Neuroinflammation, characterized by microglial cell-activation, plays a major role in the pathogenesis and progression of multiple neurodegenerative diseases, such as Lysosomal Storage Disorders and Alzheimer’s, severe human conditions currently lacking an effective treatment.
Ex vivo gene therapy based on genetically modified hematopoietic stem cells (HSCs) approach shows great promise, yet the beneficial effect of it relies on CNS-associated microglia-like cells derived from the transplanted HSCs due to their role in neuroinflammation reduction after treatment.
To increase the efficacy of such therapeutic strategy, we have developed a proprietary synthetic promoter for a gene transfer vector that allows for a controlled –low basal levels that are significantly increased upon inflammation– expression of therapeutic transgenes in immune-like cells.
The inducible promoter sequence is validated both in vitro and in vivo.


Lead Optimization/Preclinical




Download the brochure

  • A compound to mitigate the cytokine storm

    The body’s inflammatory response to fight infections and in tissue repair processes is crucial for normal functioning of the body. Yet, if not controlled, it can cause severe injury to the human body, as occurs during cytokine storm, a potentially[...]

  • Novel treatment to mitigate the Ectrodactyly-Ectodermal dysplasia- Clefting (EEC) associated blindness

    The Ectrodactyly-Ectodermal dysplasia-Clefting (EEC) syndrome is a rare genetic disorder due to mutations in the p63 gene and characterized by limb defects, orofacial clefting, ectodermal dysplasia, and ocular surface defects that lead to the loss of vision. Currently no specific[...]

  • A novel treatment for diabetic ulcers

    Diabetic chronic ulcers, a common complication of diabetes, are significant cause of morbidity and mortality in the Western world. Despite the considerable medical need, currently no specific and efficacious treatment for diabetic -and other chronic- ulcers exist. This is in[...]

Subscribe to newsletter

Follow the activities promoted by UniSMART Foundation