August 2025 · Dublin
In a feat of molecular engineering, a bespoke CRISPR therapy was rapidly developed and delivered for infant “KJ” with an ultra-rare metabolic condition—six months, design to dose. The New England Journal of Medicine covered this landmark intervention: N-of-1 medicine is officially real. Now if only clinical supply departments could keep up.
The future is here. The main bottleneck? Probably your paperwork.
Prime editing, CRISPR’s versatile cousin, arrived in early-phase human trials for chronic granulomatous disease. Enzyme function was restored in two-thirds of targeted immune cells—with no serious adverse events in initial follow-up. Peer behind the science in Nature.
Prime editing—because genomic precision doesn’t always require a jackhammer.
The UK’s NICE approved Casgevy® (exagamglogene autotemcel)—CRISPR’s reigning clinical champion—for severe sickle cell disease and transfusion-dependent beta thalassemia. Long-term data (BMJ): 28/29 SCD and 39/42 TDT patients reached event-free status at one year. Europe’s HTA just started sweating.
Sorry, stem cell therapy—there’s a new genetically engineered sheriff in town.
At the University of Minnesota, CRISPR-edited TILs (knocking out CISH) gave advanced GI cancer patients a shot at lasting remission—complete response in one, stable disease in others. Deep-dive the results at UMN.
Solid tumor teams finally get a CRISPR headline that's not just for the hematologists.
Regulators now reviewing CRISPR filings in record time—provided a two-year statistical plan and a large mug of espresso.
Clinical science optimism runs high, but manufacturing, cost, and host immune response are the new bottlenecks. Case in point: the sobering N-of-1 DMD trial illustrates both promise and evolving risk management.
Bottom line: Personalized therapies, scalable editing, regulatory approval—2025 is the year CRISPR shed its science fiction (and your finance team’s composure).
Science wins. Wallets and regulatory patience, not so much.