Caribou Biosciences, Inc., a clinical-stage CRISPR genome-editing biopharmaceutical company, announced the FDA's clearance of its Investigational New Drug (IND) application for CB-010. This allogeneic anti-CD19 CAR-T cell therapy, featuring a PD-1 knockout, is aimed at treating lupus nephritis (LN) and extrarenal lupus (ERL). The GALLOP Phase 1 clinical trial, a multicenter, open-label study, is expected to start by the end of 2024.
CB-010 has shown promising initial safety and efficacy in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL), leading to its expansion into autoimmune diseases. Rachel Haurwitz, PhD, Caribou’s president and CEO, highlighted the potential of CB-010 to improve the standard of care for lupus patients by targeting CD19-positive B cells involved in autoantibody production and autoimmune response perpetuation.
The ongoing ANTLER Phase 1 trial continues to enroll patients with large B cell lymphoma (LBCL), with initial dose expansion data to be presented in Q2 2024. CB-010's development is supported by its unique chRDNA technology, allowing for precise CAR insertion into the T cell genome, and its potential for deep depletion of disease-causing B cells, offering a path to sustained drug-free remission.
Lupus, a chronic autoimmune disease, affects approximately 320,000 patients in the US, with about 50% developing lupus nephritis. CB-010's approach, including partial HLA matching between donors and patients, aims to improve clinical outcomes by reducing therapy rejection and enhancing the persistence of therapeutic activity.
Caribou Biosciences is advancing a pipeline of off-the-shelf cell therapies from its CAR-T platform, aiming to provide readily available treatments for patients with hematologic malignancies and autoimmune diseases. The company's financial resources, including $372.4 million in cash, cash equivalents, and marketable securities as of December 31, 2023, are expected to fund operations into Q1 2026.
