BRAIN Biotech and TransCode Therapeutics join forces to develop a CRISPR-derived technology platform for cancer treatment

Development of a CRISPR-derived technology platform for cancer treatment

06-Mar-2023 - Germany

BRAIN Biotech AG announced the signing of a joint development agreement (JDA) with TransCode Therapeutics, Inc. The objective of the JDA is to co-develop a platform technology that combines a Class 2 CRISPR nuclease, the cell-killing G-dase E, developed by BRAIN Biotech's Akribion Genomics unit with TransCode's TTX nucleic acid delivery platform for the treatment of cancer.

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TransCode’s proprietary TTX platform is designed to enable systemic delivery of targeted nucleic acid-based therapeutics to tumors and metastases. Akribion Genomics’ proprietary nuclease is designed to seek out selective genomic characteristics based on the existence of specific RNA biomarkers within target cells. Combining these technologies could unlock the potential of CRISPR-like cell targeting approaches for the treatment of cancer.

In addition, the parties signed a memorandum of understanding to jointly commercialize the combined technologies in the form of joint out-licensing agreements. “Specific depletion of cancer cells based on RNA biomarkers is a novel and powerful approach,” states Dirk Sombroek, PhD, member of the Akribion Genomics team of BRAIN Biotech AG. “The partnership with Transcode and a joint development could unleash the potential of our proprietary nucleases for therapeutic applications.”

Lukas Linnig, lead of Akribion Genomics activities at BRAIN Biotech AG, emphasizes: “Our ultimate goal is to develop the combined technologies into a technology platform that provides a basis for the development of drugs to treat cancer. This partnership with TransCode is therefore an important step on our challenging path to enter the field of cancer therapeutics with a new class of cancer treatment.

Zdravka Medarova, PhD, Co-founder and CTO of TransCode, comments: “Cell targeting technology based on genomic characteristics holds tremendous potential for the treatment of cancer. However, to fulfill the promise of a Class 2 CRISPR toolbox in oncology, it is critical to achieve highly specific and targeted delivery to tumor cells. This capability relies on having a safe and effective delivery vehicle, such as TransCode’s TTX is expected to represent, and a very precise cell-depletion tool, such as BRAIN Biotech’s G-dase E nucleases is believed to be.” She adds: “Combining these technologies could enable the development of further CRISPR-derived RNA biomarker targeting drugs effective against previously undruggable but highly impactful therapeutic targets in cancer.”

“This partnership has the potential to enable a functionally unique tool to fight cancer because unlike our other therapeutic candidates, it does not rely on inhibition or enrichment of cancer-relevant genetic targets but, instead, is designed to specifically kill a cell with a critical pathway dysfunction,” comments Michael Dudley, Co-founder, President and CEO of TransCode Dudley adds, “The timing of this partnership is ideal, given that we are in the process of initiating a first-in-human study aimed at demonstrating TTX’s capability of targeting clinical metastases.”

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