New Chemical Entity

Programmable And Hyperstable Engineered Synthetic Transcriptional Repressors

SUMMARY A class of functionally enhanced synthetic transcriptional factor antagonists to modulate the MYC/MAX transcriptional network by direct competition for DNA binding. The Unmet Need: Transcription factors remain largely untapped as pharmacologic targets due to the challenges in targeting protein–protein and protein–DNA interactions… Read More

Programmable Translational-Activating RNA Therapeutic Platform Technology

SUMMARY Many human diseases arise from insufficient protein levels, often due to genetic mutations that result in haploinsufficiency or loss of protein function. Effectively treating these conditions necessitates therapeutic strategies capable of increasing the production of specific, vital proteins. There is a significant unmet need for programmable methods to… Read More

Novel Aqueous Chemical Stabilization Method for Proteins Based on the Diels-alder Reaction

SUMMARY Stabilization of therapeutic proteins and peptides is often necessary to improve pharmacokinetic properties and reduce proteolysis in vivo. However, pre-existing stabilization strategies require expensive catalysts or harsh reagents that prevent preservation of certain sterically challenging protein structures. The inventors found that the Diels-Alder reaction, when performed aqueously or in… Read More