Seattle biotech company is the first to receive approval to test B cell gene therapy in humans – GeekWire

Immusofts steps to delivering a treatment using engineered B cells. (Screen grab from Immusofts website)

Seattle biotech startup Immusoft has received approval to begin clinical trials of its novel strategy for treating genetic disease, the company announced Thursday. Immusoft says its the first to get permission to use engineered immune system cells called B cells in a human study.

The U.S. Food and Drug Administration (FDA) approved Immusofts Investigational New Drug Application for testing its immunotherapy for a rare, lethal childhood disease called MPS I.

An immune response includes a suite of players, and the B cells are responsible for producing the antibodies that stick to invading bacteria and viruses. The company is able to modify B cells into biofactories that instead crank out missing or non-functioning enzymes and proteins in the cells of patients.

This is a huge achievement for the company and a historic moment in the field of cell and gene therapies, said Sean Ainsworth, Immusofts CEO and chairman, in a statement.

The approach has potential advantages to current strategies for delivering treatments. Therapies that use a virus as its delivery mechanism can trigger immune responses that limit their effectiveness. Treatments using stem cells can have difficulties associated with chemotherapy and stem cell transplants.

Immusofts technology, called ISP-001, uses a patients own B cells, reprogramming them to make needed proteins. Other companies working on B cell therapies include Be Biopharma and Walking Fish Therapeutics.

I dont know if they are going to be successful, but its exciting for all of us that they have gotten permission to start a trial, researcher Richard James told MIT Technology Review. Jamess lab at the University of Washington is also working on B cell engineering.

The trial will be done at the University of Minnesota Medical School and led by Dr. Paul Orchard, a professor in the universitys Division of Pediatric Bone Marrow Transplantation.

Children with MPS I are not able to produce an essential enzyme that helps break down long-chain sugars inside cells. The sugars then build up in cells, causing progressive damage. Severe MPS1 occurs in about 1 in 100,000 births, and symptoms appear within a year.

Immusoft is interested in expanding its therapy to other rare diseases, as well as cardiovascular, autoimmune and central nervous system diseases.

In October 2021, the company announced a collaboration with pharma giant Takeda to develop treatments targeted to the nervous system in a deal worth potentially more than $900 million.

Immusoft was founded in 2009 and has raised more than $50 million in venture capital, according to PitchBook. In 2018, Ainsworth took over leadership from founder Matthew Scholz.

Scholz is now CEO of Oisn Biotechnologies, a startup developing preclinical therapies that target and kill damaged zombie cells. He is still on Immusofts board of directors.

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Seattle biotech company is the first to receive approval to test B cell gene therapy in humans - GeekWire