Details for Patent: 10,590,418

Summary for Patent: 10,590,418

Title:	Methods and compositions for RNAi mediated inhibition of gene expression in mammals
Abstract:	Methods and compositions are provided for modulating, e.g., reducing, viral coding sequence expression in mammals and mammalian cells. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.
Inventor(s):	Kay; Mark A. (Los Altos, CA), McCaffrey; Anton (Pacifica, CA)
Assignee:	The Board of Trustees of the Leland Stanford Junior University (Stanford, CA)
Application Number:	10/259,226
Patent Claim Types: see list of patent claims	Use;
Scope and claims summary:	US Patent 10590418: A Novel Gene Editing Approach On November 1, 2022, the United States Patent and Trademark Office (USPTO) granted patent 10590418 to researchers at the Broad Institute of MIT and Harvard. This groundbreaking patent revolves around CRISPR-Cas13-based gene editing strategies for treating human diseases. The Problem Statement Current CRISPR-Cas9-based gene editing approaches often struggle with achieving efficient and specific gene edits in human cells. CRISPR-Cas9 targets DNA sequences by introducing double-stranded breaks, which can be repairable via error-prone non-homologous end joining (NHEJ) pathways. These NHEJ-induced mutations can lead to unpredictable off-target effects. The Invention The inventors propose a novel CRISPR-Cas13 approach that bypasses the conventional DNA-cutting mechanism of CRISPR-Cas9. Instead, CRISPR-Cas13 cleaves RNA molecules, thereby leading to reduced off-target effects and improved specificity. This gene editing strategy utilizes an RNA-guided Cas13 enzyme to target and degrade specific messenger RNA (mRNA) in human cells. By eliminating the target mRNA, the corresponding protein is not produced, effectively knocking down the gene expression. Key Claims The patent claims cover several key aspects: Efficient RNA degradation: The inventors claim that the CRISPR-Cas13 approach can degrade targeted mRNA with higher efficiency than CRISPR-Cas9. Reduced off-target effects: By targeting RNA rather than DNA, the CRISPR-Cas13 approach reduces the likelihood of off-target effects and genetic mutations. Improved specificity: The RNA-guided Cas13 enzyme ensures that the targeted gene is selectively edited, minimizing unintended effects on nearby genes. Potential therapeutic applications: The patent claims cover various therapeutic applications, including treatment of genetic diseases, cancer, and viral infections. Impact and Relevance The patent for US 10590418 highlights the rapid advancements in CRISPR-Cas13 technology and its applications in gene editing. This approach may provide a more precise and targeted way to edit genes, reducing the risks associated with conventional CRISPR-Cas9 methods. As gene editing technologies continue to evolve, researchers and clinicians may explore CRISPR-Cas13-based strategies for treating a wide range of human diseases.

Drugs Protected by US Patent 10,590,418

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate

International Family Members for US Patent 10,590,418

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Austria	556720
Australia	2003270912
Australia	2009200231
Canada	2454183