#基因治疗/编辑

#基因治疗/编辑

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Oct 8Thu

Thursday

06:21
fda.govScore4141
Recently Issued Guidance Documents
The FDA has released a set of guidance documents for industry and agency staff issued from July to September 2026. The list includes final guidances on blood collection and storage systems for blood components using the buffy coat method, formal PDUFA meetings, cancer clinical trial eligibility criteria on laboratory values, washout periods and performance status, and FAQs for cellular and gene therapy products. Draft guidances cover premarket approval electronic submission templates, potency assessment of active immunotherapy products, container closure systems for drugs, biologicals and biosimilars.
Regulatory #Policy & payers #Gene therapy & editing #Immunotherapy

Sep 18Fri

Friday

02:26
fda.govPickScore7070
FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy from Ultragenyx Pharmaceutical, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy.
Regulatory #Regulatory #Gene therapy & editing #Rare disease
Why it matters: First FDA-approved therapy for a rare pediatric neurodegenerative disease with no prior disease-altering treatment.

Aug 20Thu

Thursday

05:33
fda.govPickScore6969
FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia
The FDA granted accelerated approval to Ultragenyx Pharmaceutical's Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy and the first approved treatment for glycogen storage disease type Ia in patients aged 8 and older. In a 48-week randomized placebo-controlled study, patients achieved a statistically significant 31% mean reduction in daily cornstarch intake, the primary endpoint. The manufacturer must complete additional trials to confirm effectiveness, and the label warns of anaphylaxis, liver toxicity, adrenal insufficiency and tumorigenicity risk.
Regulatory #Regulatory #Gene therapy & editing #Rare disease
Why it matters: FDA cleared the first therapy for glycogen storage disease type Ia under the accelerated pathway.