CAR-T Pipeline Focus: Blood Cancers Lead the Way in Development

Chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a transformative force in cancer treatment, particularly within the realm of cell and gene therapies (CGT) (1^✔ ✔Trusted Source
Blood cancers dominate CAR-T pipeline, reveals GlobalData

Go to source). According to a recent analysis by GlobalData, blood cancers overwhelmingly dominate the CAR-T therapy pipeline, with pharmaceutical leaders like Bristol Myers Squibb at the forefront of advancements that are reshaping treatment paradigms for diseases such as B-cell acute lymphocytic leukemia (B-ALL).

Of the 1,729 CAR-T therapies currently in development, 903—roughly 52%—are specifically targeting blood cancers. Lymphomas represent the largest segment within this category, followed by leukemias and multiple myelomas, while solid tumors, though an area of ongoing research, have yet to achieve significant commercial or clinical success with CAR-T treatments.

CAR-T Approvals and the Dominance of Blood Cancer Treatments

CAR-T therapy, which involves genetically engineering T-cells to express chimeric antigen receptors that enable them to precisely target and destroy cancerous cells, has revolutionized the treatment of blood cancers since the first FDA approval in 2017. Currently, 13 CAR-T therapies have received regulatory approval, including Yescarta (axicabtagene ciloleucel) from Gilead Sciences, which gained FDA approval in 2017, and Qartemi (varnimcabtagene autoleucel) from Immuneel Therapeutics, which was approved in India in January 2025. Like all other approved CAR-T therapies, these treatments specifically target blood cancers, reinforcing the dominance of hematologic malignancies in CAR-T development.

Among these, B-cell acute lymphocytic leukemia stands out as the most targeted blood cancer, with 159 CAR-T therapies currently in development. This aggressive cancer, which primarily affects children, has historically been difficult to treat due to frequent relapses and resistance to standard therapies. Jasper Morley, Pharma Analyst at GlobalData, highlights that CAR-T therapies have played a pivotal role in transforming B-ALL treatment, with Novartis’ Kymriah (tisagenlecleucel), which received FDA approval in 2017, demonstrating complete remission in a significant percentage of patients. He further notes that three out of the five FDA-approved therapies for B-ALL are CAR-T treatments, underscoring their growing importance in tackling this challenging disease.

The CAR-T pipeline is also seeing promising developments for regulatory approvals. Chongqing Precision Biotech’s pCAR-19B is currently in pre-registration for B-cell acute lymphocytic leukemia in China, while Curocell’s anbalcabtagene autoleucel is under regulatory review in South Korea for diffuse large B-cell lymphoma.

Bristol Myers Squibb remains the dominant player in the CAR-T landscape with two already marketed products—Abecma (idecabtagene vicleucel) for multiple myeloma and Breyanzi (lisocabtagene maraleucel) for various lymphomas and leukemias—along with 14 additional CAR-T therapies in development for blood cancers. Other key players include Shenzhen Geno-Immune Medical Institute, which is actively developing 14 CAR-T therapies, including eight currently in Phase II trials.

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Morley concludes that CAR-T therapies have fundamentally reshaped the treatment landscape for blood cancers, and with the high volume of pipeline therapies, more approvals are expected in the coming years, particularly for B-ALL and diffuse large B-cell lymphoma.

As pharmaceutical leaders like Bristol Myers Squibb and Shenzhen Geno-Immune Medical Institute continue to push CAR-T innovations forward, the impact of these therapies in blood cancer treatment is expected to grow significantly, offering new hope for patients worldwide.

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Reference:

1. Blood cancers dominate CAR-T pipeline, reveals GlobalData - (https://www.globaldata.com/media/pharma/blood-cancers-dominate-car-t-pipeline-reveals-globaldata/)

Source-Medindia