Encoded Therapeutics

Month: January 2026

Encoded Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to ETX101 for the Treatment of Dravet Syndrome

Posted on by encoded.tw

– Designation recognizes the potential of ETX101 to address a serious condition with high unmet need –

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ETX101 for the treatment of SCN1A+ Dravet syndrome. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations, all previously granted by the FDA.

“Breakthrough Therapy Designation reflects the FDA’s recognition of both the urgent need for disease-modifying Dravet syndrome treatments and the potential of ETX101 as a meaningful treatment option for this devastating disease,” said Salvador Rico, M.D., Ph.D., Chief Medical Officer of Encoded. “Data from the ongoing Phase 1/2 studies showed durable reduction of seizures and improvements in neurodevelopment following a one-time administration. We are grateful to the FDA for recognizing the potential of ETX101 and look forward to continued collaboration to bring a meaningful new treatment option to the Dravet syndrome community as soon as possible.”

Breakthrough Therapy Designation is a process designed to expedite development and review of investigational therapies that are intended to treat a serious or life-threatening condition and for which preliminary clinical evidence indicates the therapy may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints. The designation provides Encoded with more frequent and intensive FDA guidance, including the involvement of senior reviewers, as well as eligibility for rolling and priority review of the marketing application.

About Dravet Syndrome

Dravet syndrome is a severe genetic developmental and epileptic encephalopathy that begins in infancy and is most commonly caused by loss-of-function variants in the SCN1A gene. The disorder is characterized by frequent, prolonged seizures that are often resistant to treatment, as well as significant cognitive, behavioral, and motor impairments. Individuals with Dravet syndrome face a lifelong disease burden and an increased risk of premature mortality. Current treatment options are largely symptomatic and do not address the underlying genetic cause of the disease. More information about Dravet syndrome can be found at www.dravetfoundation.org.

About ETX101

ETX101 is an investigational AAV9-based gene regulation therapy designed to increase the expression of the SCN1A gene to restore sodium channel function in inhibitory interneurons. By addressing this root mechanism, ETX101 has the potential to treat the full spectrum of Dravet syndrome symptoms, including seizures, communication and cognitive impairment, behavioral issues, and motor dysfunction. The therapy is administered via a single intracerebroventricular (ICV) injection and is designed for long-term benefit. ETX101 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA, as well as Orphan Designation from the European Medicines Agency (EMA).

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage biotechnology company developing precision genetic medicines to transform the lives of patients with severe neurological disorders. The company’s vector engineering platform enables potent and cell-type-selective regulation of gene expression, allowing for targeted modulation of disease-relevant genes. Encoded is advancing a pipeline of programs across genetic epilepsies and other neurological disorders with significant unmet need. With integrated discovery, development, and manufacturing capabilities, Encoded is positioned to efficiently move programs from concept through the clinic. Encoded is driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological disorders. For more information, please visit www.encoded.com.

Investors/Business Development

Jennifer Gorzelany

communications@encoded.com

Media

Lori Rosen

lori@redhousecomms.com

Encoded Therapeutics Highlights Progress in Dravet Syndrome Program and Pipeline Ahead of Company Presentation at 44th Annual J.P. Morgan Healthcare Conference

Posted on by encoded.tw

– Positive interim efficacy data in 2025 underscore potential of ETX101 as a one-time, disease-modifying medicine for Dravet syndrome –

– Internal GMP manufacturing capable of supporting pivotal trial and commercialization of ETX101 and gene therapy pipeline –

– CEO Kartik Ramamoorthi, Ph.D. to present corporate overview at J.P. Morgan Healthcare Conference –

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc. (“Encoded”), a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today provided an update on the significant progress made during 2025 with its Dravet syndrome candidate, ETX101, including an overview of the positive interim Phase 1/2 clinical results announced last month, establishment of GMP manufacturing capabilities to support its pipeline of gene therapies, and progress across its overall pipeline.

“Last year marked a period of significant execution for Encoded, including meaningful progress across our pipeline and manufacturing capabilities. Data that were recently presented at the American Epilepsy Society Annual Meeting underscore the potential clinical impact of ETX101 for people with Dravet syndrome. With our highly experienced team and an operational GMP manufacturing facility we are well-positioned to initiate a pivotal trial of ETX101 in 2026,” said Kartik Ramamoorthi, Ph.D., Chief Executive Officer of Encoded Therapeutics. “We are excited about multiple near-term catalysts in the year ahead and are focused on executing our vision to deliver transformative gene therapies to patients.”

Progress Across Dravet Syndrome Program

The Company’s clinical development of ETX101 reached a strategic inflection point, marked by significant progress across the following clinical and manufacturing milestones:

  • ETX101 demonstrated dose-dependent and sustained reductions in monthly countable seizure frequency. Progressive and clinically meaningful gains were observed in multiple measures of neurodevelopment across initial dose levels, supporting the potential for ETX101 to prevent neurodevelopmental stagnation with early intervention.

