Cellectis S.A. — Cyborg Score 6/10

Solid
Biotechnology - Gene Therapy & Immuno-Oncology (CAR-T Cell Therapy)

Strategic Profile

Cellectis has 25 years of expertise in gene editing based on its flagship TALEN® technology and pioneering electroporation system PulseAgile. The company develops a new generation of immunotherapy product candidates with additional safety and efficacy attributes equipped to resist mechanisms that inhibit immune system activity. The allogeneic CAR-T approach offers significant competitive advantages over standard autologous therapies.

Cyborg Score Rationale

Recent Phase 1 BALLI-01 study data for lasme-cel (UCART22) demonstrated 68-100% efficacy rates depending on dose escalation. The company has strong intellectual property and technology platforms. However, as a clinical-stage company, Cellectis faces execution risk and cash burn concerns typical of pre-revenue biotech.

Top Insights

  • UCART22 (lasme-cel) Phase 1 data showed 68% overall response rate and up to 100% efficacy in target Phase 2 population
  • Cellectis' allogeneic approach uses T-cells from healthy donors with gene editing to disable genes causing graft-versus-host disease, offering off-the-shelf manufacturing advantages
  • Recently published Nature Communications article establishing circular single-stranded DNA (CssDNA) as efficient non-viral gene insertion platform for hematopoietic stem cells
  • Company maintains strong cash position of $264 million as of September 30, 2024, with strategic collaborations including AstraZeneca

Named Competitors

  • Autologous CAR-T therapies — Traditional patient-derived CAR-T cell therapies
  • Autologous CAR-T therapies — Commercially approved autologous CAR-T for hematologic malignancies
  • Allogeneic cell therapy platform — Off-the-shelf cell therapy development

Recent Developments

  • (November 2025) Published Nature Communications article on non-viral CssDNA gene insertion technology for hematopoietic stem cells
  • (October 2025) Presented Phase 1 BALLI-01 data for lasme-cel (UCART22) with promising efficacy in heavily pretreated B-ALL patients
  • (August 2025) Reported Q2 2025 financial results and business updates

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