1E Therapeutics
- Biotech or pharma, therapeutic R&D
- CMO, CRO
1E Therapeutics is developing a new modality of oligonucleotide based RNA knockdown, independent of cellular machinery, allowing for highly targeted allele and isoform specific knockdown. We have recently demonstrated in vivo proof of platform, showing benchmark and allele specific RNA knockdown in rodents. Our lead indications are focused on hematology (polycythemia vera-targeting V617F), immunology (CARD11 and VEXAS syndrome), and neurodegeneration (Huntington's and Alzheimer's). IND expected in 2027 with initial clinical POC in 2028.
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