FDA approves 1st gene therapy for Wiskott-Aldrich syndrome / Beckers

Ella Jeffries / beckershospitalreview - The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder. Waskyra is indicated for pediatric patients 6 months and older, as well as adults with Wiskott-Aldri…

#healthcare #pharmaceuticals #publichealth #biotech #drugdevelopment #fda

---

Monday, December 15, 2025, 2:21 am / permalink 16954 / 4 stories in 2 months

Related Stories

---

• FDA Lifts Hold on Intellia’s Phase 3 Gene Editing Trial / 5 wks
• FDA rejects Corcept’s cortisol modulator triggering setback / 2 months
• FDA approves Rucaparib for advanced prostate cancer / 2 months
• FDA Approves Novartis’ Itvisma Gene Therapy for SMA Across Age Groups / 3 months
• FDA Approves Ziftomenib for AML Treatment / 3 months
• FDA updates Sarepta gene therapy label with severe warnings / 3 months
• Experimental therapy reduces seizures in children with Dravet syndrome / 5 days

Related Tags

---

StackHealth Time Machine

---