Gene therapy for AADC deficiency
US12409235B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Nov 30, 2023 |
| Grant date | Sep 9, 2025 |
| Priority date | — |
| Expiry date | Dec 14, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y401/01028
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention is directed to compositions and methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×1011 vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×1011 vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×1011 vg/mL. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the met…
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.