Research & Development

Nemours Children’s Health, based in Delaware, US, has been chosen by the Foundation for the National Institutes of Health (FNIH) Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGCT) to conduct a first-of-its-kind gene therapy clinical trial for Morquio A syndrome.

Morquio A syndrome is a rare skeletal dysplasia caused by an inherited gene mutation. It affects one in 200,000 births and causes serious complications such as cervical spinal cord compression, short stature, flat feet, difficulty walking, tracheal obstruction, hearing loss and heart valvular disease.

FNIH AMP BGTC is a public-private partnership between the National Institutes of Health (NIH), US Food and Drug Administration (FDA), biopharmaceutical and life sciences companies and non-profit and other organisations, which has been created to help speed up the development and delivery of bespoke gene therapies. Currently, eight genetic diseases have been selected for clinical trials, where Nemours was selected for the Morquio A proposal, including being the clinical trial site.

For the rest of 2023, FINH and staff at Nemours will focus on refining the treatment protocol and hiring additional staff, with the aim to begin enrolment for the clinical trial in 2024.

Stuart Mackenzie MD, orthopaedic surgeon, director of the Skeletal Dysplasia Clinic, Nemours Children’s Health, Delaware, commented: “The ultimate goal of our work is to help our patients. With the knowledge we gain during this trial, we believe Nemours Children’s Health will be able to offer Morquio patients the newest and most innovative therapies available. Through this specialised public and private partnership with FNIH AMP, we are able to help realise our vision to create the healthiest generation of children.”