1
Country: USA | Funding: $3.4B
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
2
Country: USA | Funding: $1.2B
Kura Oncology, a biopharmaceutical company, advances a pipeline of precision medicines for the treatment of solid tumors and blood cancers.
Kura Oncology, a biopharmaceutical company, advances a pipeline of precision medicines for the treatment of solid tumors and blood cancers.
3
Country: UK | Funding: $1.1B
Autolus uses advanced CAR-T cell therapy to treat hematological malignancies and solid tumors. While CAR T-cell therapy has revolutionized cancer treatment, it can also cause significant immunotoxicity. Autolus has developed a novel CAR-construct with rapid binding kinetics that is similar to natural T cells and provides improved tolerability in patients with certain types of leukemia. Furthermore, Autolus addresses the issue of immunotoxicity through a personalized dosing approach. The company is also seeking to adopt CAR-T therapy to patients with acute lymphoblastic leukemia, acute myeloid leukemia, chronic lymphocytic leukemia, T-cell lymphoma and solid tumors. It is supporting several ongoing CAR-T clinical trials that are opening up new treatment options and offering new hope for these patients.
Autolus uses advanced CAR-T cell therapy to treat hematological malignancies and solid tumors. While CAR T-cell therapy has revolutionized cancer treatment, it can also cause significant immunotoxicity. Autolus has developed a novel CAR-construct with rapid binding kinetics that is similar to natural T cells and provides improved tolerability in patients with certain types of leukemia. Furthermore, Autolus addresses the issue of immunotoxicity through a personalized dosing approach. The company is also seeking to adopt CAR-T therapy to patients with acute lymphoblastic leukemia, acute myeloid leukemia, chronic lymphocytic leukemia, T-cell lymphoma and solid tumors. It is supporting several ongoing CAR-T clinical trials that are opening up new treatment options and offering new hope for these patients.
4
Country: Germany | Funding: $748.1M
MorphoSys' mission is to make exceptional, innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Our focus is on cancer.
MorphoSys' mission is to make exceptional, innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Our focus is on cancer.
5
Country: USA | Funding: $622M
Arcellx develops intelligent and adaptive cell therapies to treat the complexity of human disease. It intends to provide cancer patients with first-in-class adaptive immune cell therapies that are readily silenced, activated, and reprogrammed throughout the course of disease.
Arcellx develops intelligent and adaptive cell therapies to treat the complexity of human disease. It intends to provide cancer patients with first-in-class adaptive immune cell therapies that are readily silenced, activated, and reprogrammed throughout the course of disease.
6
Country: China | Funding: $504.7M
Ascentage Pharma is a China-based, global-oriented, clinical-stage biopharmaceutical company, dedicated to discovery and development of the “first-in-class” and the “best-in-class” targeted small-molecule cancer therapeutics.
Ascentage Pharma is a China-based, global-oriented, clinical-stage biopharmaceutical company, dedicated to discovery and development of the “first-in-class” and the “best-in-class” targeted small-molecule cancer therapeutics.
7
Country: USA | Funding: $460.5M
Poseida Therapeutics develops differentiated allogeneic cell therapies and genetic medicines using our proprietary genetic engineering platforms. The company develops the piggyBac non-viral DNA delivery system (which enables introduction of large genetic data into the genome) and the Cas-CLOVER site-specific gene editing system (which can be used for precise site-specific deletions, insertions and knockouts in various cell types). Using this platform, the company creates CAR-T therapies with high levels of desired TSCM (stem cell-derived memory T cells) for the treatment of a range of oncological, autoimmune and rare diseases, including multiple myeloma, acute myeloid leukemia and prostate cancer. Poseida was acquired by Roche in early 2025.
Poseida Therapeutics develops differentiated allogeneic cell therapies and genetic medicines using our proprietary genetic engineering platforms. The company develops the piggyBac non-viral DNA delivery system (which enables introduction of large genetic data into the genome) and the Cas-CLOVER site-specific gene editing system (which can be used for precise site-specific deletions, insertions and knockouts in various cell types). Using this platform, the company creates CAR-T therapies with high levels of desired TSCM (stem cell-derived memory T cells) for the treatment of a range of oncological, autoimmune and rare diseases, including multiple myeloma, acute myeloid leukemia and prostate cancer. Poseida was acquired by Roche in early 2025.
8
Country: USA | Funding: $414.8M
Abcuro develops first-in-class immunotherapies to treat autoimmunity and cancer.
Abcuro develops first-in-class immunotherapies to treat autoimmunity and cancer.
