BlueSphere Bio Partners with National Cancer Institute to Advance Rare Respiratory Disease Treatments

June 04, 2024 — BlueSphere Bio, a T-cell receptor (TCR) T-cell therapy company specializing in innovative therapeutic candidates for hematologic malignancies and solid tumors, has announced a strategic collaboration with the National Cancer Institute (NCI). This collaboration aims to develop a novel TCR T-cell therapy for recurrent respiratory papillomatosis (RRP), a rare orphan disease. 

Under a Cooperative Research and Development Agreement (CRADA), BlueSphere Bio will work with the Center for Cancer Research (CCR) at the NCI. The clinical studies under this CRADA will be led by Dr. Clint Allen and Dr. Scott Norberg at the CCR, NCI. Previously, through a Material Transfer Agreement, BlueSphere gained access to HPV+ tumor samples from the NCI. Utilizing its proprietary high-throughput TCR discovery platform, TCXpress™, BlueSphere identified multiple TCRs that exhibit high affinity for HPV 6 and HPV 11, which are closely associated with RRP.

"This collaboration with the National Cancer Institute is a significant milestone for BlueSphere," stated Keir Loiacono, Chief Executive Officer of BlueSphere. "With the expertise of Drs. Allen and Norberg and the NCI's extensive resources, we are well-positioned to explore the potential of TCRs identified with TCXpress™ as an innovative treatment pathway for RRP. Unlike solid tumors, RRP offers unique challenges and opportunities for therapeutic intervention without the constraints of the tumor microenvironment."

Dr. Norberg added, "This collaboration aims to determine whether TCR-T cell therapy can eliminate the underlying cause of RRP, rather than just treating its symptoms. This novel approach underscores the potential transformative impact this collaboration seeks to achieve."

RRP is commonly caused by HPV strains 6 and 11, leading to benign papillomas in air passages, which can be debilitating and difficult to treat, especially within the lungs. The collaboration will focus on preparing the identified TCRs for clinical studies and investigating their efficacy in early-stage clinical trials targeting RRP. According to the CRADA, the clinical trial may be sponsored either by the NCI or BlueSphere, with BlueSphere providing support and TCR sequences and the NCI handling testing, sequence modifications, process development, manufacturing, and clinical trials.

BlueSphere Bio, founded by UPMC Enterprises, focuses on translational sciences and uses the TCXpress™ platform to isolate and characterize TCRs efficiently. While initially focusing on oncology, BlueSphere aims to expand its platform to other therapeutic areas. The company is developing a portfolio of TCR-based assets with a primary clinical focus on acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and myeloid dysplastic syndrome (MDS). 

Under its TCX-101 program, BlueSphere expects to enroll its first patient in 2024 for treatment with its candidate, BSB-1001, a TCR T-cell therapy targeting the minor histocompatibility antigen-1 (HA-1) for use alongside allogeneic hematopoietic stem cell transplantation (alloSCT). The company has also nominated three additional miHA targeting TCRs for clinical development within the same setting as BSB-1001, enhancing its HLA coverage in hematologic indications.

Additionally, BlueSphere has broadened its AML therapy pipeline with the TCX-102 program, targeting mutant NPM-1. This program will be autologous and not combined with stem cell transplant, with an IND expected in the second quarter of 2025.

Apart from its collaboration with the NCI, BlueSphere is also identifying a broad panel of TCRs for use in its first solid tumor program, which will be announced later this year.

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