Caribou Biosciences Announces FDA Clearance of IND Application for CB-012, an Allogeneic Anti-CLL-1 CAR-T Cell Therapy for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

-- AMpLify Phase 1 clinical trial to initiate patient enrollment by mid-2024 --

-- CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption and immune cloaking to enter the clinic --

BERKELEY, Calif., Oct. 18, 2023 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that it received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-012, an allogeneic anti-C-type lectin-like molecule-1 (anti-CLL-1) CAR-T cell therapy. CB-012 will be evaluated in the multicenter, open-label, AMpLify Phase 1 clinical trial for patients with relapsed or refractory acute myeloid leukemia (r/r AML).

“Clearance of our IND application for CB-012 represents another significant milestone for Caribou as our third off-the-shelf CAR-T cell therapy enters the clinic,” said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer. “We look forward to initiating patient enrollment in the AMpLify Phase 1 trial by the middle of 2024 to evaluate the safety and tolerability of CB-012 in patients suffering from AML.”

CLL-1 is a compelling therapeutic target because it is highly expressed on AML cells and leukemic stem cells, but it is not expressed on hematopoietic stem cells.

“There is an urgent need to develop new treatments for patients with relapsed or refractory AML, for which the treatment options are predominantly limited to salvage chemotherapy regimens,” said Naval Daver, MD, associate professor and director, Department of Leukemia, The University of Texas MD Anderson Cancer Center. “An allogeneic CAR-T cell therapy that could safely and effectively target AML blasts while preserving healthy hematopoietic stem cells could provide a much-needed off-the-shelf option for these patients.”

Caribou’s patented next-generation CRISPR Cas12a chRDNA genome-editing technology platform maintains high genomic integrity and significantly improves the specificity of genome editing.

“CB-012 was engineered with five genome edits, and is the first allogeneic CAR-T cell therapy, to our knowledge, with both checkpoint disruption through a PD-1 knockout, and immune cloaking through a B2M knockout and B2M–HLA-E fusion transgene insertion,” said Steve Kanner, PhD, Caribou’s chief scientific officer. “Both armoring strategies are designed to improve the antitumor activity of CB-012 that we believe are crucial for targeting this difficult to treat indication.”