Skinny Genes: Your Weekly “Skinny” on Gene Therapy

Steven Piluso
  • 5.0 (1)
  • Life Sciences
  • Updated Weekly

Your weekly “skinny” on all things Gene Therapy related in a 15 minute briefing. Hosts Sophie and Thomas cover research breakthroughs, company news, clinical trials and regulatory updates, with an “Angelman Syndrome First” lens.

Episodes

  1. 3d ago

    10/19/2026 - Episode 3

    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.

  2. Oct 12

    10/12/2026

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

  3. Oct 7

    Skinny Genes - 10/03/2026

    Update on AS trials, general industry news

  • 3 Episodes

About

Your weekly “skinny” on all things Gene Therapy related in a 15 minute briefing. Hosts Sophie and Thomas cover research breakthroughs, company news, clinical trials and regulatory updates, with an “Angelman Syndrome First” lens.

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