9
Country: France | Funding: $393.5M
Cellectis has developed genome editing technology TALEN, which enables it to create more affordable, ready-to-use allogeneic CAR T-cell therapies. Its technology offers protection against graft-versus-host disease, reduces the risk of rejection and improves safety by integrating a self-destruct mechanism. Using this technology, the company is developing a pipeline of several candidates against non-Hodgkin's lymphoma, large B-cell lymphoma, renal cell carcinoma and melanoma. Cellectis is also advancing PulseAgile technology, which uses electroporation (controlled electric fields) to deliver messenger RNA molecules into cells. It utilizes a unique electric field waveform, which, combined with a proprietary buffer solution, allows molecules such as nucleases to effectively penetrate cells while maintaining high cell viability.
Cellectis has developed genome editing technology TALEN, which enables it to create more affordable, ready-to-use allogeneic CAR T-cell therapies. Its technology offers protection against graft-versus-host disease, reduces the risk of rejection and improves safety by integrating a self-destruct mechanism. Using this technology, the company is developing a pipeline of several candidates against non-Hodgkin's lymphoma, large B-cell lymphoma, renal cell carcinoma and melanoma. Cellectis is also advancing PulseAgile technology, which uses electroporation (controlled electric fields) to deliver messenger RNA molecules into cells. It utilizes a unique electric field waveform, which, combined with a proprietary buffer solution, allows molecules such as nucleases to effectively penetrate cells while maintaining high cell viability.
10
Country: USA | Funding: $272.5M
Senti is using the latest techniques in the fields to build the future of gene and cell-based therapies. Senti’s proprietary synthetic biology platform provides fundamental advantages for therapeutics development.
Senti is using the latest techniques in the fields to build the future of gene and cell-based therapies. Senti’s proprietary synthetic biology platform provides fundamental advantages for therapeutics development.
11
Country: USA | Funding: $146.5M
Imago BioSciences is working on a treatment that shuts down an enzyme important to blood cancer growth.
Imago BioSciences is working on a treatment that shuts down an enzyme important to blood cancer growth.
12
Country: USA | Funding: $135.7M
Amphivena Therapeutics develops treatments to address hematologic malignancies.
Amphivena Therapeutics develops treatments to address hematologic malignancies.
13
Country: France | Funding: €101.6M
MaaT Pharma (Microbiota as a Therapy) is intended to treat serious diseases linked to imbalances in the intestinal microbiome. It has developed the first treatment solution based on autologous microbiotherapy. MaaT Pharma envisages a first therapeutic application for patients suffering from leukaemia and bone & joint infections, whose major treatment contributes to dysbiosis.
MaaT Pharma (Microbiota as a Therapy) is intended to treat serious diseases linked to imbalances in the intestinal microbiome. It has developed the first treatment solution based on autologous microbiotherapy. MaaT Pharma envisages a first therapeutic application for patients suffering from leukaemia and bone & joint infections, whose major treatment contributes to dysbiosis.
14
Country: China | Funding: $101.6M
Juventas is a provider and developer of innovative immune cell therapeutic drugs.
Juventas is a provider and developer of innovative immune cell therapeutic drugs.
15
Country: Israel | Funding: $59M
BioSight is a biopharmaceutical company revolutionizing the research and development of innovative cancer targeted pro-drugs. BioSight has developed technology S2DOT TM and a pipeline of targeted pro-drugs, that can target and release chemotherapy drugs inside cancer cells, while significantly minimizing the systemic toxicity associated with conventional chemotherapy treatments.
BioSight is a biopharmaceutical company revolutionizing the research and development of innovative cancer targeted pro-drugs. BioSight has developed technology S2DOT TM and a pipeline of targeted pro-drugs, that can target and release chemotherapy drugs inside cancer cells, while significantly minimizing the systemic toxicity associated with conventional chemotherapy treatments.
16
Country: USA | Funding: $15M
Kurome Therapeutics is developing therapies that target cancer cells that have co-opted immune signaling pathways in order to avoid destruction by traditional therapeutic agents and subvert adaptive resistance mechanisms. The first goal is to to treat acute myeloid leukemia (AML) by targeting adaptive resistance mechanisms.
Kurome Therapeutics is developing therapies that target cancer cells that have co-opted immune signaling pathways in order to avoid destruction by traditional therapeutic agents and subvert adaptive resistance mechanisms. The first goal is to to treat acute myeloid leukemia (AML) by targeting adaptive resistance mechanisms.
17
Country: Poland | Funding: $3.25M
Selvita is a drug discovery company engaged in the research and development of breakthrough therapies in the area of oncology.
Selvita is a drug discovery company engaged in the research and development of breakthrough therapies in the area of oncology.
