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UK Pilot Project to Develop Antisense Oligonucleotides for Children With Ultra-Rare Brain Disorders

NEW YORK – Genomics England said on Wednesday that it is part of a pilot project that will explore the use of personalized therapies known as antisense oligonucleotides to treat children with ultra-rare and life-threatening brain disorders.

The pilot is part of a new program called the Rare Therapies Launch Pad that is supported by the UK government and aims to boost access to personalized rare disease therapies for children.

Financial details about the government funding for the program were not disclosed.

Developed by a consortium of UK groups including Genomics England, the Medicines and Healthcare products Regulatory Agency (MHRA), the Oxford-Harrington Rare Disease Centre, Mila’s Miracle Foundation, and the Association of the British Pharmaceutical Industry (ABPI), the pilot is meant to develop a sustainable and scalable approach for delivering individualized therapies for children living with a wide range of rare conditions, including establishing a regulatory pathway.

It will address diagnosis, therapy design and rapid manufacture, and treatment and aims to generate evidence for potential reimbursement for individualized therapies beyond the pilot.

"Developing medicines in small populations is challenging and is particularly so in those patients who have very rare conditions," Dan O'Connor, director of regulatory and early-access policy at ABPI, said in a statement. "That is why this opportunity is so exciting for clinicians, industry, and patients, as it will support the development of new treatment pathways, giving better access to long-needed therapies for patients with high unmet medical needs."