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Cellectis Announces Strategic Transformation To In Vivo Gene Editing Company

  • Cellectis board approved a strategic transformation on September 11, 2026, to become an in vivo gene editing company focused on long-lasting chronic disease treatments.
  • The company will advance two lead programs: .HEAL-101 for severe hypertriglyceridemia & .HEAL-201 for severe hypercholesterolemia, with preliminary Phase 1 data expected in H2 2027 & H1 2028 respectively.
  • .HEAL-101 uses APOC3 base editing via LNP to target patients with triglyceride levels ≥880 mg/dL or ≥500 mg/dL with pancreatitis history, showing >70% on-site editing & >76% triglyceride reduction in murine models.
  • .HEAL-201 utilizes PCSK9 epigenetic editing via LNP to address high-risk patients with elevated LDL-C, demonstrating >90% on-site editing & >90% reduction in plasmatic PCSK9 levels in humanized murine models.
  • Cellectis will exit the development of lasme-cel & eti-cel due to a shrinking addressable patient population & increased competition from bispecific antibodies & in vivo CAR-T approaches, while seeking strategic partnerships for these assets.
  • The company will realign its operating model to focus on the in vivo pipeline & support existing cell therapy partnerships with AstraZeneca, Allogene, Servier, & Iovance.
  • This realignment aims to extend the company's cash runway into H2 2028 to fund advancement through key development milestones.
  • A conference call & webcast regarding the announcement is scheduled for September 14, 2026, with English at 8 a.m. ET & French at 9 a.m. ET.
  • Trading of Cellectis shares on Euronext Paris will be temporarily halted on September 14, 2026, from 9:00 a.m. CET until 3:30 p.m. CET.