ISP-001 was developed using Immusoft’s proprietary platform ISP™ (Immune System Programming). To manufacture the cell product, Immusoft also uses technology related to the Sleeping Beauty transposon system, engineered by scientists at the Max Delbrück Center (MDC). Ascenion, the MDC’s technology transfer partner, has supported the license negotiations for the use of the technology between the MDC and Immusoft.
The ISP™ approach enables the reprogramming of a patient’s B cells to constantly produce therapeutic proteins. The resulting therapy does not require preconditioning such as chemotherapy or immunosuppression, both having considerable side effects, giving it a potential advantage over gene-modified stem cells and viral vector-based delivery. ISP-001 has received orphan drug designation and rare paediatric disease designations from the US Food and Drug Administration (FDA) for MPS I treatment.
MPS I is a rare genetic disease and 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. Severe MPS I occurs in about 1 in 100,000 births.
Immusoft from Seattle, WA is a clinical-stage cell therapy company focused on developing novel therapies for rare diseases using sustained delivery of protein therapeutics from the patient’s own cells.
Immusoft administers engineered B cells in a clinical trial for the first time
The US company 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).
