Seattle biotech company Immusofthas been granted approval to start clinical trials on its unique treatment for genetic diseases Immusoft announced on Thursday. Immusoft claims to be the first company to be granted approval to utilize the engineered cells of its immune system, also known as B cells in a study on humans.
The U.S. Food and Drug Administration (FDA) approved Immusoft’s Investigational New Drug Application for testing its immunotherapy against an extremely rare and fatal childhood disease known as MPS I.
An immune response is many players. The B cells are the ones responsible in the production of antibodies that adhere to invading viruses and bacteria. The company has the ability to transform B cells into “biofactories” that instead crank out inactive or malfunctioning proteins and enzymes within those cells that are found in patients.
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“This is a huge achievement for the company and a historic moment in the field of cell and gene therapies,” said Sean Ainsworth, Immusoft’s chief executive officer and chairman in an announcement.
This approach could have advantages over the current methods for providing treatments. Treatments that rely on the virus as their delivery method can create immune responses that hinder their effectiveness. Therapies that use stem cells could encounter difficulties with treatment with chemotherapy or stem cells transplants.
Immusoft’s ISP-001 technology, developed by Immusoft is based on a patient’s B cells to reprogramme them to produce proteins that are needed. Other companies that are working on B cells are Be Biopharma and Walking Fish Therapeutics.
“I don’t know if they are going to be successful, but it’s exciting for all of us that they have gotten permission to start a trial,” researcher Richard James told MIT Technology Review. James’s lab is located at The University of Washington is also conducting research on developing B-cells.
The study will take place by the University of Minnesota Medical School and will be conducted by Dr. Paul Orchard, a professor at the University’s Division of Pediatric Bone Marrow Transplantation.
Children suffering from MPS I are unable to make the enzyme that breaks the long-chain sugars in cells. The sugars are then stored within cells, leading to permanent damage. MPS1 with severe symptoms is found in approximately one in 100,000 births and symptoms begin to manifest within one year.
Immusoft is looking to expand its treatment options to other rare illnesses, along with central, autoimmune, and cardiovascular nervous system disorders.
In October 2021 it announced that it would be working with the pharmaceutical company Takeda to develop treatment options that target the nervous system, in an agreement that could be worth over $900 million.
Immusoft was established in the year 2009, and since then has since raised over $50 million of venture capital in accordance with PitchBook. In the year 2018 Ainsworth assumed the leadership role from the founder Matthew Scholz..
Scholz is now the CEO for Oisin Biotechnologies A startup that is developing therapeutics in preclinical development that kill and target the damaged “zombie” cells. He’s still on the Immusoft board of directors.
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