Regardless of the impacted protein, Alltrna is creating designed tRNA medications that can read through premature termination codons and resume production of the impacted protein.
The strategy aims to address the drawbacks of conventional rare disease medications, which usually concentrate on a specific disease and make it challenging to create cures for the great majority of rare genetic disorders.
According to Michelle Werner, CEO of Alltrna, “our engineered tRNAs can be used across multiple diseases, genes, and mutation locations.” “By using a mutation-specific approach instead of a disease-specific approach, this represents a far more universal tool to treat patients.”
To manage the challenges of creating such studies, she stated that Alltrna is collaborating closely with international regulators, such as the FDA and European Medicines Agency (EMA). According to Werner, regulators are promoting approaches that can treat several illnesses at once because they understand the difficulties in developing medications for really unusual people. [Read More: tRNA PLATFORM]