Encoded Therapeutics Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101
Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics announced today the presentation of non-human primate (NHP) validation of the company’s technology to drive desired gene and protein expression patterns at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting. An important challenge of developing gene therapy in many central nervous system (CNS) disorders is the toxicity concern associated with high transgene expression in the dorsal root ganglion (DRG). By incorporating a DRG de-targeting regulatory element (RE) derived from Encoded’s research platform into a CNS-targeted gene therapy construct, the company achieved a significant reduction in DRG protein expression to endogenous baseline. This resulted in a greater than 10-fold selective reduction in DRG transcriptional activity without impacting brain expression.
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics announced nonclinical data being presented today at the American Society for Cell and Gene Therapy 25th Annual Meeting showing how its proprietary human genomic regulatory element (RE) engineering platform has been used to develop cell-selective expression vectors for targeted gene therapy. The company efficiently discovered a spectrum of sequence-encoded genetic elements, including enhancers, promoters and UTR elements, that drive selective expression profiles in mice. These REs function within the size constraints and episomal architecture of adeno-associated viruses (AAVs) and are compatible with multiple capsids and gene delivery systems.
Preliminary observational study findings revealed high use of nantiseizure medications, significant delay in adaptive functioning, and nconsequential communication and socialization impact in young children nwith Dravet syndrome, worsening with age
Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its gene therapy for SCN1A-positive Dravet syndrome patients.