Skinny Genes: Your Weekly “Skinny” on Gene Therapy

Skinny Genes — Week of November 2, 2026 (10 minutes 38 seconds).
This week on Skinny Genes, Sophie and Tommy lead with Angelman syndrome: long-read sequencing finds three previously undescribed UBE3A variants in thirteen children, a new study maps the HERC2-UBE3A protein connection, and Ultragenyx's discontinued apazunersen studies officially close, leaving the Ionis and Oak Hill Bio Phase 3 programs carrying the field forward.
Also covered: the FDA's draft guidance that could let personalized gene-editing therapies add patient-specific versions to a single application, and a plan to expand Medicare and Medicaid's gene therapy access model; Inovio's Voceryq, the first FDA-approved DNA medicine, winning accelerated approval for recurrent respiratory papillomatosis; Beam's BEAM-301 base editing data in glycogen storage disease, with two patients fasting fifteen hours and coming off starch; YolTech's YOLT-203 CRISPR update in primary hyperoxaluria; a sobering safety report from HuidaGene's Duchenne gene-editing trial, published in Cell; a Penn Medicine backup-protein approach in Duchenne mice; Solid Biosciences' Friedreich's ataxia program earning European orphan designation; new eye and delivery science from the Hamburg gene therapy congress; and a federal judge dismissing an investor lawsuit against Rocket Pharmaceuticals

This week on Skinny Genes, Sophie and Tommy lead with Angelman syndrome: a Yale nonviral CRISPR platform that reactivates the silenced paternal UBE3A gene across the brain in preclinical models, and a new Nature paper showing that blocking a buildup of the enzyme ASNS restores brain growth and behavior in Angelman mice.
The biggest clinical story is the first Phase 3 success for a personalized mRNA cancer vaccine: Moderna and Merck's intismeran autogene plus Keytruda cut melanoma recurrence or death risk by 42 percent in the INTerpath-001 trial, with full data presented at ESMO on October 24. Also covered: Opus Genetics' FDA clearance to start a trial of OPGx-RDH12 for childhood blindness, a one-patient ALS therapy built in under a year, a sobering safety disclosure from Bristol Myers Squibb's zola-cel multiple sclerosis trial, a European orphan designation for Rznomics' RNA-editing glioma therapy, a new compact CRISPR enzyme structure, DeltaRex-G data at ESMO, and a look ahead to the ESGCT congress in Hamburg. Lawsuit watch: a securities-fraud investigation notice on Moderna, not a filed lawsuit.

Ultragenyx is selling two rare pediatric disease priority review vouchers — worth a combined $413 million — as it restructures after discontinuing apazunersen following the failed Phase 3 Aspire trial; Q3 results land in November. Meanwhile three late-stage Angelman programs keep moving: Oak Hill Bio's rugonersen in the Phase 3 BEACON trial, Ionis's obudanersen in the Phase 3 REVEAL study, and MavriX Bio's MVX-220 gene replacement in the Phase 1/2 ASCEND-AS trial.
The week's biggest clinical headline: REGENXBIO presented full pivotal data for RGX-202 in Duchenne muscular dystrophy at the Child Neurology Society meeting — the Phase 3 AFFINITY DUCHENNE trial met its primary endpoint with 93% of participants clearing the microdystrophin threshold, plus a first-ever correlation between expression level and functional improvement; a BLA is planned for Q4 2026.
Also covered: Opus Genetics reported durable two-year vision data for OPGx-LCA5 and early results for OPGx-BEST1 in retinal disease; Ocugen completed Phase 3 enrollment for OCU400 in retinitis pigmentosa; Sangamo closed its Chapter 11 asset sales (PTC acquired the Fabry gene therapy ST-920, Lilly bought the delivery platforms); Vivacta/Grit won FDA orphan designation for an in vivo CAR-T; XellSmart unveiled Phase 1 Parkinson's cell-therapy data; City Therapeutics went public on Nasdaq; and Estuary Biotherapeutics launched with $103M for kidney gene therapies.

Here is your weekly “Skinny!” from Sophie and Thomas!

Update on AS trials, general industry news