1/🚨WOW! Prime Medicine just announced that the U.S FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PM359 - $PRME leading autologous stem Cell Therapy for the treatment of p47phox-deficient Chronic Granulomatous Disease (CGD). RMAT designation is not only a super important milestone - which was granted to Prime Medicine based on PM359’s previous promising Phase 1/2 clinical data 🧵👇, but it will also provide the company with significant benefits such as an intensive FDA guidance and an expedited review through the program’s development. $XBI
Another important milestone: FDA has granted RMAT designation to PM359 for p47phox-deficient CGD — based on Phase 1/2 data published in NEJM. PM359 now holds RMAT + Fast Track + Orphan Drug + Rare Pediatric Disease Designations. #GeneEditing#RareDisease#FDA#PrimeEditing
1/🚨WOW! Prime Medicine has announced today that the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has cleared its Clinical Trial Application (CTA) for $PRME PM577a - an investigational Prime Editor for treating Wilson Disease 🧵👇. Today’s clearance represents the first clinical authorization for an In Vivo Prime Editing therapy and enables the initiation of a global Phase 1/2 study. $XBI
$PRME ✨
Major Milestone
Prime Medicine announced that New Zealand's Medsafe has cleared its Clinical Trial Application (CTA) for PM577a, a Prime Editing therapy for Wilson Disease.
✅ First-ever clinical authorization for an in vivo Prime Editing therapy from Prime Medicine
✅ Global Phase 1/2 trial expected to begin in H2 2026
✅ Initial proof-of-concept clinical data expected in 2027
✅ PM577a targets the ATP7B H1069Q mutation, responsible for approximately 30–50% of Wilson Disease cases in North America and Europe
✅ Follow-on candidate targeting the R778L mutation, common in East Asian populations, is already in preclinical development
Wilson Disease currently has no approved cure other than liver transplantation.
Prime Medicine aims to correct the root genetic cause with a single-dose Prime Editing treatment.
This represents another important step toward bringing Prime Editing into real-world medicine.
https://t.co/ayWwpos6H5
https://t.co/Wt1qCAqp7j
#PRME #PrimeMedicine #PrimeEditing #GeneEditing #WilsonDisease #Biotech
Milestone moment:
✅ New Zealand Clinical Trial Application cleared for PM577a
✅ First clinical authorization for our in vivo Prime Editing therapy
✅ Phase 1/2 trial in Wilson disease to begin
A step toward a potential one-time genetic cure. Data expected 2027.
$PRME Program Updates
Wilson’s Disease IND on track for 1H 2026 • AATD IND on track for mid-2026.
PRME remains the only gene editing company heading into the clinic for Wilson’s Disease.
There is one gene editing company for CF (presumably Intellia-Recode), but has not heard much about their progress.