  • Attained Regenerative Medicine Advanced Therapy (RMAT) designation for ETX101 following review of preliminary clinical data from POLARIS Phase 1/2 studies.

  • Launched a fully operational GMP manufacturing facility, successfully producing clinical material to supply the pivotal study while establishing a foundation that is readily scalable to support future commercial demand. The facility’s integrated CMC and GMP production capabilities enable tight control over quality and timelines that de-risk supply and preserve economic flexibility.

  • Strengthened clinical leadership team with the addition of Joseph Sullivan, M.D., as Vice President of Clinical Development.

Progress Across Preclinical Pipeline

Preclinical programs further advanced toward clinical development with robust proof-of-concept data in non-human primates (NHPs) presented at key scientific meetings:

  • Chronic pain: Durable knockdown of SCN9A (NaV1.7) following one-time intrathecal lumbar administration of AAV9-based miRNA candidate presented at the European Society of Gene & Cell Therapy Annual Congress. Level of target engagement observed in NHPs exceeded the threshold for pain relief established in rodent chronic pain models.

  • Angelman syndrome: Strong safety and target engagement data with unsilencing of UBE3A across disease relevant brain regions for AAV9-based candidate ETX201 presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, and for IV-delivered novel capsid candidate at the Foundation for Angelman Therapeutics (FAST) Global Science Summit.

  • Tauopathies: Potent and robust knockdown of MAPT (tau) with AAV9 candidates presented at the ASGCT Annual Meeting.

2026 Anticipated Milestones

The Company expects important milestones in 2026 to reflect ongoing program progress related to:

  • Interim efficacy results from the highest dose level of the POLARIS clinical program;

  • Initiation of ETX101 pivotal study; and

  • Additional progress across the early-stage pipeline, including nomination of a development candidate for chronic pain.

J.P. Morgan Healthcare Conference Presentation Details

Dr. Ramamoorthi will present a corporate overview and 2026 outlook on Monday, January 12, 2026, starting at 5:30 p.m. PT at The Westin St. Francis Hotel (Golden Gate Room, 32nd Floor).

About ETX101

ETX101 is a potential one-time, disease-modifying AAV9-based gene regulation therapy for SCN1A+ Dravet syndrome. ETX101 is designed to target the underlying cause of Dravet syndrome by increasing SCN1A expression in GABAergic inhibitory neurons. By addressing this root mechanism, ETX101 has the potential to treat the full spectrum of Dravet syndrome symptoms, including seizures, communication and cognitive impairment, behavioral issues, and motor dysfunction. ETX101 has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA, as well as Orphan Designation from the EMA.

About the POLARIS Program

POLARIS is a comprehensive clinical investigation of ETX101 in children with SCN1A+ Dravet syndrome, comprising multiple Phase 1-3 clinical trials. The first phase of POLARIS comprises three open-label, Phase 1/2 dose-escalation, multi-center trials (ENDEAVOR Part 1 (US), EXPEDITION (UK), and WAYFINDER (Australia)) in infants and young children 6 months to <7 years of age. The primary objective of the studies is to assess the safety and tolerability of ETX101. Secondary objectives are to evaluate the preliminary efficacy of ETX101 by assessing the percent change from baseline in monthly countable seizure frequency (MCSF), as well as the impact on the neurodevelopmental symptoms of Dravet syndrome, including cognition, communication, and motor function.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing one-time therapies for severe neurological disorders. Our vector engineering platform enables highly selective, potent, and durable gene modulation. The lead program, ETX101, is designed to address the underlying cause of Dravet syndrome by durably upregulating SCN1A and is currently in Phase 1/2 clinical trials. Building on this foundation, we are advancing programs to modulate validated genetic targets in chronic pain, Angelman syndrome, and Alzheimer’s disease/tauopathies. With integrated discovery, development, and manufacturing capabilities, we are positioned to efficiently move programs from concept through the clinic. We are driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological disorders. For more information, please visit www.encoded.com.

Investors/Business Development

Jennifer Gorzelany

communications@encoded.com

Media

Lori Rosen

lori@redhousecomms.com

Encoded Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026

Posted on by encoded.tw

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced that Kartik Ramamoorthi, Ph.D., Chief Executive Officer, will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026 at 5:30 p.m. PT in San Francisco, California.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing one-time therapies for severe neurological disorders. Our vector engineering platform enables highly selective, potent, and durable gene modulation. The lead program, ETX101, is designed to address the underlying cause of Dravet syndrome by durably upregulating SCN1A and is currently in Phase 1/2 clinical trials. Building on this foundation, we are advancing programs to modulate validated genetic targets in chronic pain, Angelman syndrome, and Alzheimer’s disease/tauopathies. With integrated discovery, development, and manufacturing capabilities, we are positioned to efficiently move programs from concept through the clinic. We are driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological conditions. For more information, please visit www.encoded.com.

Investors/Business Development

Jennifer Gorzelany

communications@encoded.com

Media

Lori Rosen

lori@redhousecomms.com