BERKELEY, CA — November 3, 2025—Caribou Biosciences has announced promising clinical data for its lead allogeneic (off-the-shelf) CAR-T therapy, CB-010 (vispa-cel), suggesting the ready-made treatment may be competitive with existing custom-made therapies for blood cancer—a success point where many other cell therapy developers have previously faltered. However, the company, co-founded by Nobel laureate Jennifer Doudna, faces a critical financial hurdle: it lacks the resources to fund the larger, pivotal trial required to seek FDA approval.
Vispa-cel Data Rivals Approved Therapies
Caribou reported updated results from its Phase 1 trial in patients with large B cell lymphoma (LBCL). The data, though not from a head-to-head comparison, showed efficacy comparable to approved autologous (patient-specific) CAR-T treatments like Bristol Myers Squibb’s Breyanzi and Gilead’s Yescarta.
In the 22 LBCL patients who met specific criteria:
- Overall Response Rate (ORR): 82% of patients responded to treatment.
- Complete Response (CR) Rate: 64% saw all signs of their lymphoma disappear.
- Progression-Free Survival (PFS) at One Year: 51%.
The one-year PFS data is particularly significant, as durability past six months has been a key benchmark that many previous allogeneic therapies failed to meet. “It’s been a tough period of time for the broader allo-CAR T space. We readily acknowledge that,” said Caribou CEO Rachel Haurwitz. “We have the blueprint for allo-CAR Ts.”
Optimized Strategy and Funding Gap
Caribou credits its success to using CRISPR gene editing to “armor” its cell therapy against rejection, coupled with the adoption of partial HLA matching (a measure typically used in organ transplants) and the selection of cells from younger donors (under age 30). Moving forward, the company plans to use an “optimized” criterion in future trials, including young donors and at least a two-point HLA match.
Despite the clinical momentum, CEO Haurwitz confirmed the company does not have the necessary funds to launch a pivotal trial for CB-010. “We do not have the resources on our balance sheet to fully fund this trial today,” Haurwitz said, adding that the company is exploring “a multitude of options,” ranging from an equity capital raise to a partnership. This news follows earlier staff reductions and pipeline cuts made by Caribou this year.
First Data for Multiple Myeloma Candidate
Caribou also unveiled initial data for its off-the-shelf therapy targeting multiple myeloma, CB-011. At the selected dose for expansion, 11 of 12 patients responded to treatment, with nine achieving a complete response. The company reported the therapy was generally well-tolerated, noting no cases of graft-versus-host disease (GvHD), though one patient death was reported due to blood toxicity related to CB-011.
Caribou plans to advance its dose expansion study for the multiple myeloma candidate, a trial for which it currently has sufficient funding.
Source:
https://endpoints.news/caribou-says-its-cracked-off-the-shelf-cell-therapy-but-needs-to-raise-money-to-move-forward/?utm_source=dlvr.it&utm_medium=linkedin
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