StemCell Therapy

Immusoft is heading into the clinic with what it claims is the first engineered B cell gene therapy cleared for in-human study, the company announced Thursday.

The Seattle-based company reported it has received clearance from the FDA to initiate a Phase I study assessing ISP-001 for the treatment of Mucopolysaccharidosis type I. MPS I is a rare lysosomal storage disorder characterized by an abnormal build-up of glycosaminoglycans in the body. Immusoft aims to improve treatment of MPS 1, also known as Hurler Syndrome, with its Immune System Programming platform that uses B cells as re-dosable biofactories for therapeutic protein delivery.

Immusoft stated its platform has been designed to circumvent both immunogenicity associated with virus-delivered gene therapy, as well as chemotherapy preconditioning associated with stem cell-mediated gene therapy. Additionally, the ISP platform has been designed to enable durable therapeutic delivery and the potential to re-dose patients if necessary.

The FDA has granted ISP-001 Orphan Drug designation and Rare Pediatric Disease designation for MPS 1. The Phase I study is expected to launch later this year, the company announced.

Sean Ainsworth, chairman and CEO of Immusoft, expressed excitement over the IND clearance. He called it a huge achievement for the company and a historic moment in the field of cell and gene therapies. Current adeno-associated virus therapies and lentiviral therapies have some safety concerns. The delivery mechanism in those therapies can trigger an immune response in patients, which can limit efficacy. Stem cell treatments also have some drawbacks in some patients.

Ainsworth told BioSpacethat the companys B cell approach has been designed to overcome some of the issues that were seen in other gene therapy modalities. Building on advances made in other cell therapeutic approaches such as CAR-T therapies, Ainsworth said the company is pioneering a gene therapy approach that allows for a durable therapeutic and the potential to re-dose patients.

Our engineered B cell approach is a completely novel modality that opens potential new avenues for treating intractable diseases. Having our lead program cleared to proceed into the clinic - the first B cell approach in the clinic - broadens the potential for our platform and pipeline as we advance other programs to first-in-human studies, Ainsworth said in an email this morning.

B Cell-Focused Competition

Immusoft isnt the only company focused on the development of B cell therapies. Others include Be Biopharma and Walking Fish Therapeutics.

Paul Orchard, a professor in the division of pediatric bone marrow transplantation at the University of Minnesota Medical Center, noted that a non-viral delivery method such as ISP-001 holds promise for the treatment of some diseases, such as MPS I. He said there is a clear need for new treatments for MPS I, as current options are not curative and significant disease-related morbidity still exists.

Although there is no cure for MPS I, there are several treatment options, including hematopoietic stem cell transplant and BioMarins Aldurazyme (laronidase), an enzyme replacement therapy. However, enzyme replacement therapies require frequent infusions, which affect quality of life. Immusoft hopes its therapeutic will mitigate the need for frequent dosing and improve patient outcomes.

Other companies developing therapies for MPS I include Orchard Therapeutics and REGENXBIO.

Beyond MPS I, Immusoft has partnered with the California Institute for Regenerative Medicine on the development of a therapy for MPS II, a carbohydrate metabolism disorder.

Read the rest here:
Immusoft Takes First-Ever Engineered B Cell Therapy into Clinic - BioSpace

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