Compositions and methods for inhibiting expression of the ALAS1 gene

ALAS1-specific iRNA compositions provide a faster and safer treatment for acute hepatic porphyrias by inhibiting ALAS1 gene expression, addressing the limitations of current therapies and reducing the need for invasive treatments.

AU2023266354B2Pending Publication Date: 2026-07-16MT SINAI SCHOOL OF MEDICINE +1
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Patent Information

Authority / Receiving Office
AU · AU
Patent Type
Applications
Current Assignee / Owner
MT SINAI SCHOOL OF MEDICINE
Filing Date
2023-11-16
Publication Date
2026-07-16

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Abstract

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1. 20378020_1 (GHMatters) P102746.AU.2 20 23 26 63 54 16 N ov 2 02 3 A B S T R A C T 1 6 N o v 2 0 2 3 2 0 2 3 2 6 6 3 5 4
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