UK Grants Passport Designation to Orchard’s Sanfilippo Gene Therapy

Rare Daily Staff

Kyowa Kirin’s Orchard Therapeutics has received an Innovation Passport designation in the United Kingdom for its experimental gene therapy OTL-201, aimed at treating the rare and fatal disorder mucopolysaccharidosis type IIIA, the company said Wednesday.

The designation, granted under the U.K.’s Innovative Licensing and Access Pathway (ILAP), is designed to speed the development and potential rollout of promising medicines by enabling early and coordinated engagement with regulators, health technology assessment bodies and the National Health Service.

OTL-201 is one of three therapies initially selected under the refreshed ILAP framework launched in January 2025, which prioritizes treatments with the potential to address significant unmet medical needs.

Mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A, is a rare inherited disorder that leads to progressive neurodegeneration. Caused by mutations in the SGSH gene, the disease results in toxic buildup of heparan sulfate in the body, including the brain. Children with the condition typically experience a steady loss of cognitive and motor function and often do not survive beyond adolescence. There are currently no approved disease-modifying treatments.

OTL-201 is an ex vivo autologous hematopoietic stem cell gene therapy that works by inserting a functional copy of the SGSH gene into a patient’s own stem cells before reinfusion. The therapy is being evaluated in an ongoing early-stage clinical trial in the United Kingdom and has not yet been approved by regulators.

The ILAP program, first launched in January 2021 and refreshed in January 2025, offers drug developers a structured pathway to collaborate with the Medicines and Healthcare products Regulatory Agency, the National Institute for Health and Care Excellence and other stakeholders. The goal is to shorten development timelines and enable faster patient access to new therapies.