Developing RNA-Modulating Oral Small Molecules Targeting PMS1 to Treat Incurable Neurological Disorders
Time: 9:00 am
day: Day Two
Details:
- Leveraging our proprietary, integrative RNA-targeting small molecule discovery platform to pioneer the development of first-in-class oral therapies
- Pursuing oncology and neurological disease targets, exemplified by the oncogenic transcription factor target c-MYB and the PMS1 gene
- PMS1 is a key component of the DNA mismatch repair pathway, implicated in the pathological somatic trinucleotide repeat expansion observed in Huntington's Disease (HD) and other trinucleotide repeat expansion disorders
