ImmTOR in Gene Therapy
ImmTOR has significant potential to mitigate unwanted immune responses and induce AAV-specific tolerance to enable redosing of gene therapies. ImmTOR has the potential to administer multiple low doses to achieve therapeutic benefit without risk of overdosing, the ability to treat patients typically excluded from these treatments and is a novel approach to treating systemic diseases resulting from genetic disorders.
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TRADITIONAL GENE THERAPY
TRADITIONAL GENE THERAPY
Gene therapy has traditionally been conceptualized as a one-time, curative treatment option; however, research shows that there may be a need for subsequent doses years after initial treatment. In order to introduce a functional gene in patients, a vector is used to deliver the gene, most often derived from an adeno-associated virus (AAV).
THE CHALLENGE
While they do enable the efficacy of this powerful therapeutic approach, AAV vectors also present a key challenge in gene therapy. Neutralizing antibodies (NAbs) are formed in response to AAV vector administration, so redosing is not possible due to the potentially dangerous immune response that would follow a second or third administration of the gene therapy.
IMMTOR’S POTENTIAL
AAV vectors are non-replicating, so transgene expression is expected to wane over time, especially in children, most likely necessitating re-dosing. As many gene therapies in development are being investigated for the treatment of rare, often lethal, pediatric disorders, the inability to re-dose these therapies is of particular concern.
ImmTOR has the potential to induce AAV-specific immune tolerance, reducing the severity of the body’s immune response to AAV gene therapies and enabling repeat administrations.
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