Genetic Medicines
Product Engine

We can take gene therapies from concept to commercialization, employing our end-to-end capabilities across technology, preclinical, process development and manufacturing, as well as clinical development, regulatory and quality, and commercial. Our ambition is to enable gene therapy to be a viable therapeutic option across multiple diseases, common and rare, in the coming years.

A Viable and Sustainable Gene Therapy Product Engine

Cellular Targets and Preclinical Development

Innovative therapeutic approaches that target the underlying genetic and cellular mechanism of disease for optimal safety and efficacy

Process Development / Manufacturing

Manufacturing processes, analytical tools and quality systems can be deployed across all programs

Route of Administration and Formulation

Intracerebroventricular (ICV) infusion and formulation development allow for broad biodistribution to disease-relevant brain structures

Clinical Endpoints

Similar endpoints (seizure and neurodevelopment) and natural history study design may be used across a variety of pediatric CNS indications, potentially standardizing clinical development

Encoded is independently building a pipeline of potentially best- and potentially first-in-class therapies focused on pediatric central nervous system (CNS) disorders. Our research platform is internally developed, and our programs are wholly owned. We aim to continue to develop our pipeline utilizing our end-to-end capabilities as well as leveraging our research platform to broaden our therapeutic reach independently as well as industry partnerships.

Applying Our Diverse Expertise to Precision Gene Therapy

We have the right combination of innovation and experience to realize the full potential of one-time gene therapies. Our leaders bring to Encoded diverse expertise across genomics, rare disease and all aspects of gene therapy development.

Best-in-Class Manufacturing

Although hundreds of cell and gene therapies are currently in development, issues with supply continue to stymy access and be a bottleneck for the industry. Encoded’s manufacturing ecosystem is being built on the principles of flexibility, transferability and scalability.

The operational and commercial viability of gene therapy is dependent on meeting high-quality standards and yielding efficiency that can address rare and large indication supply requirements.

Encoded has invested in these capabilities since our inception and has made tremendous strides in addressing scale challenges to ensure gene therapy meets exponential yield improvements over time, consistent with earlier biologic modalities. Critical components of our ecosystem include the following:

• Internal talent who are subject matter experts on process development, analytical development, manufacturing, supply chain and quality assurance for gene therapy
• Process Development that meets current AAV commercial-scale requirements with a clear path to exponential improvement
• Infrastructure that meets non-GMP-scale requirements for multiple programs
• GMP ecosystem that includes preferred CDMO relationships and internal scale-up