TransCode Therapeutics, Inc. announced acceptance for publication by Frontiers in Molecular Biosciences of a preclinical study using TransCode's lead therapeutic candidate, TTX-MC138, in glioblastoma multiforme (GBM). The findings of this study support further development of TTX-MC138 for the treatment of GBM by identifying a potentially successful combination treatment protocol using TTX-MC138 with temozolomide (TMZ), currently the standard- of-care therapeutic. Studies such as this further de-risks pipeline that includes multiple RNA approaches including RNAi, PRR, mRNA vaccines and gene editing with CRISPR, all of which utilize TransCode.

TransCode Therapeutics recently received IRB approval for a first-in-human clinical trial with TTX-MC138 in patients with advanced solid cancers. In this clinical trial, up to 12 patients will be given a single dose of radiolabeled TTX-MC138 followed by noninvasive positron emission tomography-magnetic resonance imaging (PET-MRI). The trial is intended to quantify the amount of TTX-MC138 delivered to metastatic lesions and the pharmacokinetics of the therapeutic candidate in cancer patients.

The trial could yield critical data regarding therapeutic dose, timing, and potential safety that could inform later stage clinical trials and further advance TTX-MC138 as a therapeutic candidate against glioblastoma and other advanced malignancies. This trial is not intended to demonstrate any therapeutic effect. TTX-CRISPR, a CRISPR/Cas9--based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines designed to activate cytotoxic immune responses against tumor cells.