Science

Immusoft’s Approach

Immusoft is developing a cutting-edge approach to sustained delivery of protein therapeutics using a patient’s own cells. By leveraging our Immune System Programming (ISP™) platform, we collect a type of the patient’s immune cells, called B cells. In response to immune stimulation, B cells can turn into a biofactory state known as a plasma cell. Plasma cells manufacture and secrete thousands of antibodies per second. We harness the plasma cell’s biofactory capabilities by engineering B cells to produce a specific therapeutic protein.

Once we have engineered the B cells, we expand and differentiate them into plasma cells (or, stated simply, coax them into the biofactory state) that produce massive amounts of our therapeutic protein. Thereafter, we infuse the programmed cells back into the same patient, where they take up residence and produce therapeutic proteins for extended periods of time.

Collect

Through a procedure known as apheresis, we harvest a patient’s own B
cells, which serve as the essential cellular foundation for our programming
approach.

select and purify

As precursors to plasma cells, B cells serve as the ideal
cellular foundation for our approach due to their robust protein-producing
capabilities. We select and purify these specific cells to initiate the programming
and differentiation process.

Program

This is the gene transfer step. Using a non-viral gene delivery system,
B cells are programmed with DNA to allow them to produce large amounts of
therapeutic protein.

expand and differentiate

Using a proprietary cell culture method, the programmed B cells are expanded and differentiated into plasma cells and
plasmablasts — “biofactory” cells optimized for production and secretion of the therapeutic protein.

Infuse

Programmed biofactory cells are injected back into the patient. As with the blood collection in the first step, this step uses standard, conventional infusion processes. The programmed cells circulate in the body and take up long term residence in the tissues of the body, resulting in sustained in vivo cell-based drug delivery into the patient’s blood stream.

At WOrk

Once engrafted, the programmed cells produce therapeutic protein around the clock, for extended periods (possibly years), to potentially bring meaningful functional improvement to the patient.

What are the advantages of B Cells?

B cells offer distinctive advantages over other systemic (delivered in the bloodstream) gene therapy / protein delivery approaches. Other gene therapy approaches predominantly entail either injection of viruses that encode the therapeutic gene, or modification of stem cells that are transferred back to the patient.

In the case of virus delivery, there is currently no way to re-deliver the treatment. So, for a successful virus gene therapy, the following are required:

  • The dose has to be right the first time
  • The effect must not diminish over time
  • As the patient grows, the amount of protein must increase to maintain a stable protein per Kg ratio
  • The benefits must outweigh the risks

The field of viral gene therapy has unfortunately encountered several serious safety hurdles in recent years.

Because our approach is non-viral and uses the patient’s own cells, Immusoft is able to give patients additional doses (which has been demonstrated clinically) to overcome the above issues.


In the case of stem cell-mediated gene delivery, patients must undergo extensive myeloablation (chemotherapy) to knock out the patient’s immune system to make room for the newly modified stem cells. Myeloablation can be a highly toxic and unpleasant procedure. It usually requires hospitalization and is sometimes fatal. Immusoft’s approach does not require myeloablation and therefore avoids the associated hospitalization and adverse events.

The other approach we believe we will improve upon is direct infusion of proteins, with Enzyme Replacement Therapies (ERTs) being an initial target. Enzyme replacement therapies require weekly infusions that can take up to four hours at a time. Aside from the substantial disruption in the patient’s and caregivers’ lives, these enzymes do not last long, and we believe patients would benefit from a sustained, steady-state delivery over extended periods.

B cells also offer the advantage of being able to produce a multitude of protein types. At Immusoft, we have successfully encoded B cells to produce enzymes, antibodies, structural proteins, and signaling proteins. Each of these protein types represents major opportunities to improve upon existing modalities or develop entirely new ones, potentially improving the lives of patients in need of better therapies.

Research

See the latest publications and presentations from Immusoft in the News section.

If you are interested in becoming a collaborator or sponsor investigator, please submit your proposal to researchproposals@immusoft.com.