Drugs / ETX101
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05419492 | May 2024 → Jan 2028 expected | Dravet syndrome | Encoded Therapeutics | Recruiting | No outcome recorded |
| Phase 1/2 | NCT06283212 | May 2024 → Oct 2030 expected | Dravet syndrome | Encoded Therapeutics | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT06112275 | Feb 2024 → Sep 2030 expected | Dravet syndrome | Encoded Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2026-09-08 | Encoded Therapeutics | Results Encoded Therapeutics Presents Updated Data from POLARIS Phase 1/2 Trials of ETX101 Gene Therapy in Dravet Syndrome at the 16th European Epilepsy Congress encoded.com ↗
ETX101 continues to demonstrate substantial and durable reductions in monthly countable seizure frequency (MCSF) following a single administration. |
| 2026-09-02 | Encoded Therapeutics | Results Encoded Therapeutics to Present New Data on ETX101 Gene Therapy for Dravet Syndrome at the 16th European Epilepsy Congress encoded.com ↗ |
| 2026-05-13 | Encoded Therapeutics | Results Encoded Therapeutics Presents New Clinical Data from POLARIS Phase 1/2 Trials of ETX101 Gene Therapy in Dravet Syndrome at the ASGCT 2026 Presidential Symposium encoded.com ↗
Treatment with a single dose of ETX101 resulted in a robust and dose-dependent antiseizure effect, with durability through 52 weeks of observation. |
| 2026-01-12 | Encoded Therapeutics | Regulatory Encoded Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to ETX101 for the Treatment of Dravet Syndrome encoded.com ↗
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. |
| 2025-12-05 | Encoded Therapeutics | Results Encoded Therapeutics Presents Positive Interim Efficacy Data from Initial Dose Levels of Phase 1/2 Trials Evaluating ETX101 Gene Therapy in Dravet Syndrome encoded.com ↗ |
| 2025-09-02 | Encoded Therapeutics | Regulatory Encoded Therapeutics Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for ETX101 in SCN1A+ Dravet Syndrome encoded.com ↗ |
| 2024-02-26 | Encoded Therapeutics | Regulatory Encoded Therapeutics Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101 encoded.com ↗
This CTA marks the third regulatory approval for ETX101 as we advance our global clinical development program, POLARIS, to bring this potential one-time AAV gene regulation therapy to people affected by SCN1A+ Dravet syndrome around the world |
| 2024-02-06 | Encoded Therapeutics | Regulatory Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101 encoded.com ↗ |
| 2020-07-22 | Encoded Therapeutics | Regulatory Encoded Therapeutics Announces $135 Million Series D Financing to Support First Clinical Trials in SCN1A+ Dravet Syndrome and Advance Preclinical Pipeline of Gene Therapies for Debilitating Neurological Disorders encoded.com ↗
The company also announced that its lead asset, ETX101, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome. |
All press releases naming this drug 15 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ETX101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05419492 ↗ |
| Action | Activate | “increases transcription of the SCN1A gene” NCT05419492 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated viral vector serotype 9 (rAAV9)” NCT05419492 ↗ |
| Route | Other | “one-time intracerebroventricular (ICV) administration” NCT05419492 ↗ |
| Target | SCN1A | “increases transcription of the SCN1A gene” NCT05419492 ↗ |