Research Areas
Our internally developed pipeline comprises investigational gene therapies that hold promise to treat not only central nervous system (CNS) disorders, but also, in the future, diseases impacting additional organ systems.
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.
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 for monogenic and non-monogenic disorders. We believe our therapies have the potential to deliver transformative benefit across a range of diseases with high unmet need.
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 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 clinical trials. This CNS-centered expertise and infrastructure, combined with our strategies for increasing output and quality, are expected to yield future efficiencies, 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 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 and gene modulation approaches to preclinical programs for other 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 patients and their families through a single treatment administration. Encoded’s genomics-based approach to modulating cell function additionally offers broad therapeutic potential for the development of gene therapies for a range of indications, spanning disorders that originate in multiple organs.
