From CRISPR-Cas9 to prime editing, gene modification technologies are revolutionizing medicine. Correct genetic mutations, cure inherited diseases, and engineer next-generation cell therapies.
From cutting to writing - the evolution of genome engineering
The revolutionary molecular scissors that enabled precise DNA cutting. Nobel Prize-winning technology (2020) now powering approved therapies for sickle cell disease and beta thalassemia.
Single-nucleotide precision without double-strand breaks. Directly converts one base pair to another. Beam Therapeutics advancing multiple clinical programs for genetic blood disorders.
Search-and-replace DNA editing. Can make all 12 types of point mutations plus small insertions and deletions. Prime Medicine leading clinical development for this next-gen platform.
Lipid nanoparticles and AAV vectors delivering editors directly to tissues. Intellia's NTLA-2001 showed 87% transthyretin reduction with single IV dose in human trials.
Edit cells outside the body, then return them to patients. Powers CAR-T therapies and CASGEVY for sickle cell. Enables quality control before administration.
Regulate gene expression without changing DNA sequence. Reversible modifications using CRISPR-based activators and repressors. Chroma Medicine pioneering epigenetic therapies.
Companies advancing the precision medicine revolution
| Company | Technology | Lead Programs | Status |
|---|---|---|---|
| CRISPR Therapeutics | CRISPR-Cas9 | CASGEVY (sickle cell, beta-thal) | FDA Approved |
| Intellia Therapeutics | In Vivo CRISPR | NTLA-2001 (ATTR amyloidosis) | Phase III |
| Beam Therapeutics | Base Editing | BEAM-101 (sickle cell) | Phase I/II |
| Prime Medicine | Prime Editing | PM359 (chronic granulomatous) | Phase I |
| Editas Medicine | CRISPR-Cas12a | EDIT-301 (sickle cell) | Phase I/II |
| Verve Therapeutics | Base Editing | VERVE-101 (hypercholesterolemia) | Phase I |
| Chroma Medicine | Epigenome Editing | Gene silencing platform | Preclinical |
| Vertex Pharmaceuticals | CRISPR Partner | CASGEVY commercialization | Commercial |
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