Umoja Biopharma Publishes Success in Generating In Vivo CAR T-cells in Primates

Umoja Biopharma, Inc., an innovative immunotherapy company, has made significant strides in the field of CAR T-cell therapies with its VivoVec in vivo CAR T delivery system. Recently, the company published groundbreaking pre-clinical data in Blood, the American Society of Hematology's flagship journal. This study indicates that VivoVec lentiviral particles can effectively generate CAR positive T (CAR+ T) cells targeting CD20 in vivo, leading to the extensive and durable depletion of B cells for over 10 weeks without the need for lymphodepleting chemotherapy.

Key Findings and Advancements:
- The study revealed that VivoVec particles (VVPs) incorporating CD80 and CD58 costimulation proteins have an enhanced capacity for in vivo CAR T-cell generation, producing CAR T-cells with superior antitumor functionality.
- A combination of anti-CD3 scFv with the ligand-binding domains of CD80 and CD58 into a multi-domain fusion (MDF) protein significantly improved the particles’ ability to bind, activate, and transduce T-cells.
- VVPs using this MDF approach generated anti-CD20 CAR T-cells in non-human primates, comprising up to 65% of circulating T-cells and achieving complete B-cell depletion for up to 76 days.

These findings are a promising development for CAR T-cell therapies, offering a potential paradigm shift by eliminating the need for extensive infrastructure and the lengthy process of ex vivo cell modification. Currently approved CAR T-cell therapies require complex logistics, including apheresis facilities and the manufacturing of patient-specific CAR T-cells, which limits their accessibility and adoption. By contrast, Umoja's VivoVec platform aims to simplify and streamline this process.

Dr. Byoung Ryu, Executive Vice President of Vector Biology and In Vivo Sciences at Umoja, expressed optimism about these results and the upcoming human trials. He noted that the VivoVec platform could address significant challenges in current CAR T-cell therapies by enabling direct in vivo generation of CAR+ T-cells, thus removing the need for external cell collection and modification.

Dr. Christopher Nicolai, the lead author of the publication, highlighted the data's role in validating the VivoVec platform. He emphasized its ability to generate CAR+ T-cells directly within the body, avoiding the extensive testing and genetic modification typically required.

The VivoVec platform employs advanced lentiviral particles engineered with a multidomain fusion protein designed to bind, activate, and transduce T-cells, resulting in CAR expression. The CAR transgene reprograms the T-cells to target specific cancer cells, potentially transforming the current CAR T-cell therapy landscape.

The data collection for this study was conducted in collaboration with the Washington National Primate Research Center. Umoja plans to commence first-in-human trials for VivoVec-generated candidates in the latter half of 2024.

Umoja Biopharma is committed to developing off-the-shelf therapeutics that enhance the reach, effectiveness, and accessibility of CAR T-cell therapies for both cancer and autoimmune diseases. The company’s state-of-the-art lentiviral vector development and manufacturing facility in Louisville, Colorado, supports the VivoVec technology. Umoja’s approach aims to enable a patient’s immune system to combat disease more effectively, potentially offering broader access to advanced immunotherapies and improving patients' quality of life.

This promising progress marks a significant step forward in CAR T-cell therapy, with VivoVec potentially overcoming many of the logistical and manufacturing challenges associated with traditional methods. As Umoja moves towards clinical trials, the new data provides a strong foundation for the future application of their innovative technology in human medicine.