CRISPR Therapeutics Reports Progress Across Gene Editing Pipeline

CRISPR Therapeutics outlined continued momentum across its gene editing and cell therapy programs, with several candidates advancing in cardiovascular disease, autoimmune disorders, oncology, and regenerative medicine.

The company is steadily building out its in vivo gene editing portfolio using its lipid nanoparticle delivery platform. In cardiovascular disease, CTX310 has shown consistent and lasting reductions in key lipid measures following a single intravenous treatment, alongside a safety profile that supports further development. Based on these results, the program has moved into a Phase 1b study, with an initial focus on patients facing severe lipid conditions that remain difficult to treat.

Progress continues in additional cardiovascular programs. CTX320 delivered meaningful reductions in lipoprotein(a) levels during early clinical testing. At the same time, CRISPR Therapeutics is preparing a next-generation candidate, CTX321, which demonstrated higher potency in preclinical studies while relying on the same delivery system. That program is now undergoing the studies needed before entering the clinic.

Beyond these efforts, the company is advancing earlier-stage candidates aimed at genetic liver disease and treatment-resistant hypertension, extending its in vivo platform into additional therapeutic areas.

In cell therapy, zugocabtagene geleucel (zugo-cel) remains a central focus. Clinical trials are ongoing across several autoimmune diseases, with early patients showing sustained disease control and prolonged immune effects following treatment. Development is also continuing in blood cancers, supported by an ongoing Phase 1/2 study. To broaden the oncology program, the company has entered into a collaboration with Eli Lilly to evaluate zugo-cel alongside an established targeted therapy in aggressive B-cell lymphomas.

The update also highlighted progress in regenerative medicine, including diabetes. Data from an earlier program demonstrated continued biological activity one year after implantation, helping shape the company’s next steps in hypoimmune cell engineering. That work has led to the selection of CTX213, which has produced strong preclinical results and is moving toward clinical evaluation.

Taken together, the update reflects a pipeline that is advancing on multiple fronts, with programs generating data, entering new trial phases, and laying the groundwork for future clinical milestones across a range of serious diseases.