AcuraStem was recently featured in Genetic Engineering & Biotechnology News (GEN) in an article titled "Rewriting Disease: Oligonucleotides Take Aim at the Untreatable." The piece examines rapid advancements in nucleic-acid-based drugs and explores how the biotechnology sector is increasingly turning to antisense oligonucleotides (ASOs) to address severe neurodegenerative conditions.
GEN prominently details AcuraStem’s targeted approach within the highly specialized field of oligonucleotide therapeutics. By utilizing its proprietary iNeuroRx platform, AcuraStem bypasses traditional animal models to develop precise patient-based cellular models of disease. Modeling disease in this way allows researchers to accurately observe human cellular pathology and design oligonucleotides capable of halting disease progression for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
In the article, AcuraStem CEO Sam Alworth highlighted the fundamental advantage of AcuraStem’s methodology for uncovering novel therapeutic targets, noting, "AcuraStem uses transcription factor-mediated lineage conversion to reprogram patient-derived iPSCs into induced motor neurons that retain the aging and pathological markers of the donor."
Discussions in the broader article address both the biological challenges and the recent technical breakthroughs associated with translating genetic insights into clinical applications. Advancing ASO programs targeting PIKFYVE and UNC13A demonstrates the capability of therapies to correct RNA processing errors and restore vital cellular clearance mechanisms. GEN underscores an industry-wide shift (led by AcuraStem) toward treatments tailored to the molecular drivers of complex neurological disorders.
AcuraStem is actively advancing precise, RNA-targeted interventions into clinical trials to provide disease-modifying therapies for ALS and FTD patients.
Read the full article at: https://www.genengnews.com/topics/drug-discovery/rewriting-disease-oligonucleotides-take-aim-at-the-untreatable/