Pipeline

Pipeline

At Epic Bio, we are building the world’s most expansive therapeutic pipeline based on the emerging science of epigenetic engineering.

Pipeline
We are leveraging our platform to pursue treatments for a range of serious diseases that are insufficiently addressed by today’s genetic medicines. More about 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.

Program
Research
IND-Enabling
Clinical

Facioscapulohumeral Muscular Dystrophy (FSHD)

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, Epic Bio can target re-methylating the D4Z4 region and suppressing DUX4 expression to prevent further muscle cell death.

Heterozygous Familial Hypercholesterolemia (HeFH)

What is it?

HeFH is a genetic disorder resulting in high levels of LDL cholesterol or “bad cholesterol.” Patients suffering from HeFH have elevated bad cholesterol levels from birth which dramatically increases over time. HeFH increases the risk of hardening of the arteries, can lead to heart attacks, strokes, and coronary artery disease. HeFH represents one of the most common monogenic diseases.

What causes it?

HeFH is an autosomal dominant disease most commonly caused by a single variant in one of three genes (APOB, LDLR, PCSK9), affecting the processing of cholesterol.

How do we treat it?

Standard of care includes cholesterol-lowering medications such as statins. More recently, injectable PCSK9 inhibitors have been shown to reduce bad cholesterol by increasing LDL receptors (LDLR) expression on cell surfaces. However, lifelong medications are known to be challenging with long-term adverse effects, decreased efficacy from chronic use, and loss of adherence over time.

Epic Bio is developing a differentiated therapy that will (1) minimize adverse effects by specifically targeting key pathways known to reduce cholesterol, (2) be efficacious and long-lasting, and (3) be safe with no DNA alteration.

Alpha-1 Antitrypsin Deficiency (A1AD)

What is it?

A1AD is a genetic disorder that may result in lung and liver diseases. Lung symptoms start with shortness of breath and develop into chronic obstructive pulmonary disease. Liver symptoms may start from birth and progress into liver cirrhosis. A1AD is considered one of the most common genetic diseases worldwide.

What causes it?

A1AD is due to mutations in a gene called SERPINA1, that encodes the A1AT protein. Instead of being secreted, A1AT mutant protein aggregates in the liver and causes damage. Reduced levels of secreted A1AT result in a deficiency in the lung, which in turn causes elastase to accumulate and degrade the lung.

How do we treat it?

The only available treatment for liver disease is liver transplantation. The standard of care for lung disease includes weekly IV infusions of A1AT. However, the treatment is suboptimal, expensive and requires lifestyle adjustments. A1AD has been the subject of several gene augmentation therapies, with mixed success, and none of them addressing the liver disease phenotype.

At Epic Bio, we are developing a unique approach for the treatment of both liver and lung diseases, designed to work on all SERPINA1 mutations and free of any DNA alteration. This “suppress and replace” therapeutic approach is based on a single AAV vector that can simultaneously inhibit endogenous mutated A1AT expression (suppress) and produce normal A1AT (replace).

Retinitis Pigmentosa 4 (RP4)

What is it?

RP is a group of genetic disorders characterized by degeneration of the retinal cells, which causes progressive loss of vision. There are many genes involved in RP, with autosomal dominant, recessive, X-linked, and maternal inheritance patterns. Patients typically have trouble seeing at night and experience tunnel vision. RP is the leading cause of inherited blindness. Autosomal dominant RP4 (AdRP4) represents about 30% of all autosomal dominant RP cases.

What causes it?

AdRP4 is a hereditary autosomal dominant degenerative disorder caused by mutations in the RHO (Rhodopsin) gene, encoding a light-sensitive receptor protein involved in visual phototransduction. Accumulation of misfolded RHO in the endoplasmic reticulum ultimately leads to photoreceptor degeneration. There are more than 140 mutations that have been identified with AdRP4.

How do we treat it?

There is no cure for any form of RP and the condition is usually managed by low vision aids. Developing gene therapy treatment for AdRP4 has been challenging because of its dominant negative nature and because there are more than 140 mutations associated with this condition. Studies indicate that augmentation or suppression strategies alone are insufficient.

At Epic Bio, we are developing a unique treatment designed to work on all 140 RHO mutations and free of any DNA alteration. This “suppress and replace” therapeutic approach is based on a single AAV vector that can simultaneously inhibit endogenous mutated RHO expression (suppress) and produce normal RHO (replace).

Retinitis Pigmentosa 11 (RP11)

What is it?

RP is a group of genetic disorders characterized by degeneration of the retinal cells, which causes progressive loss of vision. There are many genes involved in various forms of RP, with autosomal dominant, recessive, X-linked, and maternal inheritance patterns. Patients typically have trouble seeing at night and experience tunnel vision. RP is the leading cause of inherited blindness. Autosomal dominant RP11 (AdRP11) represents about 10% of all autosomal dominant RP cases.

What causes it?

AdRP11 is a hereditary autosomal dominant degenerative disorder caused by mutations in the PRPF31 gene, a splicing factor essential for the formation of the spliceosome. The exact mechanisms leading to retinal degeneration is still not clear but are associated with haploinsufficiency mechanisms. There are more than 100 identified mutations of PRPF31.

How do we treat it?

There is no cure for AdRP11 and the condition is usually managed by low vision aids. Augmentation gene therapy approaches have avoided haploinsufficiency diseases because evidence suggests that haploinsufficient genes are toxic when overexpressed.

At Epic Bio, we want to leverage the power of our technology to create a treatment for AdRP11, that precisely restores PRPF31 gene expression to normal physiological levels and develop the first in vivo gene therapy product to treat AdRP11.

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