Wugen's Allogeneic CAR-T Therapy Shows Promising Long-Term Survival Data in T-Cell Leukemia Patients
核心洞察
Wugen (搜索)'s investigational allogeneic CAR-T therapy Soficabtagene Geleucel (搜索) demonstrated over one-hundred-fold expansion and persisted in circulation for up to three months in Phase 1/2 studies.
Three patients who received successful allogeneic stem cell transplants remain alive approximately two years after Sofi-cel (搜索) infusion, with no late adverse events of special interest reported.
The therapy uses CRISPR/Cas9 gene editing to delete CD7 (搜索) and TRAC (搜索) genes, preventing CAR-T cell fratricide and reducing graft-versus-host disease risk.
Wugen (搜索) Inc. will present encouraging long-term follow-up data for its investigational allogeneic CAR-T cell therapy Soficabtagene Geleucel (搜索) (Sofi-cel (搜索)) at the American Society of Hematology Annual Meeting this week, showing sustained survival in patients with relapsed or refractory T-cell acute lymphoblastic leukemia (搜索) and T-cell lymphoblastic lymphoma (搜索) (R/R T-ALL/LBL).
Robust Cellular Expansion and Persistence
In the Phase 1/2 study, Sofi-cel (搜索) demonstrated over one-hundred-fold expansion following infusion and persisted in circulation for up to three months. The serum cytokine profile was consistent with the therapy's mechanism of action, indicating appropriate immune activation without excessive inflammatory response.
The long-term follow-up study revealed no reported late adverse events of special interest or drug-related serious adverse effects. Notably, three patients who received successful allogeneic stem cell transplants in the Phase 1/2 study remain alive approximately two years after Sofi-cel (搜索) infusion, highlighting the therapy's potential in this challenging patient population.
"The robust cellular pharmacokinetics and long-term survival observed in heavily pretreated patients with R/R T-ALL/LBL in the Phase 1/2 study give confidence in the therapeutic potential of Soficabtagene Geleucel (搜索)," said Cherry Thomas, M.D., Wugen (搜索)'s Chief Medical Officer. "The T-RRex investigators bring extensive experience in this challenging disease and are committed to delivering meaningful clinical benefit for patients with limited treatment options."
Innovative Gene Editing Approach
Sofi-cel (搜索) represents a potential first-in-class allogeneic, anti-CD7 (搜索) CAR-T cell therapy engineered to overcome technological challenges in treating T-cell cancers. The therapy employs CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC (搜索)) genes, thereby preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host disease (GvHD).
The therapy is manufactured using healthy donor-derived T cells, eliminating the risk of malignant cell contamination historically observed in autologous CAR-T settings. This off-the-shelf approach enables scalable treatment with biologics-like cost of goods margins.
Pivotal Trial Progress
The pivotal Phase 2 T-RRex study (NCT06514794) is a single-arm trial evaluating the safety and efficacy of Sofi-cel (搜索) in patients with R/R T-ALL/LBL. The study is currently enrolling patients aged ≥1 year with R/R T-ALL/LBL and will include an exploratory cohort assessing patients with minimal residual disease (MRD)-positive status.
Regulatory Recognition
Sofi-cel (搜索) has received multiple regulatory designations recognizing its potential therapeutic value. The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations. The European Union has awarded Priority Medicines (PRIME) Scheme designation for the treatment of relapsed or refractory T-ALL/T-LBL. These designations provide increased agency support to expedite development and review of the therapy.
The clinical data will be presented as a poster on Sunday, December 7, from 6:00-8:00 p.m. ET at the ASH Annual Meeting in Orlando, with Alexander S. Hamil, Ph.D., from Wugen (搜索) serving as the presenter.