Sickle Cell Disease
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Vertex Unveils New CASGEVY® Data—including First Results in Children Aged 5‑11—at ASH Annual Meeting, Announces Global Regulatory Plans
Vertex presented Phase 3 data showing CASGEVY’s efficacy and safety in children 5‑11 with severe sickle cell disease (SCD) or transfusion‑dependent beta thalassemia (TDT). All 11 SCD patients remained vaso‑occlusive‑crisis‑free for ≥12 months; 12 of 13 TDT patients achieved ≥12 months transfusion independence, with one fatal conditioning event. Results mirror those in patients ≥12 years. Vertex will file global supplemental BLAs in early 2026, aiming for accelerated review, expanding the first‑in‑class gene‑editing therapy to the pediatric market.
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Lobe Sciences Secures European Unitary Patent for DHA-Based Sickle Cell Treatment
Lobe Sciences announced the validation of its European Unitary Patent EP4117446 across 17 EU member states, covering its proprietary DHA-based composition for Sickle Cell Disease (SCD). This patent protects Altemia®, an innovative medical food designed to improve red blood cell integrity and address fatty acid deficiencies in SCD patients. The validation significantly strengthens Lobe’s intellectual property in crucial European territories, advancing its commercialization roadmap for Altemia®.
