Biotech company Cellectis has announced a strategic shift in its CAR-T therapy pipeline, choosing to deprioritize UCART123, a leukemia-targeted candidate, to focus resources on two other promising therapies.
UCART123, which targets CD123, was under evaluation in a phase 1 trial for patients with relapsed or refractory acute myeloid leukemia. While the study has yielded valuable insights into CD123-targeted CAR-T therapy’s role in treating this aggressive cancer, Cellectis has decided to halt further development on UCART123. “The findings have contributed meaningfully to our understanding and future platform development,” the company noted in its Q3 earnings release on October 4.
The biotech will now focus its efforts on two CAR-T therapies advancing in clinical stages: UCART22, aimed at relapsed or refractory B-cell acute lymphoblastic leukemia, and UCART20x22, a dual-target CAR-T designed to tackle CD20 and CD22 proteins in B-cell non-Hodgkin lymphoma. UCART22 is currently in a phase 1/2a study with data expected next year, while UCART20x22 is in early-stage evaluation. Cellectis anticipates releasing data from both studies in 2025 and plans to outline its strategy for late-stage development at that time.
Beyond its internal pipeline, Cellectis is actively collaborating with major pharmaceutical companies to expand its reach in cell and gene therapy. The biotech has partnered with Servier and Allogene on a phase 2 study of an anti-CD19 CAR-T for large B-cell lymphoma. In addition, a partnership with AstraZeneca is leveraging Cellectis’ gene editing technology to pursue CAR-T therapies for both blood cancers and solid tumors, as well as an in vivo gene therapy for a genetic disorder.
Cellectis remains optimistic about the progress of its clinical trials and strategic partnerships. “We are excited to advance our ambition in cell and gene therapy, particularly through our collaboration with AstraZeneca, which aims to bring potentially lifesaving therapies to patients with significant unmet needs,” said André Choulika, CEO of Cellectis.
As of the end of September, Cellectis reported $264 million in cash reserves, including a recent $140 million equity investment from AstraZeneca linked to their collaboration. The company projects that this funding will sustain its operations through 2027.