Seattle biotech startup immusoft announced on Thursday that it has been approved to start clinical trials of its new strategy for treating genetic diseases. Immusoft said that this is the first time to obtain permission to use engineered immune system cells called B cells in human research.
The US Food and Drug Administration (FDA) approved immusoft’s investigational new drug application to test its immunotherapy against a rare and fatal childhood disease called MPs I.
The immune response includes a group of participants, and B cells are responsible for producing antibodies that adhere to invading bacteria and viruses. The company is able to transform B cells into “biological factories” to produce missing or ineffective enzymes and proteins in patient cells.
Sean Ainsworth, chief executive officer and chairman of immusoft, said in a statement: “this is a great achievement of the company and a historic moment in the field of cell and gene therapy.”.
This approach has potential advantages for current strategies for providing treatment. Treatments using viruses as their delivery mechanism can trigger immune responses that limit their effectiveness. The treatment using stem cells may have difficulties related to chemotherapy and stem cell transplantation.
Immusoft’s technology is called isp-001, which uses the patient’s own B cells and reprogrammes them to make the required proteins. Other companies dedicated to B-cell therapy include biopharmaceuticals and walking fish therapy.
“I don’t know if they will succeed, but what excites us all is that they have obtained permission to start the experiment,” researcher Richard James told the MIT Technology Review. James’s laboratory at the University of Washington is also studying B-cell engineering.
The trial will be conducted at the University of Minnesota School of medicine, led by Dr. Paul ochard, Professor of pediatric bone marrow transplantation at the University.
Children with MPS I cannot produce an essential enzyme that helps break down intracellular long-chain sugars. Then, sugars accumulate in the cells, causing progressive damage. Severe Mps1 occurs in about 1 in 100000 newborns, and symptoms appear within one year.
Immusoft is interested in extending its treatment to other rare diseases, as well as cardiovascular, autoimmune and central nervous system diseases.
In October 2021, the company announced that it would cooperate with Takeda, a pharmaceutical giant, to develop treatments for the nervous system. This transaction may be worth more than $900 million.
According to pitchbook, immusoft was founded in 2009 and has raised more than $50 million in venture capital. In 2018, Ainsworth took over the leadership from founder Matthew Scholz.
Scholz is now the CEO of OIS í n biotechnologies, a startup that develops preclinical therapies with the goal of killing damaged “zombie” cells. He is still a board member of immusoft.