Lentiviral vectors (LVs) are integrative viral vectors, that can transfer genes into the patient’s DNA. LVs therefore offer the advantage of long-term expression of therapeutic genes and are particularly adapted to multiplying cells, where non-integrative approaches, such as adeno-associated vectors (AAVs), lead to suboptimal results. LVs however, can generate a strong immune response, which impairs both their efficacy and safety profile and limits their use in vivo.
Genespire has generated a new class of lentiviral vectors, the Immune Shielded Lentiviral Vectors (ISLVs), that are masked from the patient’s immune system. ISLVs are designed for direct use in the liver and provide for long term and stable hepatocyte-specific expression of the therapeutic gene.
Because of their intrinsic characteristics, ISLVs have broad applications and address the need for one-time treatments of diseases requiring life-long liver gene expression, such as inherited metabolic diseases and plasma protein deficiencies. With our proprietary ISLV platform, we aim at providing a unique and durable solution for people suffering from genetic diseases including for young patients.