Clinical-stage biotechnology company Immusoft of CA, a subsidiary of Immusoft Corporation, announced on Monday that it has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for ISP-002, its investigational engineered B cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), a rare and life-threatening lysosomal storage disorder.

ISP-002 uses Immusoft's proprietary engineered B cell platform, which programs a patient's own B cells to continuously produce therapeutic enzymes inside the body.

Immusoft said that clinical progress in mucopolysaccharidosis type I (MPS I) has provided validation of its engineered B cell platform. ISP-001, the company's lead investigational therapy, is the first engineered B cell therapy to be tested in humans and is currently being studied in an ongoing Phase 1/2 clinical trial. According to the company, early clinical experience has demonstrated a favourable safety and tolerability profile to date, including successful re-dosing without lymphodepletion, immunosuppression, or any pre-conditioning, supporting further development of the platform and its expansion into additional indications.

Sean Ainsworth, Immusoft chief executive officer, said: "Orphan Drug Designation for ISP-002 is an important milestone for our MPS II program and further validates the potential of our engineered B cell platform. This designation underscores our commitment to developing durable, re-dosable therapies that address the long-term needs of patients and families living with rare genetic diseases."