Cell/gene therapy, RNA editing, and DMD treatment advances
Key Questions
What progress has been made in gene therapies for Huntington's disease?
UniQure's AMT-130 showed 75% progression reduction and received FDA accelerated filing nod. Machine learning models predict conversion with 88-98% accuracy, aiding patient stratification.
Which gene therapies have received recent regulatory approvals?
Casgevy was approved for children age 2+, OTOF gene therapy gained FDA approval, and SMA gene therapy was approved in Europe. Dyne Therapeutics' z-rostudirsen received BLA acceptance with Priority Review.
What advances exist for Duchenne muscular dystrophy treatments?
Sarcomatrix plans first human trials of S-969 in 2027 as a mutation-agnostic oral therapy. Novartis filed for FDA approval of del-zota for exon 44, while Capricor's Deramiocel faces FDA scrutiny over efficacy data.
How is in vivo gene editing being applied clinically?
Intellia's lonvo-z Phase 3 for HAE showed 87% attack reduction, and VERVE-102 base editing halved LDL in early trials. First-in-human in vivo base editing for PCSK9 has also occurred.
What cell therapies are advancing for Parkinson's disease?
Kenai Therapeutics enrolled the REPLACE trial of iPSC-derived dopaminergic neurons, and Scripps reported 12-month interim data from autologous iPSC transplants showing functional potential.
What safety concerns have arisen in gene-editing trials?
A Chinese university is investigating a girl's death in a gene-editing trial, highlighting oversight issues. Intellia's nex-z trial hold was lifted after a liver toxicity death with updated monitoring.
Which rare disease therapies received new designations or funding?
CHOP received a $38.9M ARPA-H award for personalized gene editing in rare liver diseases, and multiple therapies like those for Leigh syndrome and Friedreich's ataxia gained FDA rare pediatric disease or orphan designations.
What epigenetic or RNA editing approaches are emerging?
Epigenetic editing achieved 70% cholesterol reduction in monkeys without DNA cuts, with trials underway for hepatitis B. CAMP4's ASO targets regRNA for SYNGAP1 haploinsufficiency, and personalized ASOs reduced seizures by 90% in SCN2A cases.
UniQure's AMT-130 Huntington's gene therapy (75% progression reduction, FDA accelerated filing). Intellia's lonvo-z Phase 3 for HAE shows 87% attack reduction. Epigenetic editing shows 70% cholesterol reduction in monkeys. Sarcomatrix plans first human trial of S-969 for DMD in 2027. Safety note: Chinese university investigating girl's death in unregistered base editing trial, raising red flags for gene therapy safety. Capricor's deramiocel for DMD cardiomyopathy: FDA panel voted 9-3 against effectiveness but supportive on upper limb function; PDUFA Aug 22. FDA briefing documents dispute Capricor's Phase 3 efficacy claims, with advisory committee meeting July 29. New: One-time gene therapy for SOD1-ALS extends lifespan 100 days in mice, using AAV microRNA. New: Vamorolone early intervention in DMD boys under 4 shows motor improvement. New: Atsena Therapeutics gains EMA orphan status for retinal gene therapies. New: Voyager's VY1706 tau gene therapy (one-dose IV, 75% tau reduction in NHP, IND cleared, first-in-human imminent). New: CHOP receives $38.9M ARPA-H THRIVE award for personalized gene editing. New: Intellia's nex-z trial hold lifted after liver toxicity death. New: Affinia Gene Therapy wins FDA orphan for BAG3 cardiomyopathy. New: Gene therapy for MLD saves baby at CHOP, underscoring newborn screening gaps. New: In vivo CAR gene therapy review highlights promise and safety concerns (off-target integration, immunogenicity). New: NB-4746 SARM1 inhibitor enters Phase 1/2 trial in Canada for ALS, targeting axonal degeneration. New: Ocugen's gene therapy OCU410 gets FDA RMAT for geographic atrophy, showing 31% GA lesion reduction in Phase II. New: Gene therapy for infantile-onset Pompe disease: single AAV9 infusion enabled 5/6 children to stop ERT and remain ventilator-free for 1 year; motor milestones achieved, GAA activity restored. New: First personalized CRISPR base editing therapy for CPS1 deficiency in infant, demonstrating rapid custom therapy development. New: FDA expands Casgevy to age 2 for SCD/TDT, expanding pediatric access. New: Affinia Gene Therapy wins FDA orphan for BAG3 cardiomyopathy. New: Sana's hypoimmune gene-edited islet cells survive 14 months without immunosuppression in T1D (proof-of-concept). New: CRISPRa-driven β-tubulin compensation strategy for muscular dystrophy offers compensatory gene therapy approach. New: Rocket Pharmaceuticals' RP-A501 gene therapy for Danon disease shows positive safety update under modified Phase 2 protocol (no TMA or capillary leak in first 3 patients). New: Opus Genetics completes Phase 3 enrollment for LCA5 gene therapy for inherited retinal disease.