catapult.org.uk or visit www.gov.uk/innovate-uk.

About University of Aberdeen

Established in 1495, the University of Aberdeen is the fifth oldest in the UK and is consistently ranked in the top 1% of the world’s universities.

Renowned for its world-leading research in health, energy, food and nutrition and environmental and biological sciences, 76% of research was judged ‘world leading’ or ‘internationally excellent’ through the UK's Research Excellence Framework (REF 2014)

With a community of students and staff covering 120 nationalities, the University of Aberdeen is renowned for its international outlook, and in 2016 was ranked by Times Higher Education in the top 40 of the world’s 200 ‘most international universities’, making it the second highest Scottish university to be included in the rankings.

About SNBTS

The Scottish National Blood Transfusion Service (SNBTS) is the specialist provider of safe high quality blood, tissues and cell products and services in Scotland.

Our key priority is to ensure that NHSScotland has enough blood to meet the transfusion needs of patients in Scotland. It is our responsibility to make sure that blood, tissues and cells are available when patients need them.

SNBTS undertakes a portfolio of research and development projects designed to support the strategic and operational priorities of the organisation.

The Research, Development and Innovation department works in collaboration with Universities, other UK Blood Services and the wider NHSScotland and scientific research communities to ensure relevant and efficient use of research resources.

SNBTS carries out research and development in microbiological surveillance and safety, best clinical use of blood and blood components and the development of novel cellular therapeutics.

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