Bioengineered Immune Cells for Deadly Genetic Disease

Immusoft, a biotechnology company based in Seattle, has announced that the US Food and Drug Administration (FDA) “has agreed to let it move forward with the first study in humans of a new type of gene therapy, using genetically engineered B cells.” The are immune system cells that make antibodies, MIT Technology Review reports.

Immusoft plans to harness B cells to treat a rare inherited disease called MPS I. “I feel 100% confident in stating we are the first to get the green light to enter clinical trials,” said Sean Ainsworth, CEO of Immusoft, as reported by MIT Technology Review.

A press release issued by Immusoft explains that MPS I is a rare genetic disease that affects the body’s ability to produce an essential enzyme. It results in progressive damage to tissues and organs. Children with MPS I require frequent infusions, which impacts quality of life and is expensive.

The disease affects the body’s ability to break down long-chain sugars inside cells. When the sugar chains cannot be broken down and disposed of, they accumulate in the cells and cause progressive damage. This accumulation can happen in the tissues, including the brain.

Children affected by the most severe form of the disease rarely live longer than ten years after diagnosis. Severe MPS I occurs in about 1 in 100,000 births. And symptoms appear within a child’s first year of life.

In what is referred to as attenuated MPS I, symptoms appear later in childhood. It occurs in about 1 in 500,000 births.

Patients with the illness are currently treated with weekly infusions of the missing enzyme. But it’s not enough to cure the disease outright. Immusoft says it can engineer B cells to produce the enzyme instead.

“There is a clear need for additional therapies for MPS I, as current interventions are not curative and significant disease related morbidity still exists,” said Paul Orchard, a professor at University of Minnesota who will be the principal investigator on the study.

Immusoft’s ISP-001 (Immune System Programming 001) will be the first engineered B cell therapy to enter into human clinical trials. The ISP technology permits engineering B cells to create biofactories for in vivo therapeutic protein delivery. The concept is to engineer B cells so that they manufacture other proteins instead of antibodies.

In other words, ISP modifies a patient’s B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to persist for many years.

“The ISP platform is a first-in-class technology and a pioneering new approach to treat human disease,” said Robert Sikorski, Chief Medical Officer of Immusoft. “It has been rewarding to collaborate with patients, the FDA, investigators, and the Immusoft organization to achieve this important milestone and to launch the first-in-human clinical study.”

In the Immusoft press release, Ainsworth refers to the FDA green light as “a historic moment in the field of cell and gene therapies.”