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New Type A ERT Candidate on Track for Clinical Trial
Japanese company, JCR Pharmaceuticals, has received an orphan drug designation from the European Commission for their Sanfilippo type A drug candidate JR-441. This orphan designation will enable the further development of JR-441 in the European Union. The company has plans to prepare for a global clinical trial with JR-441 in patients with Sanfilippo type A to start in 2023.
JR-441 is an enzyme replacement therapy (ERT) utilising JCR’s J-Brain Cargo® BBB-penetrating technology. This form of ERT can cross the blood-brain barrier to deliver sulfamidase, the enzyme deficient in Sanfilippo type A, to where it is most needed.
In other ERT-based clinical trials for Sanfilippo, the enzyme has been unable to cross the blood-brain barrier if it is delivered into the bloodstream. Instead, the enzyme has been delivered via direct injection into the fluid surrounding the brain or the spinal canal to target the neurological aspects of the disease.
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