Gene Editing Therapeutics: Base, Prime & CRISPR Enter the Clinic
Tracks the transition of gene-editing modalities from lab to clinical practice: base editing (CS-101 β-thalassaemia), prime editing, Casgevy and follow-on CRISPR-Cas9 therapeutics, in-vivo delivery platforms (lipid nanoparticles, RVG-exosomes), and the regulatory/payer response. Includes population-scale genomics discoveries that seed new editing targets. Distinct from cancer-immunotherapy (which houses CAR-T and neoantigen vaccines) — this campaign is the home for germline/somatic genome edits at the DNA level.
Tracked Metrics
Signals
Timeline
US and UK researchers engineer ThermoCas9 — a Cas9 variant from Geobacillus thermodenitrificans re-tuned so that editing activity is conditional on the DNA-methylation pattern typical of tumor cells.…
Landmark Science paper (Vol 392, Issue 6795, April 16, 2026; DOI 10.1126/science.adz8994) by an international team led by Holman et al. demonstrates that CRISPR-edited hematopoietic stem and…
Harvard molecular biologists publish a modified CRISPR/Cas9 system that inserts the XIST inactivation sequence into the extra chromosome 21 of Down syndrome cells, silencing the full chromosome…
Phase 1 trial (n=5) of CS-101, a "transformer" base editor targeting the BCL11A binding motif in the HBG1/HBG2 promoters to reactivate fetal haemoglobin. All five transfusion-dependent β-thalassaemia…
The US Patent and Trademark Appeal Board has once again confirmed that the Broad Institute — not Nobel laureates Jennifer Doudna and Emmanuelle Charpentier — holds the key patent rights for CRISPR…
Quantitative snapshot of gene-editing/cell-and-gene-therapy (CGT) ecosystem as captured in a March 30, 2026 systems-framework paper (Programmability and Persistence Score, DOI…
Journal of Clinical Investigation (Feb 2, 2026; article 196402): an adenine base editor (ABE) packaged in AAV and injected intracerebroventricularly on postnatal day 2 rescues seizures and sudden…
Journal of Hepatology (October 2025): world's first genetically modified pig-to-human auxiliary liver xenotransplantation in a living recipient — a 71-year-old patient with large hepatocellular…