Sangamo's Gene Therapy Shows Sustained Benefits in Fabry Disease Patients with Improved Kidney Function
• Updated Phase 1/2 STAAR study data demonstrates sustained α-Gal A enzyme activity for up to 47 months, with all 18 ERT-dependent patients successfully discontinuing enzyme replacement therapy.
• Patients showed significant kidney function improvements with a positive mean annualized eGFR slope of 3.061 mL/min/1.73m2/year, indicating potential disease-modifying effects.
• Sangamo plans BLA submission in second half of 2025 following FDA's agreement to consider Accelerated Approval pathway based on 52-week eGFR data.
Royal Free London Opens New Clinical Trials Offering Hope for Pancreatic Cancer Patients
• The Royal Free London is launching multiple clinical trials to explore novel treatments for pancreatic cancer, a disease with historically poor survival rates.
• These trials will assess new immunotherapies, targeted drugs like Ginisortamab, and agents that enhance immune recognition of cancer cells in patients at all stages of the disease.
• A 'liquid biopsy' program will analyze cancer DNA to identify genetic abnormalities, potentially enabling personalized treatment strategies and improved trial selection.
• The initiative aims to provide more patients access to cutting-edge therapies and improve outcomes for a disease where treatment options have remained limited for years.
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