Research Areas

Our internally developed pipeline comprises investigational cell-selective gene therapies that hold promise to treat not only pediatric central nervous system (CNS) disorders, but also, in the future, liver, metabolic and cardiovascular diseases.

Encoded is independently building a wholly owned pipeline of potential best-in-class gene therapies using our novel approach and evolving understanding of genetic targets and biology.

In certain disease areas, we see the potential to be the first market entrant to deliver a one-time disease-modifying therapy.

How We Identify Targets

Each one of our programs is born at Encoded as a solution to a biological problem. We combine genomics-driven data with internal biological expertise and access to worldwide thought leaders to employ best-in-class identification of therapeutic targets, initially for monogenic disorders. We believe Encoded therapies have the potential to impact incident and prevalent populations across a number of indications.

Evolving understanding of genetic targets and biology

Proven and applicable cell-selective technology

Targeting disorders with significant unmet need

Lower manufacturing requirements due to therapeutics with greater selectivity, potency and intracerebroventricular (ICV) route of administration

World-class manufacturing, clinical and regulatory expertise to independently develop best- and potentially first-in-class therapies

The pediatric CNS disorders on which we are initially focused, including Dravet syndrome, have significant unmet need, with lifelong devastating impact for patients and families. These disorders also have no approved products to address the underlying cause of disease.


Leveraging CNS-Centered Infrastructure and Expertise

Our plans to achieve lifelong transformative outcomes for people with pediatric CNS disorders with high unmet need will be accelerated by the knowledge, technology and infrastructure we are developing to support our SCN1A+ Dravet syndrome program.

ETX101 has been generated using in-house capabilities – from preclinical discovery to our planned clinical trials. This CNS-centered expertise and infrastructure, combined with our strategies for increasing output and quality, are expected to yield future efficiencies, therefore expediting time to market for our emerging pipeline programs. We are paving the way for broader development of and access to gene therapies for Dravet syndrome and other difficult-to-treat pediatric CNS disorders.

Broad Therapeutic Potential

Our initial program, ETX101, targets SCN1A+ Dravet syndrome, which is the most common developmental and epileptic encephalopathy. Encoded also is applying its proprietary cell-selective gene therapy approach to preclinical programs for other pediatric CNS disorders with high unmet medical need. But we’re not stopping there.

Developing Best- and Potentially First-in-Class Treatments for Diseases That Cannot be Addressed with Current Technology

With an initial focus on monogenic disorders, our goal is to provide long-lasting and life-changing benefits to many patients and families through a single treatment administration. Encoded’s genomics-based approach to modulating cell function additionally offers broad therapeutic potential for liver, metabolic and cardiovascular diseases. We are powered to develop and manufacture one-time gene therapies for a broad range of indications, spanning disorders that originate in multiple organs.