Caribou Biosciences (NASDAQ:CRBU) executives outlined the company’s allogeneic CAR T-cell pipeline, upcoming clinical updates and cash runway during a presentation at the H.C. Wainwright BioConnect Conference.
Tina Albertson, chief medical officer of Caribou Biosciences, said the company is advancing two allogeneic CAR T-cell programs: vispa-cel, also known as CB-010, for large B-cell lymphoma, and CB-011, a BCMA-targeted therapy for multiple myeloma. She said Caribou’s next-generation CRISPR-Cas technology is designed to enable more specific editing with fewer off-target effects, allowing the company to make multiple gene edits while maintaining cell integrity.
Allogeneic CAR T Positioning
Albertson said autologous CAR T therapies remain the “gold standard” in second-line large B-cell lymphoma, but she argued that access remains a major challenge. She said 75% of patients either cannot access autologous CAR T or cannot wait for it due to medical ineligibility, geography or financial constraints.
She said allogeneic, off-the-shelf CAR T therapies such as vispa-cel could address patients who need faster treatment or care closer to the community setting. Albertson also said Caribou has learned from treating more than 140 patients that “young donor T cells matter a lot” for durability, noting that both autologous CAR T and in vivo CAR T rely on a patient’s own T cells.
Addressing the emerging in vivo CAR T field, Albertson said the technology remains early and could require additional engineering over the next five to 10 years. She said Caribou believes allogeneic CAR T is “ready to go,” with vispa-cel expected to move toward a pivotal trial this year.
Vispa-cel Data and Pivotal Trial Plans
Albertson highlighted data presented late last year for vispa-cel in 35 patients treated with product made from young donors and partially matched at HLA. She said the therapy showed efficacy and safety “on par with auto CAR T,” including a 12-month progression-free survival rate of 53% and high overall response and complete remission rates.
She also described the safety profile as similar to the best autologous CAR T products, citing low rates of grade 3 cytokine release syndrome and neurotoxicity. Albertson said this profile could support use in community centers, with the added benefit of an off-the-shelf product that does not require manufacturing wait times.
Caribou uses a partial HLA matching strategy for vispa-cel. Albertson said the company expects a modest number of manufacturing lots to cover nearly all patients, estimating that about 10 lots could match more than 99% of patients at two or more HLA alleles. She said matching would occur in the background through a blood test and algorithm, without creating a lag in treatment.
Albertson said updated ANTLER data expected at the European Hematology Association meeting would likely provide additional follow-up on the same patient population, helping “cement” the existing dataset as Caribou prepares for a pivotal study.
The company has reached alignment with the U.S. Food and Drug Administration on a phase 3 trial in patients who cannot access or wait for autologous CAR T and are not eligible for autologous transplant. Albertson said the trial will randomize 250 patients 1:1 to a single dose of vispa-cel or non-curative control regimens. The control arms include polatuzumab-containing regimens with bendamustine and rituximab or R-GemOx, as well as tafasitamab and R-GemOx for patients who previously received polatuzumab in the front line.
CB-011 Expansion in Multiple Myeloma
Albertson also reviewed CB-011, Caribou’s immune-cloaked BCMA-targeted allogeneic CAR T therapy for multiple myeloma. She said Caribou disclosed data last November from 48 patients in a phase 1 study and selected 450 million cells as the recommended expansion dose.
In 12 patients treated at that regimen who had not previously received BCMA-targeted therapy, Albertson said Caribou observed high response and complete remission rates, with 91% achieving minimal residual disease negativity. She said the company is expanding that cohort and has begun treating patients previously exposed to BCMA agents, an area she described as a growing unmet need.
Albertson said bispecific antibodies remain the benchmark for CB-011 in both BCMA-naive and BCMA-exposed populations. She said Caribou believes it has exceeded that bar in BCMA-naive patients, while the appropriate bar for BCMA-exposed patients remains to be determined. She also noted that CB-011 recently received RMAT designation.
At EHA, Albertson said Caribou expects to provide follow-up on dose-escalation patients previously discussed in the fall. Initial expansion data are expected later in the year.
Cash Runway
Sri Ryali, chief financial officer of Caribou Biosciences, said the company ended the first quarter with just under $120 million in total cash. He said Caribou has guided that its cash runway extends into the second half of 2027.
Ryali said the current balance sheet fully funds the CB-011 expansion cohort and allows Caribou to begin critical startup activities for the vispa-cel pivotal trial, ANTLER 3. However, he said the company will need additional funding to fully fund ANTLER 3 through data readout.
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.
