Batu Biologics Utilizes CRISPR/cas9 Gene Editing to Modify T Cells

Batu Biologics has recently filed intellectual property and initiated discovery research on a new method of augmenting an anti-tumor immune response through ex vivo manipulation of the patient’s T cells.

“Batu Biologics plans to utilize this groundbreaking technology to support our efforts in harnessing and unleashing the power of the patient’s immune system,” said Samuel C. Wagner, President and CEO of Batu Biologics. “This is another step in the direction of breaking immunological tolerance to cancer.”

The inhibition of popular “immune checkpoints” has proven to be an effective way to potentiate T cell responses as a treatment for multiple solid tumor indications. Blocking these immune checkpoints in vivo has shown significant effectiveness in open label placebo controlled clinical trials, resulting in the FDA approval of Bristol-Myers Squibb’s Nivolumab and Merck’s Pembrolizumab, both antibodies that target Programmed Cell Death 1 (PD – 1). A major drawback of these therapeutics is the high cost, often upward of $120,000 for a single course of treatment. In contrast, Batu Biologics seeks to leverage the established clinical benefit of targeting this pathway by genetically altering the patient’s T cells themselves, thereby reducing the cost of each treatment while seeking rapid FDA development as a minimally manipulated autologous cell therapy.

The Immittam program is designed around the concept of expanding a patient’s T cells ex vivo to clinically relevant numbers and using the CRISPR/cas9 gene-editing platform to silence immune checkpoints in these cells before they are transfused back into the patient. By permanently modifying the patient’s T cells to no longer express these immune checkpoints, the patient’s immune response will have a greater ability to overcome the local defenses and immune suppression of the tumor microenvironment, leading to more powerful and effective anti-tumor responses. Batu Biologics has identified 20 sequences targeting 4 major immune checkpoints using the CRISPR/cas9 gene-editing platform and is currently conducting proof-of-concept experiments in vitro to screen for the best possible drug candidate.

“The cas9 system is a state-of-the-art method of genetically altering a subset of cells, and has several advantages over the traditional mRNA methods, including a higher number of transfected cells and a permanent alteration of their genome,” added Mr. Wagner. “Batu Biologics plans to utilize this groundbreaking technology to support our efforts in harnessing and unleashing the power of the patient’s immune system. This is another step in the direction of breaking immunological tolerance to cancer.”

Batu Biologics is an immuno-oncology company developing novel gene and cellular based therapies for the treatment of cancer. The company has filed an IND application for its lead therapeutic, ValloVax, a multivalent therapeutic vaccine for Non Small Cell Lung Cancer targeting several tumor-angiogenesis associated antigens. ValloVax has demonstrated strong inhibition of tumor growth in several histologically distinct tumor models, and the company is currently raising funds that will enable the completion of a Phase I clinical study. The company maintains research facilities at JLABS, located in the Torrey Pines-area biotech cluster of San Diego.