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Gates Institute Advances Cell and Gene Therapy Programs with New Scientific Advisory Board and Clinical Trials

• The Gates Institute has formed a scientific advisory board to guide cell and gene therapy programs, focusing on patient impact and exploring new platforms. • A Phase 1 clinical trial for pediatric patients with relapsed or refractory pre B-cell acute lymphoblastic leukemia (B-ALL) has enrolled its first patient. • Charles River Laboratories and the Gates Institute have established a CDMO agreement for lentiviral vector development for CAR T-cell therapies. • Researchers at the Gates Institute are exploring novel molecules to target solid tumors with CAR T-cell therapy, supported by Gates Grubstake funding.

The Gates Institute at the University of Colorado Anschutz Medical Campus is making significant strides in cell and gene therapy through strategic collaborations, clinical trials, and innovative research. A newly formed scientific advisory board, comprising five biotechnology experts, will provide strategic and scientific guidance to specific cell and gene therapy programs. This board will focus on enhancing patient impact, prioritizing short- and long-term goals, and exploring novel platforms, complementing the existing Gates Institute Advisory Board.

Clinical Trial Advancement

Navin Pinto, MD, has been appointed as the medical lead at the Gates Institute, where he will oversee clinical trials, leveraging his expertise in chimeric antigen receptor (CAR) T-cell therapy. A Phase 1 study of CAR T-cell therapy for pediatric patients with relapsed or refractory pre B-cell acute lymphoblastic leukemia (B-ALL) at Children’s Hospital Colorado has enrolled its first patient. The Gates Biomanufacturing Facility is producing the CAR T-cell product for this trial, marking the fifth such project supported by the facility at the University of Colorado Anschutz Medical Campus.

CDMO Agreement with Charles River Laboratories

To bolster its manufacturing capabilities, the Gates Institute has entered into a contract development and manufacturing organization (CDMO) agreement with Charles River Laboratories International, Inc. This partnership will enable the Gates Institute to develop Good Manufacturing Practice (GMP)-grade lentiviral vectors (LVVs) for use in novel CAR T-cell therapies targeting hematological cancers. This collaboration aims to streamline the transition from research to clinical application, addressing the challenges inherent in GMP production during early-phase clinical trials.

Targeting Solid Tumors with Novel Molecules

Recognizing the limited success of CAR T-cell therapy in solid tumors due to the lack of suitable cancer-associated antigens, Joshua Thurman, MD, and his team are exploring novel molecules that the immune system uses to tag tumor cells. This research, supported by Gates Grubstake funding, could pave the way for creating CAR T cells that effectively target solid tumors, potentially expanding the applicability of this therapy to a broader range of cancers.

Supporting Regenerative Medicine Research

The Gates Institute continues to support regenerative medicine research through initiatives like the Gates Grubstake Fund, which awarded over $1.4 million to University of Colorado Anschutz researchers in 2023. These awards support investigators developing regenerative medicine-related technologies, fostering innovation and accelerating the translation of research into clinical applications.
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Reference News

[1]
Gates Institute's Top Stories of 2024
news.cuanschutz.edu · Dec 20, 2024

Gates Institute at CU Anschutz had a successful 2024 with groundbreaking research, new scientific advisory board, challe...

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