Immusoft has dosed the first subject with its investigational engineered B cell therapy, ISP-001, in a human Phase I clinical trial of mucopolysaccharidosis type I (MPS I), a rare genetic disease.

The first patient was dosed at M Health Fairview University of Minnesota Medical Center in the US.

This patient was administered the investigational therapy without requiring a preconditioning treatment, which is needed for gene-modified stem cells or immunosuppression, required for systemic virus-delivered gene therapy. 

Preconditioning comprises myeloablative chemotherapy, which can lead to infertility, hair loss and nausea, as well as a long stay at the hospital. It could also cause life-threatening infections. 

Immunosuppression is linked to significant morbidity and can intensify infection risk. 

Immusoft’s new engineered B cell therapy does not require chemotherapy or immunosuppression thereby backing the redosability approach using engineered B cells.

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By GlobalData

Created using the Immune System Programming (ISP) platform of the company, ISP-001 has been granted orphan drug designation and rare paediatric disease designations from the FDA for MPS I.

Immusoft CEO Sean Ainsworth said: “This achievement marks an important milestone for patients with MPS I who have a significant need for additional therapeutic options. 

“This is the first patient in the world to receive an engineered B cell therapy – a major accomplishment for Immusoft and a major advancement in cell and gene therapy.”

MPS I impacts the ability of the body to create the enzyme alpha-L-iduronidase (IDUA), which is vital for disintegrating long-chain sugars inside cells. 

These sugars build up in the body without the IDUA enzyme, subsequently impacting eyes, heart, and bones among others.

Immusoft chief medical officer Robert Sikorski said: “I look forward to the next steps with this pioneering team of scientists and investigators as we advance the initial clinical trial in MPS I. 

“We are excited by the potential of expanding the Immusoft-engineered B cell platform to trials in additional therapeutic areas.”

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