FDA Grants Fast Track and Rare Pediatric Disease Designations to WU-CART-007 in R/R T-ALL and LBL

The FDA has granted fast track and rare pediatric disease designations to the CAR T-cell therapy WU-CART-007 for the treatment of patients with relapsed/refractory T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma.

The FDA has granted fast track and rare pediatric disease designations to the CAR T-cell therapy WU-CART-007 for the treatment of patients with relapsed/refractory T-cell acute lymphoblastic leukemia (R/R T-ALL) and lymphoblastic lymphoma (LBL).1

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR T-cell therapy. The product is designed to overcome the technological challenges of harnessing CAR-T cells to treat CD7-positive hematological malignancies. WU-CART-007 is currently under evaluation in a phase 1/2 trial (NCT04984356) in patients with relapsed/refractory T-ALL or LBL.

“We are very pleased to have received both fast track and rare pediatric disease designations, which re-affirm the great unmet need for new treatment options for people with R/R T-ALL/LBL,” said Dan Kemp, PhD, president and chief executive officer of Wugen, stated in a press release. “Earlier this year, we dosed the first patient in our ongoing phase 1/2 trial of WU-CART-007 for relapsed/refractory T-ALL/LBL and are currently in the dose escalation phase of the study. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development.”

Preclinical studies showed that WU-CART-007 displayed high affinity and specificity for human CD7.2 Investigators observed strong cytotoxicity against CD7-expressing cells including CCRF-CEM T-ALL cells, primary T and NK cells in vitro, but not seen in CD7 cells such as myeloid cells, B cells, hepatocytes, astrocytes, cardiomyocytes, epithelial cells, and endothelial cells. Additionally, they did not observe cytotoxicity against hematopoietic progenitor cells in human bone marrow or cord blood.

The global, open-label, first-in-human trial is enrolling patients with evidence of relapsed or refractory T-ALL or T-LBL, defined by WHO classification with bone marrow blasts of at least with 5% or evidence of extramedullary disease at screening.3 Other key inclusion criteria for the study include adequate renal, hepatic, respiratory, and cardiovascular function, plus a life expectancy of more than 12 weeks.

The lower age limit of the trial is 12 years, and patients between 12 and 17 years old will be eligible to enroll at dose level 3 of the dose-escalation portion of the trial after a review of safety, efficacy and cellular pharmacokinetics and discussion with regulatory agencies.

Key exclusion criteria include prior treatment with any anti-CD7 therapy; prior treatment with anti-T cell monoclonal antibodies other than daratumumab (Darzalex); failure to recover from a prior therapy; active or latent hepatitis B, active hepatitis C, any uncontrolled infection, or untreated HIV positive; and any serious active infection at the time of treatment, or another serious underlying medical condition that could affect study treatment.

Once enrolled, patients will undergo lymphodepletion with daily doses of 500 mg/m2 of cyclophosphamide and 30 mg/m2 of fludarabine 30 mg/m2 on days -5 to -3. On day 1, patients will receive an intravenous infusion of WU-CART-007.

The primary end points of the trial include incidence of adverse effects, establishing the maximum tolerated dose, overall response rate, duration of response, and progression-free survival. Secondary end points consist of overall survival and hematopoietic stem cell transplant rate.


  1. Wugen receives US FDA fast track and rare pediatric disease designations for WU-CART-007 for the treatment of R/R T-ALL/LBL. News release. Wugen. July 19, 2022. Accessed July 21, 2022. https://bwnews.pr/3yZkJdH
  2. Leedom T, Hamil AS, Pouyanfard S, et al. Characterization of WU-CART-007, an allogeneic CD7-targeted CAR-T cell therapy for T-cell malignancies. Blood. 2021;138(suppl 1):2772. doi: 10.1182/blood-2021-153150
  3. A phase 1/2 study of the safety and efficacy of anti-CD7 allogeneic CAR-T cells (WU-CART-007) in patients With relapsed or refractory T-ALL/LBL. ClinicalTrials.gov. Updated June 23, 2022. Accessed July 21, 2022. https://clinicaltrials.gov/ct2/show/NCT04984356