Other Therapeutic Candidates

TTX-siPDL1
TTX-siPDL1 is a therapeutic candidate in development with TransCode to target PD-1/PD-L1. As traditional checkpoint inhibitors simply block PD-1/PD-L1 from binding each other, they may be ineffective in many cancers, including pancreatic cancer. However, TTX-siPDL1 employs an RNAi approach with the intention of preventing the synthesis of PD-L1 altogether instead of blocking its function.

As a result of successful animal studies, TTX-siPDL1 has received Orphan Designation Status from FDA for pancreatic cancer.

​64Cu-TTX-MC138
The use of radioisotopes is a (re)emerging approach for many drug development companies in the treatment of cancer. TransCode believes our TTX has the potential for efficient localized penetration of radioisotopes inside tumor cells. 64Cu-TTX-MC138 has potential benefits that include:

• PK profile that minimizes off-target exposure, maximizes high tumor uptake
• Achieves localization, penetration, and persistence across tumor histologies while maximizing systemic clearance
• Deeper tumor penetration to enable more homogeneous irradiation of tumor mass
• Achieving the above enables use of off-target toxicity reducing short-range alpha emitters

In pre-clinical studies, delivery of 64Cu-TTX-MC138 to metastatic sites has been demonstrated; a first-in-human microdosing study is anticipated in Q4 2022. 

TTX-RIGA
Immunotherapy represents a powerful alternative to traditional clinical treatments for cancer. Recent developments in the use of Pattern Recognition Receptors, or PRRs, specifically retinoic acid-inducible gene I-like receptors (RIG-I), aim to harness the innate power of the immune system for anti-cancer therapy. TTX-RIGA is in development to target cancer via the RIG-I signaling pathway. Delivery of TTX-RIGA combines with a tumor specific RNA inside tumor cells to produce  a potent agonist of the RIG-I signaling pathway.

RIG-I engagement has been shown to promote tumor cell death, and to activate the innate and adaptive immune systems. These factors suggest it could be an attractive therapeutic approach in oncology.

TTX-siLIN28B
TTX-siLIN28B is in development to target multiple solid tumors. LIN28B is a biomarker of tumor survival and an actionable therapeutic target for solid tumors. 

Additional TTX Therapeutic Opportunities Being Explored in Our Lab

• TTX can be designed to deliver CRISPR/Cas9 therapy inside tumor cells to delete and/or repair cancer-causing gene sequences (TTX-CRISPR). 

• TTX-mRNA, a messenger RNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells.

Diagnostics
We are also studying approaches for early cancer detection.
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TransCode executed exclusive option agreements with Massachusetts General Hospital (MGH), for TTX-siLin28b and 64Cu-TTX-MC138, under which TransCode has the right to negotiate an exclusive license for these assets.