Viral vector CDMO Genezen has announced a strategic partnership with Japanese biotech firm Optieum Biotechnologies to support the cGMP manufacturing of lentiviral vector (LVV) constructs for OPTF01, a novel chimeric antigen receptor (CAR)-T cell therapy being developed for glioblastoma treatment.
The collaboration, announced on May 5, 2025, will leverage Genezen's expertise in viral vector manufacturing to advance Optieum's innovative therapy toward clinical development. Under the agreement, Genezen will handle technology transfer, process development, and cGMP manufacturing of the LVV construct essential for producing the OPTF01 CAR-T product.
A Novel Approach to Glioblastoma Treatment
Glioblastoma (GBM) is an aggressive form of brain cancer that primarily affects older adults, with current treatments offering limited efficacy in slowing disease progression and improving survival rates. OPTF01 represents a potential breakthrough in addressing this challenging malignancy.
The therapy is derived from Optieum's proprietary Eumbody System, which received US patent protection earlier this year. What distinguishes OPTF01 from conventional CAR-T approaches is its targeting mechanism. The therapy specifically targets Fibroblast Activation Protein-alpha (FAP-α), which is expressed not only on tumor cells but also on surrounding pericytes and cancer-associated fibroblasts (CAFs) within the tumor microenvironment.
"OPTF01 targets FAP-α, leveraging its broad expression on cells within the tumor microenvironment to drive therapeutic activity beyond direct tumor cell engagement," explained Dan Macleod, CSO at Optieum. "Solid tumors such as GBM often evade immune surveillance by creating a protective, immunosuppressive environment, limiting the effectiveness of current CAR-T and other immunotherapies."
This dual-targeting approach could potentially disrupt the immunosuppressive microenvironment while simultaneously attacking malignant cells, addressing a fundamental challenge in solid tumor immunotherapy.
Financial Backing and Development Timeline
Prior to the Genezen partnership, Optieum secured substantial financial support for OPTF01's development. The company received $39 million in funding from the Japan Agency for Medical Research and Development (AMED), highlighting the therapy's potential significance in addressing an unmet medical need.
While the therapy remains in preclinical development, the manufacturing partnership represents a critical step toward clinical trials. The collaboration aims to accelerate progress toward delivering next-generation therapies for glioblastoma and potentially other solid tumors.
The Eumbody System Platform
OPTF01 is built on Optieum's Eumbody System, a proprietary platform that represents a significant advancement in CAR-T cell therapy development. The system employs rapid and expansive functional screening to identify and optimize CAR constructs.
The platform works by dynamically harmonizing single-chain variable fragment (scFv) sequences to enhance the functional capabilities of T cells. This approach allows for more precise targeting and potentially improved efficacy compared to conventional CAR-T designs.
Manufacturing Expertise and Global Reach
Genezen brings over a decade of experience in gene therapy manufacturing to the partnership, with state-of-the-art viral vector facilities capable of supporting projects from concept to commercialization.
"Genezen is honored to support Optieum's best-in-class CAR-T programs with our best-in-class LVV expertise to bring these critical therapies to life," said Steve Favaloro, Chairman and CEO of Genezen. "This collaboration with Optieum underscores our rich experience and the capabilities of both our people and state-of-the-art facilities to support customers on a global scale – now including a leading Japanese biotech."
The partnership highlights Genezen's expanding global footprint and ability to support international biotech companies in advanced therapy development.
Addressing Critical Unmet Needs
For patients with glioblastoma, the development of effective therapies represents a critical unmet need. Current treatment options are limited and typically offer poor prognoses.
Shun Nishioka, CEO of Optieum, emphasized the company's mission: "At Optieum, we are committed to redefining the future of CAR-T therapy through relentless innovation and scientific rigor. Partnering with Genezen's team of experts ensures that critical materials for our groundbreaking therapies are manufactured to the highest standards, accelerating our progress toward delivering next-generation therapies for glioblastoma and other solid tumors."
If successful in clinical development, OPTF01 could potentially transform the treatment landscape for glioblastoma patients and establish a new paradigm for targeting solid tumors with CAR-T therapy.
