Delivering trials for pioneering treatments

The CRF’s investment with commercial partners in 2019 enabled it to become a national referral site for cutting-edge trials. This led University Hospital Southampton (UHS) to invest in a dedicated new centre for emerging technologies and therapies.

Key facts
  • An innovative co-investment by the CRF, local R&D and commercial partners has enabled Southampton to offer patients access to Advanced Therapeutic Interventional Medicinal Products (ATIMPs)

  • These include a cutting-edge gene therapy trial for the blood clotting disorder haemophilia A, which could protect against potentially life-threatening bleeds without the need for regular injections

  • As a result, Southampton has become a national referral/dosing site for such studies.

  • This attracted investment by UHS for a new Southampton centre for Emerging Technologies and Therapies (SETT)

Delivering a potentially life-saving gene therapy trial

 

Despite the pandemic, Southampton continued to deliver vital trials for conditions other than COVID-19. These included a trial of a gene therapy treatment for the blood clotting disorder haemophilia A.

 

Haemophilia A is an inherited genetic disorder leading to a lack of the Factor VIII protein critical for blood clotting. This means even minor wounds or bruises can cause potentially life-threatening bleeds. Regular injections of synthetic Factor VIII, as often as every 48 hours, are currently the only effective way to manage it.

 

The trial tested a gene therapy, human cells are genetically engineered to produce Factor VIII. The patients then have surgery to insert them into their tummy. The cells continue to produce Factor VIII there, which enters their bloodstream and replaces the missing protein.

Establishing the resources, processes and facilities needed

 

Southampton’s ability to offer patients access to this gene therapy treatment and other cutting-edge treatments (known as ATIMPs) is due to an investment by our CRF, together with local R&D and commercial partners. 

 

This investment combined commercial revenue and pump-priming from a commercial partner. It enabled UHS to create a dedicated ATIMP pharmacist post. It also funded the processes and facilities needed to safely enable such treatments.

Southampton has become a national referral and dosing site for such studies as a result.

We shared our learning from this process across the UK’s CRF network. This adds to the UK’s ability to deliver gene therapy and other pioneering studies.

The new Southampton Centre for Emerging Technologies and Therapies

 

Southampton’s improved capability for pioneering ATIMP trials, together with a clear patient need, led UHS to invest in a new centre.

 

The Southampton Centre for Emerging Technologies and Therapies (SETT) aims to enhance and expand Southampton’s and the UK’s development of these trials.

 

It will draw on Southampton’s expertise, resources, and specialist processes to deliver these trials. The centre will be supported by a new CRF Associate Director for Emerging Therapies, Associate Professor Helena Lee.

 

Our CRF will be a key partner and contributor to research delivered through the SETT centre. Our researchers will use the same leadership, collaboration and resourcefulness already shown to bring pioneering new treatments to patients.