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

The FDA has granted accelerated and rare pediatric disease designations to 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 accelerated and rare pediatric disease designations to 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, ready-to-use, 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 being evaluated in a Phase 1/2 trial (NCT04984356) in patients with relapsed/refractory T-ALL or LBL.

“We are very pleased to have received both the Accelerated Designations and the Rare Pediatric Disease Designations, which reaffirm the great unmet need for new treatment options for people with R/R T-ALL/LBL,” said Dan Kemp, PhD, president and CEO of Wugen, said in a press release. “Earlier this year, we administered 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 escalation phase of study dose. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development. »

Preclinical studies have shown that WU-CART-007 exhibits high affinity and specificity for human CD7.2 Researchers observed strong cytotoxicity against CD7-expressing cells, including CCRF-CEM T-ALL cells, primary T and NK cells in vitro, but not observed 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 world’s first open human trial is enrolling patients with evidence of relapsed or refractory T-ALL or T-LBL, defined by WHO classification with at least 5% bone marrow blasts or screening signs of extramedullary disease.3 Other key inclusion criteria for the study include adequate kidney, liver, respiratory and cardiovascular function, as well as a life expectancy of more than 12 weeks.

The lower age limit of the trial is 12 years, and patients between the ages of 12 and 17 will be eligible to enroll in dose level 3 of the dose escalation part of the trial after a review safety, efficacy and cellular pharmacokinetics and discussion with regulators.

Key exclusion criteria include prior treatment with anti-CD7 therapy; prior treatment with anti-T cell monoclonal antibodies other than daratumumab (Darzalex); inability to recover from previous 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 any other serious underlying medical condition that could affect study treatment.

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

The primary endpoints of the trial include incidence of adverse events, establishment of maximum tolerated dose, overall response rate, duration of response, and progression-free survival. Secondary endpoints are 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. Press 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-targeting CAR-T cell therapy for T-cell malignancies. Blood. 2021;138(supplement 1):2772. doi:10.1182/sang-2021-153150
  3. A Phase 1/2 study of the safety and efficacy of allogeneic anti-CD7 CAR-T cells (WU-CART-007) in patients with relapsed or refractory ALL-T/LBL. ClinicalTrials.gov. Updated June 23, 2022. Accessed July 21, 2022. https://clinicaltrials.gov/ct2/show/NCT04984356