Caribou Biosciences (NASDAQ:CRBU) executives used Citi’s Virtual Oncology Summit for 2026 to outline the company’s strategy for bringing off-the-shelf, allogeneic CAR-T cell therapies to broader populations of patients with hematologic cancers. President and CEO Rachel Haurwitz and CFO Sri Ryali discussed the company’s CRISPR platform, key clinical learnings from its two lead programs, and plans for a pivotal study in second-line large B cell lymphoma (LBCL) that management said will require additional capital to fund through a progression-free survival (PFS) readout.
Core technology and pipeline focus
Haurwitz said Caribou’s foundation is its chRDNA genome-editing platform, which she described as offering “orders of magnitude” greater specificity than first-generation CRISPR-Cas9. Caribou has focused that technology on developing allogeneic CAR-T therapies intended to be delivered “off the shelf,” with two programs currently in the clinic for blood cancers.
- Vispa-cel, an off-the-shelf CAR-T program being developed in second-line LBCL. Haurwitz said Caribou has concluded a phase 1 study and described the program as “pivotal-ready.”
- CB-011, an off-the-shelf BCMA-targeted CAR-T therapy for multiple myeloma being studied in a phase 1 trial called CaMMouflage.
Why allogeneic CAR-T: access, speed, and community care
She attributed the gap largely to (1) patients who cannot wait “four, six, eight, 12 weeks” to receive a dose and (2) geographic access, noting that many patients are treated in community settings while autologous CAR-Ts are typically available at top academic centers. Haurwitz said Caribou is exploring a pivotal design for vispa-cel that would include community hospital sites, which she argued could support a future commercial footprint spanning both community and academic settings.
Vispa-cel: phase 1 dataset and pivotal trial concept
Haurwitz said Caribou had enrolled 84 patients in the phase 1 ANTLER study of vispa-cel as of a data cutoff near the end of 2025, beginning with late-line B cell non-Hodgkin lymphoma histologies before proposing to the FDA a shift into second-line LBCL. She said the FDA “green-lit” that approach. Across Caribou’s programs, she added, the company has dosed more than 140 patients with its allogeneic CAR-T therapies, building what she characterized as one of the larger clinical translational datasets in the allogeneic CAR-T field.
Two translational learnings were highlighted as central to Caribou’s “optimized” go-forward strategy for vispa-cel:
- Donor age: Haurwitz said donors aged 18–29 were associated with more durable outcomes than donors aged 30 and older, and that the company has applied this approach across manufacturing for both vispa-cel and CB-011.
- Modest HLA matching: She said Caribou found that HLA matching mattered, but that the required level was “incredibly modest”—two matched alleles out of 12 HLA class I and II alleles. She said Caribou’s supply plan could cover 99% of patients with at least two matched alleles using 10 donors (10 lots), with an expected average of seven alleles matched.
Haurwitz pointed to a 35-patient “optimized profile” cohort (young donor material plus at least two matched HLA alleles) with a median follow-up of “almost a year.” She said overall response rate and complete response rate were “bang on” with autologous CAR-T outcomes in published pivotal studies, and that 12-month PFS was “north of 50%,” also aligning with autologous CAR-T benchmarks. She added that the cohort includes patients followed for more than three years who have maintained complete responses after a single dose.
On the planned pivotal trial, Haurwitz said Caribou is engaging with the FDA on a randomized controlled study of approximately 250 patients in second-line LBCL focused on patients who are not eligible for autologous CAR-T and not eligible for autologous stem cell transplant. She cited an estimate from Headland Strategy that about 60% of the second-line population is “dual ineligible,” largely due to the need for urgent therapy. She said the company has discussed PFS as an event-driven endpoint with the agency and is continuing discussions on the specific standard-of-care regimens to include in the control arm, citing examples such as Pola-BR, R-GDP, and R-GemOx.
Timeline and financing considerations
Ryali said trial initiation depends on completing ongoing, iterative discussions with the FDA, including finalizing control-arm regimen options and other trial elements. He also said Caribou will need additional capital to fund the pivotal study through data readout. Assuming these items come together in the first half of 2026, he said the company could initiate the pivotal trial “shortly thereafter” and remain on track for a PFS data readout sometime in 2028.
Haurwitz also said Caribou plans additional analyses of ANTLER, including an update at a medical meeting later in 2026.
CB-011 in multiple myeloma: dose escalation learnings and next steps
Turning to CB-011, Haurwitz said key opinion leaders have framed bispecific antibodies as the benchmark for an off-the-shelf therapy in multiple myeloma, particularly given the “one-and-done” appeal of CAR-T versus repeated treatment burden and infection challenges associated with bispecifics.
She said the CaMMouflage phase 1 dose escalation enrolled 48 patients, testing two lymphodepletion (LD) regimens and multiple CAR-T doses. The LD regimens varied cyclophosphamide (Cy) dosing from 300 mg/m² to 500 mg/m² (with a standard fludarabine dose). At the lower Cy level, she said only one of 13 patients showed CAR-T expansion and persistence, and that patient remained in stringent complete response with MRD negativity 21 months later. Caribou then moved to the 500 mg/m² Cy regimen, which she said “unlocked” stronger activity.
Based on efficacy, Caribou selected a dose of 450 million CAR-T cells with 500 mg/m² Cy as the recommended dose for expansion. In 12 BCMA-naïve patients treated at that regimen as of the 2025 data cut, Haurwitz reported:
- Overall response rate (ORR): 92%
- CR/sCR rate: 75%
- MRD negativity rate: 91%
She said the expansion cohort can enroll up to 30 patients and that Caribou plans to fill the cohort to better define the dataset. The company has guided to two CB-011 updates in 2026: an initial look at dose expansion data and longer follow-up on dose escalation.
Haurwitz also referenced translational data presented at the ASTCT/CIBMTR Tandem Meeting, describing a high proportion of central memory T cells at peak and noting that the phenotypic profile was described as similar to published translational data for CARVYKTI.
On registrational strategy for CB-011, Haurwitz said there are “many potential paths forward,” ranging from broad development aimed at larger populations to more focused approaches such as extramedullary disease or relapse after prior BCMA-targeted therapy. She said the company plans initial FDA discussions later in 2026.
Finally, asked about commercialization, Haurwitz said Caribou is preparing to run the vispa-cel pivotal study itself and aspires to launch vispa-cel commercially in the U.S., while expecting that partnerships would be “mission-critical” for broader global reach and could also be considered in the U.S. to accelerate or expand reach.
About Caribou Biosciences (NASDAQ:CRBU)
Caribou Biosciences, Inc is a clinical-stage biopharmaceutical company that leverages its proprietary CRISPR-Cas gene-editing platform to develop transformative cell therapies and in vivo treatments for a range of cancers and genetic diseases. The company’s core technology enables precise modification of cellular genomes, allowing the design of engineered T-cell and NK-cell therapies aimed at improving safety, efficacy and persistence in patients with hematologic and solid tumor malignancies. Alongside its oncology portfolio, Caribou is advancing in vivo editing programs targeting monogenic disorders, with initiatives in areas such as Duchenne muscular dystrophy and familial amyloidosis.
Established in 2011 and headquartered in Berkeley, California, Caribou Biosciences was co-founded by Nobel laureate Jennifer Doudna, one of the pioneers of CRISPR gene-editing technology.
