Cell/gene therapy, RNA editing, and DMD treatment advances
Key Questions
What progress has UniQure's AMT-130 made in Huntington's disease?
AMT-130 gene therapy demonstrated a 75% reduction in disease progression and received FDA accelerated filing nod for Huntington's.
Which gene therapies recently gained expanded approvals for sickle cell disease?
Casgevy (exagamglogene autotemcel) was approved for patients as young as 2 years old, with real-world data showing functional cures and 100% crisis-free outcomes in pediatric cases.
What gene therapy restored hearing in deaf patients?
OTOF gene therapy (OTARMENI) received FDA approval; a single injection restored hearing in 100% of treated DFNB9 patients in initial trials and 90% in larger studies.
What funding and trials are advancing for Rett syndrome?
Neurogene raised $125M to support its Rett syndrome gene therapy program.
How is Scribe Therapeutics advancing gene editing?
Scribe Therapeutics plans an IPO and is developing STX-1150 for epigenetic silencing of PCSK9 in high cholesterol, alongside in vivo base editing showing dose-dependent LDL reduction.
What approvals occurred for SMA gene therapy in Europe?
Itvisma, a one-time SMA gene therapy, gained EU approval for patients of all ages 2 and older.
What early data supports EPI-321 in FSHD?
Gene-silencing therapy EPI-321 showed early increases in muscle volume in a Phase 1/2 FSHD study with a favorable safety profile.
Which CAR-T and cell therapies received recent FDA nods?
Tregzi was approved for GVHD with 78% cGVHD-free survival at 12 months, while Qihan dual-target CAR-T earned FDA triple crown status and REGENXBIO's RGX-202 advanced for DMD.
UniQure's AMT-130 Huntington's gene therapy (75% progression reduction, FDA accelerated filing nod); Spot Biosystems $40M non-viral EV DMD; Qihan dual-target CAR-T FDA triple crown; REGENXBIO RGX-202 DMD filing; first-in-human AMT-260 for epilepsy; Servier/n-Lorem ASO; first-in-human circ-arRNA DMD; Capricor's deramiocel FDA advisory; Novartis del-brax AOC for FSHD; CIRM $662M budget; Moderna in vivo CAR-T; Hatch BioFund nanoparticle platform; Unixell iPSC PD cell therapy; world-first gene therapy for rare immune disease; SAT-3247 Fast Track; Casgevy approved for children 2+ (100% crisis-free, 5,500 patient market); Tregzi approved for GVHD; Amondys 45/Vyondys 53 seeking full approval by Feb 2027; Neurogene $125M for Rett gene therapy. New: Casgevy pediatric clinical endpoints validated. New: First-in-human in vivo base editing for PCSK9 shows dose-dependent LDL reduction. New: Gene-silencing EPI-321 shows early muscle volume increase in FSHD. New: OTOF gene therapy (OTARMENI) FDA-approved; single injection restored hearing in 100% of deaf patients (DFNB9); 90% recovery in larger trial. New: Real-world Casgevy success in sickle cell patient. New: SMA gene therapy (Itvisma) approved in Europe for all ages. New: Opus Genetics FDA alignment on Phase 3 for OPGx-LCA5 gene therapy for LCA5-associated blindness; enrollment nearly complete. New: Scribe Therapeutics plans IPO; lead candidate STX-1150 (epigenetic silencing of PCSK9).