CRISPR gene therapy

This invention

This invention is a CRISPR-Cas9 gene editing platform for treating sickle cell disease by correcting the underlying genetic mutation directly in the body. It pairs the Cas9 editing machinery with adeno-associated virus (AAV) vectors for in-vivo delivery, and it uses tissue-specific promoters to focus editing where it's needed. The work sits at the meeting point of molecular biology, viral vector delivery, and the treatment of inherited blood disorders — squarely in therapeutic genome editing and gene therapy.

Where it fits

Your idea lives in a well-developed corner of Genetic Engineering (C12N), which shows up in all 50 of these results and runs about 48.6× more concentrated here than across the patent corpus as a whole. That tight clustering is exactly what you'd expect for therapeutic CRISPR. The work also touches Pharmaceutical Preparations (A61K) and Therapeutic Drug Activity (A61P). Filings have stayed steady across many years, with strong activity from 2017 onward — for example, 9 in 2020 and several each year through 2024. Several groups are active here, most prominently The Broad Institute, The Trustees of the University of Pennsylvania, Harvard College, and the University of Massachusetts. That's a sign this is a real, actively pursued direction. The field also connects to bioinformatics and computing tools, which these particular results touch less.

Closest related work

US-12161674-B2 — CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof (Vertex Pharmaceuticals · 0 citations · 15-member family; Vertex has 1 patent in this result set) (filed 2024, recent)

This patent covers treating severe sickle cell disease and β-thalassemia using CRISPR-Cas9–edited CD34+ hematopoietic stem and progenitor cells. It shows how Vertex approached the same disease target you're aiming at, focusing on the blood stem cell population that gives rise to red cells. It offers a useful window into editing applied to sickle cell biology, and it complements your in-vivo AAV-delivery angle.

US-2020392533-A1 — In vivo gene editing of blood progenitors (Harvard College · 5 citations · 2-member family)

This work modifies the genome of hematopoietic stem and progenitor cells in vivo by introducing an AAV that carries a sequence-targeting nuclease. It names sickle cell disease and β-thalassemia as targets. It comes strikingly close to your combination of AAV delivery plus in-vivo editing of blood progenitors, and it shows how Harvard framed the in-body delivery problem rather than the ex-vivo route.

US-2024216534-A1 — Direct rAAV-mediated in vivo gene editing of hematopoietic stem cells (University of Massachusetts · 0 citations · 3-member family) (filed 2024, recent)

This patent details an AAV expression construct with homology arms for in-vivo editing of hematopoietic stem cells. It shows how the University of Massachusetts engineered the AAV cassette itself — homology-directed repair templates flanked by AAV inverted terminal repeats. That speaks directly to the delivery and construct-design challenges at the heart of your platform.

US-2018110877-A1 — Dual AAV vector system for CRISPR/Cas9 mediated correction of human disease (University of Pennsylvania · 29 citations · 6-member family)

This describes a dual-AAV system that delivers Cas9, sgRNA, and donor templates to correct disease genes, including tissue-targeted (for example, hepatocyte) delivery. Because AAV has limited cargo capacity, splitting components across vectors is a recurring theme. It shows how Penn solved the packaging problem — relevant if your tissue-specific, in-vivo design runs into the same size constraints.

What you can do next

• Explore & build on it. Browse the related work above — new, differentiated ideas often come from combining or improving on existing approaches (for example, a specific tissue-specific promoter, a particular AAV serotype, a guide RNA design, or an editing strategy others haven't pinned down for sickle cell).
• If you'd like to protect it. Filing a provisional application (usually with a patent attorney) is a common first step. Most inventions can be protected in some form — what