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New deal could bring first treatment for rare terminal disorder in children

Scientists from The University of Manchester believe children suffering from Sanfilippo – a rare terminal disorder that affects children’s brains – could be treated successfully for the first time. There are currently four types of Sanfilippo syndrome, Type A, B, C and D, which are caused by a lack of enzymes, which helps to break down and recycle long chain sugars and results in a build-up of sugars in the body and particularly the brain.

Sanfilippo Type B affects around one in 200,000 live births, whereas the most common of the four types, Sanfilippo Type A affects around one in 100,000 live births in the UK.Affected children suffer from hyperactivity, speech loss, profound mental disability and eventually lose the ability to walk, talk or eat.

Professor Brian Bigger

Prof Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester, said a stem cell gene therapy technology developed in his laboratory and recently published in the prestigious journal Brain1 and selected for Editor’s choice, will be used by Orchard Therapeutics to treat Sanfilippo syndrome Type B after the company recently acquired an exclusive licence.

Prof Bigger said: “UK-based clinical-stage biotechnology company Orchard Therapeutics is already a trusted collaborative partner and the acquisition of the licence for Sanfilippo Type B complements the existing collaboration program already in place for Sanfilippo Type A.

“We are very hopeful that this will be transformative for patients, and that this technology will change the lives of children with Sanfilippo Type A and Type B.
“As it stands the disease is incurable and people with Sanfilippo syndrome normally live until adolescence or early adulthood after suffering for many years.
“Children with Sanfilippo begin showing symptoms of hyperactivity, severe behavioural problems and miss development milestones as toddlers. Later they show symptoms similar to dementia with most never achieving a developmental age beyond two years.”

Dr Jesus Garcia-Segovia, Orchard’s VP Clinical Development, CNS and Metabolic Disorders commented: “The incorporation of Sanfilippo syndrome type B into our development pipeline is a significant milestone in the consolidation of our neurometabolic franchise, which is currently focused on the development of autologous ex-vivo haematopoietic stem-cell gene therapy for children suffering from Sanfilippo syndrome type A. We are very excited at the possibility of bringing effective treatments capable of addressing the high unmet medical need in children suffering from these devastating conditions.”

Furthermore, Prof Bigger said the stem cell gene therapy, if successful, could also lead to major breakthroughs in treatment of more common diseases.
“If this is successful we could be in line for treating more common diseases such as Parkinson and Alzheimer diseases.
“If we can show that it is possible to treat single gene brain diseases such as Sanfilippo with stem cell gene therapy, it will pave the way for other treatments.
“We are also grateful to The University of Manchester’s technology transfer company, UMI3 Ltd, for their role in negotiating the terms of this major deal with Orchard Therapeutics.”

1 https://academic.oup.com/brain/article-lookup/doi/10.1093/brain/awx311
DOI 10.1093/brain/awx311

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