The staggering progress of CRISPR-Based Therapies: An Act in Three Scenes
- Pietro Gatti
- May 15
- 4 min read
TL;DR
CRISPR leapt from its first genome-snip in human cells (2012) to the first FDA-approved CRISPR therapy, Casgevy (2023), and—within two years—to an IV infusion (CTX310) that rewrites DNA inside the body (2025). This blistering timeline shows medicine crossing a threshold: when editing a genome becomes as routine as administering an IV, healthcare stops being reactive and starts becoming programmable—moving chronic disease from lifelong management to a one- or few-shot correction.
Understanding how such technologies redraw the map of healthcare—and the ripple effects on regulation, reimbursement, and competitive strategy—is essential groundwork for anticipating tomorrow’s markets. It also underpins how we guide business, innovation, and society toward the opportunities ahead.
Scene 1 – A nineteen-year-old named DJ
Early this year, Deshawn “DJ” Chow rolled his wheelchair into City of Hope’s transplant unit clutching a stuffed penguin and a consent form thicker than a Tolstoy novel. Hours later his blood stem-cells were whisked away, snipped with CRISPR scissors, blasted with chemo, and finally dripped back into his veins. The goal: silence the sickle-cell mutation that had sabotaged his childhood. The FDA had approved the therapy—Casgevy—just weeks before, lauding it as the first CRISPR-based medicine on the planet. But for DJ it still felt like space travel: thrilling, dangerous, and astronomically expensive. (WIRED, U.S. Food and Drug Administration)
“The process of getting this drug is very different from just taking a pill", says his haematologist
Casgevy proved regulators would endorse gene-editing when the benefit is undeniable. It also exposed the moon-landing nature of first-gen CRISPR: patients must check into hospital for weeks, endure myeloablative chemo, and hope their edited cells thrive. The science was dazzling; the user experience, lagging behind.
Scene 2 – The conference call that stole the headlines
Fast-forward to 6 May 2025. On a quarterly earnings call, CRISPR Therapeutics’ CEO Samarth Kulkarni dropped a slide that jolted every cardiologist on the line: in the Phase 1 study of CTX310, a single lipid-nanoparticle infusion chopped LDL-cholesterol and triglycerides by about 80 %. No bone-marrow harvest, no chemo—just an IV bag and an hour of Netflix (GlobeNewswire, ir.crisprtx.com).
For the first time, CRISPR edits were happening inside living livers, not in petri dishes flown back into patients later. The star of the show wasn’t a new nuclease—it was the delivery vehicle: a lipid bubble engineered to dodge Kupffer cells, punch through an endosome, and unbox its genetic cargo exactly where ANGPTL3 genes idle.
Suddenly CRISPR no longer resembled bespoke cell surgery; it looked like a quick jab any infusion suite could handle. Portfolio models lit up accordingly—no longer capped at a few-thousand rare-disease patients, the target pool expanded to the tens of millions who swallow statins every morning.
Scene 3 – Prime time for precision
Even as Kulkarni’s slide deck went viral, another story was brewing in Cambridge, MA. Prime Medicine had just received the FDA’s blessing to dose humans with PM359, the world’s first prime-editing therapy. Prime editing doesn’t smash DNA and hope for tidy repairs; it rewrites letters with molecular White-Out, sidestepping double-strand breaks and much of the off-target angst. First patients will receive PM359 later this summer (investors.primemedicine.com, GEN).
If CTX310 showed CRISPR can reach the liver, PM359 hints it can do so with surgical elegance—opening the kidney, the brain, maybe even the heart to redosable “software patches”. The lipid-nanoparticle chassis perfected for Cas9 can slot a prime editor without redesign—same hardware, new payload—underscoring how platform assets, not one-off prototypes, end up owning the game.
The thread that binds the scenes
Casgevy (2023) settled the existential safety debate.
CTX310 (2025) proved delivery is king—move the edit to the bedside and everything changes.
PM359 (H2 2025) promises finesse, expanding CRISPR’s reach from heroic one-off cures to scalable chronic-disease franchises.
What looks like break-neck technical progress is, at heart, a very human story: a teenager willing to gamble on untested science; an engineer obsessed with nanoparticle geometry; a post-doc who wondered, “What if we just rewrite the typo?” Creativity, intuition, and stubborn optimism—all the un-patentable traits—are the real motors of biotech.
A parting reflection
Innovation rarely sprints in straight lines. It zig-zags, slamming into real-world hurdles at every bend: off-target analytics that must convince risk-averse regulators; GMP lipid-nanoparticle runs that won’t collapse under scale; immunogenicity assays to prove the body won’t nuke the edit on dose two; and health-economics dossiers hefty enough for payers to green-light five-figure vials. CRISPR’s past two years show how breakthrough science only becomes clinical practice by grinding through these chokepoints—swapping elegant bench protocols for scalable, quality-controlled, reimbursement-ready platforms, and eventually feel as ordinary as an IV drip.
The next time someone claims biology crawls, point them to December 2023 through May 2025: two years, three milestones, and an industry pivoting from “Can we edit a gene?” to “Where else can this platform reach next?” That’s not just science—it’s human audacity in real time, and a signal for leaders to start mapping the opportunities before the next leap lands. Reading those signals now is how leaders future-proof strategy and steer health, business, and society toward what comes next.
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