On microdystrophin, the FDA is doing what the accelerated-approval standard has always required: asking that a biomarker be shown to predict real benefit before it is trusted. Much of the commentary around REGENXBIO's RGX-202 has framed the FDA's stance as regulatory inconsistency, arguing that…

RGX-202 BLA submission for Duchenne muscular dystrophy on track for Q3 2026 initiation, with potential accelerated approval in 2H 2027 Long-term surabgene lomparvovec (sura-vec, ABBV-RGX-314) data highlight durable safety and efficacy profile ahead of key catalyst Topline data from...

This edition highlights major developments across gene and cell therapy, including FDA approval of TREGZI, China's approval of satri-cel, progress toward the RGX-202 BLA submission, initiation of the Phase IIb/III NAAVIGATE study for sura-vec, expanded eligibility for CASGEVY, European approval of Itvisma for spinal muscular atrophy, a new Korean cell and gene therapy alliance led by GC Cell, and MeiraGTx's financing to support commercialization of late-stage gene therapy programs.

This biweekly Gene and Cell Therapy video recap highlights regulatory milestones, clinical progress, manufacturing collaborations, financing activity, and advancing development across multiple therapeutic areas. Key highlights include: 🧬 RGX-202 BLA path advances 👁️ Sura-vec NAAVIGATE begins 🧫 Satri-cel wins China approval 🩸 TREGZI gains FDA approval 👶 CASGEVY label expands to age 2+ 🧬 Itvisma wins EU SMA approval 🤝 GC Cell forms Korean CGT alliance 💰 MeiraGTx secures Oberland financing Chapters: 0:00 Introduction 0:09 RGX-202 BLA path advances 0:35 Sura-vec NAAVIGATE begins 0:58 Satri-cel wins China approval 1:18 TREGZI gains FDA approval 1:42 CASGEVY label expands to age 2+ 2:04 Itvisma wins EU SMA approval 2:24 GC Cell forms Korean CGT alliance 2:46 MeiraGTx secures Oberland financing 3:08 How to reach us 📢 Stay Ahead in Gene and Cell Therapy Research! ✅ Like, share, and subscribe for future updates on Gene and Cell Therapy ✅ Visit www.lqventures.com for expert healthcare insights and consulting services. ✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Gene and Cell Therapy innovations and clinical research. 🔔 Turn on notifications so you never miss an update! #GeneandCellTherapy #GeneTherapy #CellTherapy #FDA #ClinicalTrials #CART #RareDisease #Biotech #PharmaNews #LucidQuest

REGENXBIO announces the successful conclusion of dosing in its confirmatory study of RGX-202, a promising gene therapy for Duchenne muscular dystrophy, paving the way for FDA approval.

Confirmatory study completed ahead of schedule due to strong patient demand and robust investigator interest On track to initiate BLA in Q3 2026 under the accelerated approval pathway supporting potential approval in 2H 2027 ROCKVILLE, Md., June 24, 2026 /PRNewswire/ -- REGENXBIO Inc....

Reported Wednesday, Jun 24 - 7:06am EDT (TheFly subscription required to read more.)

Confirmatory study completed ahead of schedule due to strong patient demand and robust investigator interest On track to initiate BLA in Q3 2026 under the accelerated approval pathway supporting potential approval in 2 H 2027. ROCKVILLE, Md., June 24, 2026/ PRNewswire/-- REGENXBIO Inc. today announced the successful completion of dosing in the confirmatory...

This edition highlights key developments in gene and cell therapy, including UK MHRA approval of Vyjuvek for dystrophic epidermolysis bullosa and pivotal Duchenne muscular dystrophy data from REGENXBIO’s RGX-202 program. Additional updates include Beam Therapeutics’ BEAM-302 clinical results, Regeneron’s EMA filing acceptance for Otarmeni, Lilly and Verve’s VERVE-102 advancement toward Phase 2, and emerging activity across cardiovascular and neurodegenerative cell therapy programs. Strategic collaborations and regulatory momentum continue to shape the advanced therapeutics landscape.

REGENXBIO Inc. has announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne...

Regenxbio’s DMD gene therapy showed strong results, boosting muscle protein and safety, moving closer to FDA approval.

Reported Thursday, May 14 - 7:24am EDT (TheFly subscription required to read more.)

In a recent update, REGENXBIO highlighted its Q1 2026 performance, revealing major milestones in gene therapy development, particularly RGX-202.

REGENXBIO has released promising Phase III trial results for RGX-202, a gene therapy that shows high microdystrophin expression in Duchenne muscular dystrophy patients.