Transcription Factor Activator ASO

Riboway Therapeutics is developing a preclinical antisense oligonucleotide (ASO) program designed to activate a high-value, previously undruggable transcription factor for the treatment of neurodegenerative and metabolic diseases. The company utilizes its AI-enabled Smart ASO platform to decode hidden RNA regulatory events, enabling precise modulation of protein expression, including upregulation and activation. This represents a first-in-class approach, as the ASO is designed to directly activate a transcription factor target that was previously considered undruggable by traditional therapeutic modalities. While the specific target has not been publicly disclosed, the program is intended to address rare, genetically defined diseases before expanding into larger indications such as Amyotrophic Lateral Sclerosis (ALS) and Alzheimer's disease.

Other names

Transcription Factor Activator ASOSmart ASO transcription factor program