RARE Daily

Great Ormond Street Hospital Devises Plan to Expand Access to Gene Therapies

April 9, 2024

Rare Daily Staff

Great Ormond Street Hospital is piloting a new approach to expand access to gene therapies for children living with a rare disease.

The hospital said that due to small patient populations for many rare diseases, gene therapies to treat them are often not commercially viable for companies to invest in and keep on the market, even if the treatment itself can cure a condition.

Great Ormond Street Hospital (GOSH), with support from GOSH Charity and the British medical charity LifeArc, said under its plan, a National Health Service Trust would hold the market authorization for a gene therapy so that patients can directly benefit.

GOSH will now explore if the hospital could take on the licenses for these sorts of treatment to make them accessible to children at GOSH and in other hospitals via collaborative NHS agreements.

The new approach will be piloted with a lentiviral gene therapy for ADA-SCID that was co-developed between GOSH and UCL Great Ormond Street Institute of Child Health in the United Kingdom and UCLA in the United States.

ADA-Severe Combined Immuno-deficiency (ADA-SCID) is a rare condition that means the immune system doesn’t develop. It affects up to three children every year and leaves them very vulnerable, meaning even minor infections can become life-threatening. Children with ADA-SCID have a faulty gene that is responsible for the production of an enzyme, ADA, which plays a key role in helping cells in the immune system grow and divide. The gene therapy works by replacing the faulty gene with one that works, allowing the production of ADA.

“For rare and ultra-rare diseases, it’s a sad fact that there is little commercial reason for companies to develop costly treatments with very small markets. This means that treatments that have been shown to work through research are not getting to patients who could benefit from them. It’s simply not good enough that we have treatments that we know work, but we can’t get them to our patients,” said Claire Booth, professor in gene therapy and pediatric immunology at UCL Great Ormond Street Institute of Child Health and GOSH. “We must do something radical to tackle this. We are exploring what we can do in a place like GOSH, where we have the academic facilities to manufacture treatments combined with a rich history of working with other NHS hospitals, in research and clinical care.”

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