New commercial agreement for gene therapy at GOSH

16 Mar 2023, 9:57 a.m.

Staff working at cell and Gene therapy clean room at the ZCR

Great Ormond Street Hospital for Children (GOSH) and Leucid Bio have entered a new commercial agreement to manufacture a gene therapy product. 

Gene and cell therapies are the cutting-edge in clinical research for cancer treatments. They hold promise to offer personalised cancer treatments, including for rare forms that sadly affect children and young people. This year, GOSH opened new facilities for manufacturing gene and cell therapies. These facilities have increased our capacity to support development of cell and gene therapies from the very start in discovery research to clinical trials where therapies are tested in patients.

A new agreement with GOSH and Leucid Bio

In a new agreement, GOSH will work with Leucid Bio, a biotechnology company, at the state-of-the-art cleanroom facilities at the Zayed Centre for Research into Rare Disease in Children and provide quality assurance services required for manufacture of their gene therapy product, called LEU011.

LEU011 is a type of CAR T-cell therapy which reprogrammes these immune cells to recognise and destroy cancer cells. The company’s therapy has a particular structure on the cell surface that has been shown through early-stage research to work more effectively at targeting cancers than other types of CAR T-cell therapies.

Leucid Bio will submit for clinical trial approvals to trial LEU011, a type of gene therapy product over the next few months. In this first trial for patients, they hope to show that LEU011 improves outcomes and can save the lives of patients with solid tumours and blood cancers.

At Great Ormond Street Hospital, we see children with the most rare and complex cancers. There have been incredible strides forward in the treatment that we can offer, however, sadly for many these still don’t work. Gene therapies offer hope for better, and potentially curative treatments which is why we’re excited to support innovation in gene therapies through access to these highly specialist and unique facilities.“

Stephen Mathew, Head of Innovation at GOSH

We are extremely pleased to enter this first of its kind collaboration with GOSH to manufacture our lead asset, LEU011, for use in human studies. Additionally, we are pleased to have started a process with GOSH to examine the feasibility of applying our approach in the paediatric setting where there remains a great unmet need for new therapies.”

Ian Miscampbell, Interim Executive Chairman of Leucid Bio

More information about CAR T-cell therapy

Creating communities and lasting involvement

At GOSH, patient and public involvement and engagement (PPIE) builds lasting relationships and communities that shape research over time.

Building trust in data and research

At GOSH, patients and young people are helping researchers understand not just what data can do, but how it should (and shouldn’t) be used, explained and protected.

Celebrating 25 years of saving children’s lives with gene therapy

Marking 25 years since delivering the first ground-breaking gene therapy here at GOSH, having now treated more than 135 children with rare and life-threatening conditions across the world.

A catalyst to unlock the next generation of research leaders

The NIHR GOSH Biomedical Research Centre (BRC) Fellowships are transforming early-career researchers into independent leaders - driving innovation, attracting millions in funding and shaping the future of paediatric science.