PAH-modifying compositions and methods
Patent Information
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- TESSERA THERAPEUTICS INC
- Filing Date
- 2025-12-05
- Publication Date
- 2026-07-23
AI Technical Summary
Current methods for integrating longer sequences into a genome are inefficient and lack site specificity, and there is a need for improved compositions and methods to modify the PAH gene effectively to treat Phenylketonuria (PKU), as existing treatments like CRISPR/Cas9 and Cre/loxP are limited in efficacy and require multiple steps.
A gene modifying system comprising a template RNA with specific gRNA spacers, scaffolds, and a heterologous object sequence, along with a gene modifying polypeptide, is used to introduce targeted modifications to the PAH gene, including insertion, alteration, or deletion, utilizing a reverse transcriptase domain to alter genomic sequences.
The system enables precise and effective modification of the PAH gene, potentially correcting pathogenic mutations and treating PKU by administering a gene modifying system, such as a lipid nanoparticle formulation, to introduce mutations or correct mutations in the PAH gene.
Smart Images
Figure US2025058494_23072026_PF_FP_ABST