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

GOSH Surgeon Paolo De Coppi makes first TIME100 Health List

Professor Paolo De Coppi has been added to TIME's inaugural TIME100 Health List of the 100 most influential people in global health this year

Two years of advancing personalised healthcare for children in collaboration with Roche

As we mark the milestone of our second year working together with Roche, we took some time to reflect on the incredible progress we have made in our journey towards advancing personalised healthcare for children.

New treatment for brain tumour approved after over 20 years of research

The first-ever targeted treatment for brain tumours in children has been approved for NHS patients, following decades of research by a Great Ormond Street consultant.

Help pioneer new treatments for millions of people this DNA Day

DNA Day is coming up this Thursday (25 April) and the team behind the DNA, Children + Young People’s Health Resource (D-CYPHR) are encouraging children and young people to contribute to important health research.