Encoded Therapeutics to Present New Data for CNS Gene Therapy Programs at the ASGCT 28th Annual Meeting

• AAV9-based Angelman Syndrome candidate, ETX201, demonstrates safety and target engagement in non-human primates (NHPs), with unsilencing of UBE3A across disease-relevant brain regions

• Multiple neurodegeneration candidates in Alzheimer’s disease / tauopathies demonstrate bulk knockdown of MAPT in cortical and hippocampal brain regions of NHPs with AAV9-based intracerebroventricular delivery, confirming potency of lead candidates identified in in vitro screens

• Vectorized miRNA candidates for chronic pain demonstrate potent knockdown of Scn9a and analgesic effect in a well-established rat model

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced three presentations that underscore the strength of the Company’s vector engineering platform and demonstrate continued progress of the ETX201 in Angelman syndrome (AS), chronic pain and Alzheimer’s disease / tauopathies programs. The presentations will be made at the 28^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, LA, May 13 – 17, 2025.

“Our vector engineering platform continues to demonstrate its potential to bring life-changing therapies to patients,” commented Chief Scientific Officer and co-founder Stephanie Tagliatela. “Specifically, in our Angelman syndrome and Alzheimer’s / tauopathies programs, new findings further validate the ability of our engineered vectors to modulate disease-relevant genes in bulk NHP CNS tissue with AAV9. In our SCN9A-targeted pain program, we have achieved robust and durable efficacy in a well-established rat model of chronic pain. These results fuel our commitment to diligently advancing these therapies towards the clinic."

Poster Presentation Details

Title: ETX201: An AAV9-based Vectorized miRNA Therapeutic Candidate for Angelman Syndrome. Poster # 547. Presenter, Sirika Pillay, PhD

Date & Time: Tuesday, May 13, 6 – 7:30 PM CT
Location: Poster Hall I2

Title: Potent and Specific AAV9-miRNA Candidates Demonstrate Robust Reduction of Microtubule-associated Protein Tau (MAPT) in Non-Human Primates. Poster # 1440. Presenter, Veda Tatavarty, PhD

Date & Time: Wednesday, May 14, 5:30 – 7 PM CT
Location: Poster Hall I2

Title: Developing an AAV-based Gene Therapy for Chronic Pain Through Identification of Potent and Selective Artificial miRNA Candidates to Knockdown SCN9A. Poster # 1925. Presenter, Chao Tai, PhD

Date & Time: Thursday, May 15, 5:30 – 7 PM CT
Location: Poster Hall I2

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially one-time, disease-modifying therapies for severe CNS disorders. Our proprietary vector engineering technology combines novel regulatory elements and payloads with AAV vectors, unlocking innovative solutions for intractable CNS conditions. Our lead clinical-stage program, ETX101 for Dravet syndrome, targets the underlying cause of the disorder through selective upregulation of SCN1A for potentially long-lasting benefit. Encoded’s second program, ETX201, is a development-stage vectorized miRNA-based gene therapy designed to restore expression of UBE3A in individuals with Angelman syndrome. In parallel, we are advancing potentially best-in-class programs for common CNS conditions, including chronic pain and Alzheimer’s disease / tauopathies. Harnessing our proprietary technology platform and expertise, we can efficiently advance programs from discovery through clinical development. Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit www.encoded.com.