Seattle-based biotech startup, Immusoft received the green light to start clinical trials to treat MPS I, a rare, lethal childhood disease. This approval makes it the first company to be permitted to use engineered immune system cells (B cells) in a human study.
The company’s technology called ISP-001 uses a patient’s B cells and reprograms them to make missing or non-functioning enzymes and proteins. The approach has the potential to advance current strategies for delivering treatments and mitigate difficulties associated with therapies that use viruses and stem cells.
“This is a huge achievement for the company and a historic moment in the field of cell and gene therapies,” said Sean Ainsworth, Immusoft’s CEO and chairman.