Startseite Kongressberichte 2020 All-Virtual 62nd ASH Annual Meeting and Exposition Press Briefings Press Briefing on Advancing New Frontiers: Genome Editing & Cellular Therapy

Advancing New Frontiers: Genome Editing & Cellular Therapy (Moderator - Catherine Bollard, MD, Children’s National Research Institute)

Advancing New Frontiers: Genome Editing & Cellular Therapy press release

Abstracts presented:

4 Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells

Haydar Frangoul, Yael Bobruff, Maria Domenica Cappellini, et al.
Conclusions: These data demonstrate that CTX001, a first-in-human, CRISPR-Cas9–modified autologous HSPC product, has resulted in increases in HbF and total Hb in the first 7 patients infused. All patients infused with CTX001 demonstrated hematopoietic engraftment with a post-infusion safety profile generally consistent with myeloablation. All 5 patients with TDT have been transfusion-free since ~2 months after CTX001 infusion and the 2 patients with severe SCD have had no VOCs during follow-up after CTX001 infusion. These early data demonstrate that CTX001 is a potential functional cure for the treatment of TDT and SCD. Data will be updated for the presentation.

556 CD58 Aberrations Limit Durable Responses to CD19 CAR in Large B Cell Lymphoma Patients Treated with Axicabtagene Ciloleucel but Can be Overcome through Novel CAR Engineering

Robbie G. Majzner, Matthew J. Frank, Christopher Mount, et al.
Conclusion: We have identified that CD58 status is an important biomarker for durable response to CAR T cells in LBCL. We modeled the biologic basis for this finding and generated CAR T cells capable of overcoming CD58 loss in B cell malignancies. CD58 mutations have been reported in many cancers, including multiple myeloma and colon cancer, and are likely to play a role in immune evasion for CAR T cells as they are developed for additional histologies. These data provide a rationale for investigating CD58 status for patients receiving CAR based therapeutics and devising next-generation CARs capable of overcoming this newly discovered mechanism of resistance.

700 Primary Analysis of Zuma-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL) 

Caron Jacobson, Julio C. Chavez, Alison R. Sehgal, et al.

Conclusions: Axi-cel had a considerable and durable clinical benefit in patients with iNHL, with high ORR and CR rates. Axi-cel had a manageable safety profile, with lower rates of Grade ≥ 3 NEs observed in patients with FL vs those in patients with MZL and those previously reported in aggressive NHL (Locke, et al. Lancet Oncol. 2019).