Immusoft’s mission is to develop a breakthrough autologous cell therapy platform for treating a variety of human diseases through our proprietary Immune System Programming (ISP™) technology. ISP™ technology can effectively re-program a patient’s own cells to become miniature drug factories in the body. The technology was designed to address current challenges faced with the production and delivery of conventional protein therapeutic drugs (biologics). The ISP™ platform enables safe insertion of genes encoding the correct human homolog of a missing or defective protein(s) into a patient’s immune cells using the Sleeping Beauty (SB) transposon system – a non-viral vector.
For decades, researchers have focused on gene therapy approaches to treat human diseases. Engineering viruses to insert or replace missing or defective genes with functional copies has been commonplace. However, engineered viruses require significant modification to efficiently infect target cell types, may be difficult to produce in the quantities needed for therapeutic use, most viruses’ have payload limitations, and are subject to immune clearance if seen as immunogenic (foreign to the body). Gene therapy involving viral transduction continues to be associated with real or perceived safety issues for therapeutic use in humans.
Immusoft’s ISP™ platform technology is hybrid cell/gene therapy approach, which uses a clinically validated, non-viral vector for safe, reliable insertion of functional genes into immune cells. Once administered back into the patient, a subset of ISP™ modified cells reside within survival niches in the body, continuously secreting gene-encoded protein(s). The platform’s broad utility to produce virtually any biologic drug entity, i.e. antibodies, proteins or enzymes, has the potential to disrupt the current standard of care for many diseases requiring recombinant enzyme replacement therapy, as well as, address orphan diseases with very small patient populations.
The development of the Immusoft ISP™ platform has been enabled by the licensing/acquisition of several key technology assets. First, Immusoft has acquired the exclusive rights to the ‘Sleeping Beauty’ transposon technology from Discovery Genomics and we have further optimized it for use with the ISP™ platform. This provides Immusoft with a highly efficient alternative to the current viral gene-delivery vehicles. In addition, we have an exclusive license option to core B-cell technology from the Baltimore lab at Caltech which provides a reliable methodology for the differentiation/amplification of modified B-cell-derived, plasma cell subsets expressing therapeutic proteins for introduction into the patient. We have filed two additional patent applications covering our extensive modifications to the technology.
Our lead indication is MPS-1 Mucopolysaccharidosis Type I (MPS-1) a lethal disease which results from the lack of a functional enzyme called α-L iduronidase (IDUA). In its most severe form, children die at the early age of 3 yrs. Otherwise known as Hurler Scheie-Syndrome, the disease requires frequent IDUA infusions, which are expensive and palliative at best. Long-term survival is difficult to achieve. A key 2015 Immusoft goal is the submission of an IND for the treatment of MPS-1 using re-programmed, patient-derived plasma cells to express high levels of IDUA. Achievement of this milestone will pave the way to the first clinical trial involving use of the ISP™ platform to achieve successful enzyme replacement therapy.
Successful validation of the ISP™ platform in human trials will position Immusoft to successfully build a pipeline of gene-based therapies to treat a number of serious diseases that currently have no effective therapeutic options. Our long term strategy is to advance internal programs for select indications and to work with strategic partners to find gene-based treatments for other serious diseases that represent areas of high medical need.