At Epicrispr, we are building the world’s most expansive therapeutic pipeline based on the science of epigenetic engineering.
Our pipeline
We aim to develop products that change the expression of certain genes inside cells to ameliorate specific defects. Our therapeutic candidates are utilizing a wide spectrum of mechanisms of action (see below), with clinically validated in vivo and ex vivo delivery approaches, and a mutation-agnostic technology.
As we strive to develop this new class of treatments, we are committed to working closely with regulatory agencies, patient advocacy groups and healthcare professionals to bring our products to patients.
Other
Mechanisms:
Suppress: Suppress the endogenous gene either by downregulation or by permanently turning off the expression of a gene
Activate: Upregulate the expression of a gene either to physiological levels (i.e., to restore haploinsufficient gene activity) or above physiological levels
Suppress + replace: Suppress the endogenous mutated gene and replace it with exogenous wildtype version of gene
Undisclosed
The persistence and durability of Epicrispr’s hypercompact GEMS allow for one-time treatment of human diseases, which provides advantages over other approaches requiring chronic administration. Learn more in this video.