BlueSphere Bio Partners with NMDP BioTherapies℠ for TCR-based Product in Allogeneic Cell Transplant Trial

BlueSphere Bio, a company specializing in developing novel T-cell receptor (TCR) based therapies for both oncology and non-oncology applications, has announced a significant collaboration with NMDP BioTherapies. This partnership aims to push forward BlueSphere’s first clinical trial, which will test an innovative cell therapy designed for patients with high-risk leukemias. The focus is on acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and myeloid dysplastic syndrome (MDS).

Under the collaboration, NMDP BioTherapies will provide BlueSphere with hematopoietic stem cells from healthy donors who are HLA-matched. These stem cells will comply with the stringent requirements of the Food and Drug Administration (FDA) and BlueSphere's own eligibility criteria. These cells are critical for BlueSphere’s upcoming Phase 1/2a trial (TCX-101), which has received FDA clearance. The trial will investigate the efficacy of BlueSphere's first-in-human product candidate, BSB-1001. This TCR T-cell therapy product will be administered alongside allogeneic stem cell transplants to patients with relapsed or refractory leukemia, thereby targeting residual cancer cells and aiming to reduce relapse rates and the incidence of graft versus host disease (GvHD).

Dr. Steve Devine, Chief Medical Officer at NMDP, expressed enthusiasm about the partnership, highlighting the potential of BSB-1001 to offer a novel and effective treatment for leukemia patients. The therapy aims to minimize the severe side effects associated with standard allogeneic hematopoietic stem cell transplantation (alloHSCT), particularly GvHD, where donor T cells can attack the patient’s healthy cells.

BSB-1001 is engineered to target the blood-restricted antigen HA-1, which allows it to selectively kill leukemia cells without harming healthy tissues. This specificity is expected to reduce the risk of GvHD while potentially maximizing the graft versus leukemia effect. The TCX-101 trial is unique in its design, focusing on patients with active disease or at high risk of relapse, and administering BSB-1001 simultaneously with alloHSCT to target residual leukemia cells effectively.

NMDP BioTherapies will not only provide the necessary stem cells but will also leverage their expertise in bioinformatics to identify suitable donors. They will manage the entire supply chain logistics, from donor consent to the delivery of stem cells to BlueSphere’s clinical sites, ensuring compliance with regulatory standards.

Keir Loiacono, CEO of BlueSphere Bio, emphasized the critical role of NMDP BioTherapies in providing the resources and support needed for the trial. He expressed confidence that the partnership would facilitate the delivery of this promising therapy to leukemia patients who have exhausted other treatment options. BlueSphere plans to enroll the first patient in the trial by the fourth quarter of 2024.

NMDP BioTherapies is known for its comprehensive cell and gene therapy solutions, backed by decades of industry experience and a robust infrastructure. They operate the world’s most diverse registry of potential blood stem cell donors, comprising over 7 million individuals. Their services extend from donor identification and stem cell collection to long-term follow-up, ensuring the successful delivery and efficacy of cell therapies worldwide.

BlueSphere Bio, founded by UPMC Enterprises, has developed a proprietary TCR discovery platform called TCXpress™, which enables rapid and efficient identification of TCRs for various clinical applications. Initially focusing on oncology, BlueSphere is building a portfolio of TCR-based therapies, with BSB-1001 being the first clinical candidate under the TCX-101 program. The company plans to expand its pipeline further, including a new program, TCX-102, targeting another subset of AML patients, with an IND expected by mid-2025.

Additionally, BlueSphere is collaborating with the National Cancer Institute on a novel TCR T-cell therapy for recurrent respiratory papillomatosis (RRP), and is developing a broad panel of TCRs for its first solid tumor program, set to be announced later this year.