Immusoft of CA, a clinical-stage biotechnology company developing engineered B cell therapies, announced on Monday that it has received Rare Pediatric Disease (RPD) designation from the US Food and Drug Administration (FDA) for ISP-002, an investigational therapy for the treatment of mucopolysaccharidosis type II (MPS II).

MPS II, also known as Hunter syndrome, is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS).

ISP-002 is designed to deploy a patient's own B cells as long-lived protein biofactories capable of continuously producing and secreting therapeutic levels of IDS.

The FDA offers RPD designation for serious or life-threatening diseases that mainly impact children 18 years old or younger, and affect fewer than 200,000 people nationwide.

Sean Ainsworth, Immusoft CEO, said: "The FDA's Rare Pediatric Disease designation for ISP-002 recognises the serious unmet medical need in paediatric patients with MPS II and underscores the potential of Immusoft's platform as a novel therapeutic approach. This designation supports our continued efforts to advance engineered B cell therapies designed to deliver durable protein expression while maintaining a favourable safety and tolerability profile."