NEW YORK – Immusoft on Friday said that the first patient with mucopolysaccharidosis type I (MPS I) has received its autologous engineered B-cell therapy ISP-001 in a Phase I trial.
MPS I is a rare genetic disease that impairs the ability to produce the enzyme alpha-L-iduronidase, which the body needs to break down long-chain sugars inside cells. When these sugar chains accumulate in cells, they damage tissues throughout the body, including the brain. In 1 in 100,000 births, MPS I manifests in a severe form in the first year of a child's life and can be lethal. Attenuated MPS I appears later in life and occurs in about 1 in 500,000 births.
ISP-001 is the first drug Immusoft has developed using its Immune System Programming platform. It involves reprogramming a patient's B cells so that they continue to produce alpha-L-iduronidase.
The first patient in the Phase I trial was dosed with ISP-001 at M Health Fairview University of Minnesota Medical Center. "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," Immusoft CEO Sean Ainsworth said in a statement.
San Francisco-based Immusoft said in a statement that this patient didn't need a preconditioning regimen, which is typically required for gene modified stem cell therapies and can cause adverse events, or immunosuppression, which is needed with systemic virus-delivered gene therapies and carries morbidity and infection risks.
The US Food and Drug Administration has granted orphan drug and rare pediatric disease designations for ISP-001 in MPS I.