Business Overview and History
Caribou Biosciences, Inc. (CRBU) is a leading clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. The company's robust pipeline of allogeneic, or off-the-shelf, cell therapies leverages its proprietary chRDNA technology to enhance the activity and durability of its cell therapy products.
Caribou Biosciences was founded in 2011 and is headquartered in Berkeley, California. The company has built a strong foundation in CRISPR genome-editing technologies, including its novel chRDNA platform, which enables more precise genome editing to develop cell therapies that are armored to improve activity against diseases.
Since its inception, Caribou has devoted substantial resources to organizing and staffing, business planning, raising capital, expanding its genome-editing platform technologies, developing a pipeline of product candidates, and building strategic partnerships. The company has primarily relied on proceeds from the sales of its capital stock to fund its operations, including its initial public offering in 2021, which generated approximately $321 million in net proceeds, and a follow-on public offering in 2023, which generated approximately $134 million in net proceeds. Additionally, Caribou has received revenue from its license and collaboration agreements, as well as proceeds from the sale of shares of Intellia Therapeutics, Inc. common stock.
Financials
Caribou's financial performance has reflected its focus on research and development. The company has incurred operating losses since its inception and had an accumulated deficit of $412.9 million as of September 30, 2024. For the fiscal year 2023, the company reported total revenue of $34.48 million, a net loss of $102.07 million, and an operating cash outflow of $93.29 million. As of September 30, 2024, Caribou had $281.0 million in cash, cash equivalents, and marketable securities, which management expects will fund the current operating plan into the second half of 2026.
For the third quarter of 2024, Caribou reported total revenue of $2.02 million, a net loss of $34.68 million, and an operating cash outflow of $32.66 million. Revenue decreased by 91.5% year-over-year, primarily due to a decrease of $21.5 million in revenue recognized under the now-terminated Collaboration and License Agreement with AbbVie. Net income decreased by $24.68 million year-over-year, driven by increased research and development expenses to advance the company's clinical programs.
For the nine months ended September 30, 2024, the company reported licensing and collaboration revenue of $7.92 million. Research and development expenses were $99.69 million, and general and administrative expenses were $35.97 million for the same nine-month period. Caribou incurred a net loss of $113.61 million for the nine months ended September 30, 2024.
Pipeline and Clinical Programs
Caribou's pipeline is centered around its allogeneic CAR-T cell therapy platform, which includes three lead product candidates:
1. CB-010 (CB-10.0): An allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout, which is being evaluated in the ongoing ANTLER Phase 1 clinical trial for relapsed or refractory large B-cell non-Hodgkin lymphoma (r/r LBCL). The company is currently enrolling patients in the dose expansion phase of the ANTLER trial, including a cohort evaluating the impact of partial HLA matching.
2. CB-011 (CB-11.0): An allogeneic anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach, which is being assessed in the CaMMouflage Phase 1 trial for relapsed or refractory multiple myeloma (r/r MM). Caribou recently implemented a higher lymphodepletion regimen and plans to present initial dose escalation data in the first half of 2025. CB-011 includes both beta-2-microglobulin (B2M) knockout and insertion of a B2M-HLA-E transgene to reduce CAR-T cell rejection.
3. CB-012 (CB-12.0): An allogeneic anti-CLL-1 CAR-T cell therapy with both checkpoint disruption and immune cloaking strategies, which is being studied in the AMpLify Phase 1 trial for relapsed or refractory acute myeloid leukemia (r/r AML).
In addition to its oncology programs, Caribou is expanding the clinical development of CB-010 to include autoimmune diseases. The company plans to initiate the GALLOP Phase 1 trial evaluating CB-010 in patients with lupus nephritis (LN) and extrarenal lupus (ERL) by the end of 2024.
Caribou's product candidates have received various regulatory designations from the U.S. Food and Drug Administration (FDA), including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations, which may help expedite their clinical development and regulatory review.
Collaborations and Partnerships
Caribou has established several licensing and collaboration agreements to advance its pipeline and technology. Notable partnerships include:
- Pfizer Inc.: In June 2023, Pfizer made a $25 million equity investment in Caribou and entered into an Information Rights Agreement, providing Pfizer with certain rights related to the development of Caribou's anti-BCMA CAR-T cell therapy, CB-011.
- Edge Animal Health: Caribou has an Exclusive License Agreement for Veterinary Therapeutics with Edge Animal Health, granting Edge exclusive worldwide rights to Caribou's CRISPR-Cas9 intellectual property in the field of veterinary therapeutics.
- Memorial Sloan Kettering Cancer Center (MSKCC): Caribou has an Exclusive License Agreement with MSKCC, which provides the company with access to know-how, biological materials, and intellectual property related to the development of its allogeneic CAR-T cell therapy programs.
Risks and Challenges
As a clinical-stage biopharmaceutical company, Caribou faces several risks and challenges, including:
- Successful development, regulatory approval, and commercialization of its product candidates - Ability to maintain and expand its intellectual property portfolio - Competition from other companies developing similar technologies and therapies - Potential safety and efficacy issues that may arise during clinical trials - Ability to secure additional funding to support its operations and pipeline advancement
Despite these challenges, Caribou's innovative CRISPR-based technologies, diverse pipeline, and strategic partnerships position the company as a promising player in the rapidly evolving field of genome-editing therapeutics.
Financial Performance and Outlook
Caribou's financial performance has been characterized by significant research and development expenses as the company advances its pipeline of CRISPR-based therapies. The company's net losses have ranged from $67.56 million in 2023 to $102.07 million in 2022, reflecting the capital-intensive nature of clinical-stage biopharmaceutical development.
While Caribou has not provided specific financial guidance, the company's focus on executing its clinical development strategy and advancing its portfolio of novel therapies suggests it will continue to prioritize investment in R&D to drive long-term growth and value creation.
The biotechnology industry, and specifically the CRISPR genome-editing sector, has seen substantial growth in recent years, with a compound annual growth rate (CAGR) of around 20-25% projected over the next 5-10 years as these novel therapies advance through clinical development and reach commercialization.
Liquidity
As of September 30, 2024, Caribou Biosciences maintained a strong liquidity position with $281.0 million in cash, cash equivalents, and marketable securities. This substantial cash reserve is expected to fund the company's current operating plan into the second half of 2026, providing a runway for advancing its clinical programs and supporting ongoing research and development efforts.
The company's debt-to-equity ratio stood at 0.075 as of December 31, 2023, indicating a low level of debt relative to equity. Caribou has no outstanding debt and has access to an at-the-market equity offering program for up to $100 million in additional capital. The company's current ratio and quick ratio both stand at 8.75, suggesting a strong ability to meet short-term obligations.
Conclusion
Caribou Biosciences is a pioneering clinical-stage CRISPR genome-editing company that is leveraging its proprietary technologies to develop a diverse pipeline of allogeneic cell therapies for the treatment of hematologic malignancies and autoimmune diseases. With multiple clinical programs underway, regulatory designations, and strategic partnerships, Caribou is well-positioned to contribute to the advancement of CRISPR-based therapeutics and address significant unmet medical needs. However, as a clinical-stage biopharmaceutical company, Caribou faces inherent risks and challenges that will require careful navigation in the years ahead.
The company's focus on advancing its pipeline of allogeneic CAR-T cell therapies, leveraging its CRISPR genome-editing platform, positions it at the forefront of innovative treatments for various hematological malignancies and autoimmune diseases. As Caribou continues to progress its clinical trials and provide updates throughout 2025, investors and stakeholders will be closely watching for signs of clinical efficacy and potential commercialization prospects for its lead candidates CB-010, CB-011, and CB-012.