Encoded Therapeutics Reports Clinical Progress of ETX101 Gene Therapy for Dravet Syndrome, Recaps 2024 Corporate Achievements, and Provides 2025 Outlook

Encoded Therapeutics, a clinical-stage biotechnology company pioneering AAV (adeno-associated virus) vector-based gene therapies for severe central nervous system (CNS) disorders, has announced significant progress in its lead program, ETX101, for Dravet syndrome, along with a recap of its 2024 achievements and a detailed outlook for 2025. The company is leveraging its proprietary AAV packaging and vector engineering platform to develop potentially one-time, disease-modifying therapies for rare and common CNS conditions.

ETX101 for Dravet Syndrome: POLARIS Program

ETX101 is an AAV9-based gene regulation therapy designed to address the root cause of SCN1A+ Dravet syndrome, a severe genetic epilepsy. The therapy utilizes a novel AAV packaging system to deliver a regulatory element that selectively upregulates the expression of the SCN1A gene in inhibitory neurons, restoring normal function and potentially providing long-lasting benefits.

• Clinical Progress:
  • The POLARIS program, a global clinical trial launched in 2024, is currently enrolling infants and children (ages 6 months–7 years) in the U.S., UK, and Australia.
  • As of February 12, eight patients have been treated across multiple dose levels, with no treatment-related serious adverse events reported.
  • Preliminary safety and efficacy data are expected in the second half of 2025, with additional dosing and data readouts planned.
  • Encoded has aligned with the FDA and MHRA on the design of a sham-controlled, delayed-treatment confirmatory trial to support potential regulatory approval.
• Regulatory Designations:
  • ETX101 has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA, as well as Orphan Designation from the EMA, underscoring its potential as a transformative therapy for Dravet syndrome.

ETX201 for Angelman Syndrome: Advancing to IND-Enabling Studies

Encoded’s second program, ETX201, is an AAV9-based vectorized microRNA (miRNA) therapy designed to treat Angelman syndrome, a rare neurogenetic disorder. The therapy targets the UBE3A antisense transcript (UBE3A-ATS) to unsilence paternal UBE3A expression, addressing the underlying cause of the disease.

• Preclinical Progress:
  • Positive results from a non-human primate (NHP) study were presented at the Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in November 2024. The data demonstrated that ETX201 was well-tolerated and achieved widespread UBE3A upregulation across critical brain regions.
  • Following FDA guidance, Encoded has initiated IND-enabling studies, with a potential IND filing targeted for 2026.
• AAV Vector Engineering:
  • ETX201 utilizes Encoded’s proprietary AAV packaging technology to deliver the miRNA payload efficiently to CNS tissues, highlighting the company’s expertise in AAV vector design and manufacturing.

Research Pipeline: Expanding AAV-Based Therapies for CNS Disorders

Encoded is advancing a robust pipeline of AAV-based gene therapies for both rare and common CNS conditions, including chronic pain and Alzheimer’s disease.

• Chronic Pain Program:
  • This program focuses on an AAV9-based miRNA therapy designed to knock down the expression of SCN9A (NaV1.7), a key sodium channel involved in pain signaling.
  • Preclinical studies in rodent models demonstrated robust and durable correction of pain phenotypes, with ongoing NHP studies expected to support the nomination of a development candidate in 2H25.
• Alzheimer’s Disease Program:
  • Encoded is developing an AAV9-based miRNA therapy targeting MAPT (tau), a protein implicated in Alzheimer’s pathology.
  • NHP studies showed up to 32% knockdown of tau across disease-relevant brain regions, with data to be presented at scientific meetings in mid-2025.
• AAV Packaging and Manufacturing:
  • Encoded’s internal GMP facility, set to be fully operational in 1Q25, will support the production of AAV vectors for ETX101 and pipeline programs, ensuring high-quality, scalable manufacturing capabilities.

Corporate Achievements and Strategic Updates

• Collaboration with Prevail Therapeutics (Eli Lilly):
  • Encoded signed an agreement with Prevail Therapeutics, a subsidiary of Eli Lilly, granting access to its proprietary AAV regulatory elements for gene therapy development. This collaboration highlights the value of Encoded’s AAV vector engineering platform.
• Workforce Reduction:
  • To extend its financial runway through 3Q26 and focus resources on key programs, Encoded implemented a 29% reduction in its workforce, primarily affecting early-stage research and technology functions.

2025 Outlook: A Transformative Year Ahead

Encoded is poised for a transformative year in 2025, with several key milestones expected:

• ETX101: Completion of dosing and preliminary data readouts from the POLARIS program in 2H25.
• ETX201: Continued progress in IND-enabling studies, with a potential IND filing in 2026.
• Pipeline Programs: Nomination of development candidates for chronic pain and Alzheimer’s disease, supported by robust preclinical data.
• GMP Facility: Full operationalization of the internal GMP facility to enhance AAV vector production capabilities.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage biotechnology company developing AAV-based gene therapies for severe CNS disorders. Its proprietary platform combines novel AAV vector engineering, regulatory elements, and payloads to create potentially one-time, disease-modifying treatments. Lead programs include ETX101 for Dravet syndrome and ETX201 for Angelman syndrome, with additional research in chronic pain and Alzheimer’s disease. Encoded is committed to pioneering breakthrough therapies for CNS conditions through its innovative AAV packaging and manufacturing capabilities.

Reference:
https://encoded.com/press-releases/encoded-therapeutics-reports-clinical-progress-of-etx101-gene-therapy-for-dravet-syndrome-recaps-2024-corporate-achievements-and-provides-2025-outlook/

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