Insights

September 18, 2026 · Regulatory Approvals Sanfilippo A Has Its First Gene Therapy. The Comparator Was an Untreated Historical Cohort The FDA approved Fayuvi for MPS IIIA, the first treatment for Sanfilippo syndrome type A. The efficacy study was open-label and single-arm, read against an untreated historical control cohort. September 11, 2026 · Financing & Clinical Development Encoded's $275M Dravet Round Pays for the Pivotal Trial and Its Own Manufacturing at Once Encoded Therapeutics closed a $275M Series F for ETX101, its Dravet syndrome gene therapy. It will support the pivotal study and commercial scale-up of in-house GMP manufacturing. September 10, 2026 · Clinical Development Elicera's CAR-T Escalation Never Found a Dose Limit. The Highest Dose Goes to Phase IIa Elicera's CARMA Phase I finished with no dose-limiting toxicities reported, so the safety board picked the highest dose tested for the Phase IIa expansion. September 8, 2026 · Gene Editing & Clinical Data A Base Editor That Skips the Mutation Just Worked Across Four Different Genotypes CorrectSequence treated four patients from Nigeria, Laos, Malaysia and Pakistan, carrying four different genotypes. The editor never touches the causative mutation — it switches a second gene back on, which is why one strategy can span all four. September 7, 2026 · Regulatory Submissions uniQure Filed on September 2 With Evidence the FDA Declined in March and Accepted in June On 2 September uniQure filed a Huntington’s BLA on a three-year Phase I/II analysis against an Enroll-HD external control. On 2 March the FDA said that evidence was not sufficient and recommended a sham surgery-controlled study. On 17 June it said the same analysis would serve as the primary basis of a BLA. September 2, 2026 · Safety & Manufacturing Three Deaths Paused Novartis' Autoimmune CAR-T. BMS Paused Too, and Both Used Rapid Manufacturing Novartis halted eight autoimmune and neuroscience trials of its CD19 CAR-T after three deaths from serious IEC-HS. Days later BMS paused its own. Same target, different companies, and both built on a rapid manufacturing platform. August 31, 2026 · Clinical Data & Durability The Liver Replaces Its Own Cells - This CRISPR Edit's Effect Held Flat for a Year A year after one CRISPR infusion, ANGPTL3 and lipid levels were still low and flat — in a liver that replaces nearly 20% of its cells every year. An investigator says the FDA has asked for 15 years of follow-up. August 25, 2026 · Patient Safety & Regulatory A Fix for One REGENXBIO Gene Therapy Just Triggered a Hold on Another REGENXBIO's RGX-121 was placed on a new FDA clinical hold after asymptomatic spine findings in 5 of 48 trial participants — caught only by monitoring built for a different drug. August 21, 2026 · Regulatory Approvals FDA Approved a $2.7M Gene Therapy for GSDIa - 1 in 10 Patients Had Anaphylaxis in Trials GENGLYCOS just became Ultragenyx's first approved gene therapy. Its own trial data lists seven serious adverse events, including a 10% anaphylaxis rate. August 19, 2026 · Patient Safety & Regulatory No Law Required RiboX to Disclose This Death - the FDA Relied on the Data Anyway Three patient deaths in China's trials surfaced within weeks. Days before the third, the FDA leaned on the same trial-data class for an approval. August 18, 2026 · M&A & Bankruptcy Bankrupt Sangamo's Pipeline Just Sold for $163.55M Sangamo filed for Chapter 11 in June 2026. Six weeks later, PTC and Eli Lilly bought its gene therapy assets for $163.55 million.