Enhanced Gene Therapy for Mucopolysaccharidosis Type I

A novel gene therapy system for treating Mucopolysaccharidosis Type I (MPS I), providing sustained release of iduronidase enzyme and improved efficacy through enhanced uptake by IDUA-deficient tissues.

The original patent (Method for the treatment of mucopolysaccharidosis type I) describes a method for treating MPS I using AAV vectors. However, this approach has limitations, including the need for repeated administrations and potential immune responses. The new inventive concept addresses these limitations by providing a gene therapy system that integrates a corrective copy of the iduronidase transgene into the albumin locus of hepatocytes in vivo, ensuring prolonged exposure of the enzyme to IDUA-deficient tissues.

The enhanced gene therapy system comprises a gene therapy vector that integrates a corrective copy of the iduronidase transgene into the albumin locus of hepatocytes in vivo. The system further includes a mechanism for sustained release of the iduronidase enzyme into the circulation, providing prolonged exposure of the enzyme to IDUA-deficient tissues. The gene therapy vector can be engineered to express a modified iduronidase enzyme with increased half-life and/or enhanced uptake by IDUA-deficient tissues. Additionally, the system can include a device for monitoring GAG levels in the subject, providing real-time feedback to adjust the gene therapy vector dosing and/or administration schedule.

The new claims introduce several novel features, including the integration of the iduronidase transgene into the albumin locus, the use of a sustained release mechanism, and the engineering of a modified iduronidase enzyme with improved properties. These features provide a significant improvement over the original patent, offering a more efficient, safer, and more effective treatment for MPS I.

Alternative embodiments of the inventive concept include the use of different gene therapy vectors, such as lentiviral or adenoviral vectors, or the incorporation of additional therapeutic components, such as small molecules or antibodies, to enhance the treatment's efficacy. Variations of the system can also include different mechanisms for sustained release, such as nanoparticles or implantable devices.

The enhanced gene therapy system for MPS I has significant commercial potential in the rare disease market, offering a potential treatment option for patients with this debilitating condition. The market for MPS I treatments is expected to grow significantly in the coming years, driven by the increasing awareness and diagnosis of the condition.