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.
What is it?
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscle disorder mostly affecting the face, scapula, and the humerus region. The loss of movement is accompanied with chronic pain, anxiety, and depression. Symptoms usually start before age 20 and may lead to being wheelchair-bound by age 50. It is estimated to be the second most common muscular dystrophy.
What causes it?
FSHD is caused by a loss of methylation of the D4Z4 region, leading to an abnormal expression of the DUX4 gene on chromosome 4, normally only expressed during embryogenesis. Abnormal expression of DUX4 in the muscles is toxic, eventually leading to muscle cell death and tissue degeneration.
How do we treat it?
There is currently no cure for FSHD. The standard of care requires a team of specialists who can only manage symptoms, highlighting the unmet need for a transformative therapy. Therefore, Epicrispr can target re-methylating the D4Z4 region and suppressing DUX4 expression to prevent further muscle cell death.
Neuromuscular
Other therapeutic area
Oncology
Key
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 and replace: Suppress the endogenous mutated gene and replace it with exogenous wildtype version of gene
Multiplex activation: Activate several genes to add specific feature to cells (i.e., neuroprotection capabilities)
Multiplex suppression: Suppress several genes at a time to remove specific features to cells
Multidirectional modulation: Simultaneously suppress and activate different genes
Undisclosed