Exciting news from our portfolio company Immusoft.
This week at the ASGCT 2026 in Boston, Immusoft is presenting engineered B-cell therapy clinical data from the world’s first engineered B-cell therapy ever tested in human patients.
Their lead therapy, ISP-001, targets MPS I, a rare genetic disease with no cure that affects roughly 1 in 100,000 children. In its most severe form, the disease is often fatal within the first decade of life.
Current treatment requires lifelong weekly enzyme infusions, where enzyme levels spike after each dose and drop before the next. Immusoft’s approach is different: reprogram the patient’s own immune cells to continuously produce the missing enzyme from inside the body. This engineered B-cell therapy approach
Engineered B-Cell Therapy Clinical Data Shows Progress in MPS I Treatment.
Key milestones to date include:
- Favorable safety and tolerability were observed across all patients in the trial
- First-ever successful re-dosing of an engineered B-cell therapy in a human patient, achieved in 2025, 18 months after the initial dose, with continued improvements in mobility and quality of life reported
- ISP-001 holds FDA Orphan Drug, Rare Pediatric Disease, and Fast Track designations
- Trial backed by the California Institute for Regenerative Medicine
- ISP-002, targeting MPS II (Hunter syndrome), received FDA Orphan Drug Designation in December 2025 and Rare Pediatric Disease Designation in January 2026
- Pipeline programs extend into Parkinson’s disease, Gaucher disease, and other indications
Congratulations to Sean Ainsworth and the entire Immusoft team on this remarkable progress in engineered B-cell therapy clinical development.
