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NEWS #PharmaBuzz
25 Mar 2026
// PHARMIWEB
https://www.pharmiweb.com/press-release/2026-03-25/encoded-therapeutics-announces-fda-alignment-initiation-of-pivotal-study-and-asgct-presidential-sy
12 Jan 2026
// PHARMIWEB
https://www.pharmiweb.com/press-release/2026-01-12/encoded-therapeutics-announces-us-fda-breakthrough-therapy-designation-granted-to-etx101-for-the-t
08 Jan 2026
// PHARMIWEB
https://www.pharmiweb.com/press-release/2026-01-08/encoded-therapeutics-highlights-progress-in-dravet-syndrome-program-and-pipeline-ahead-of-company-pr
05 Jan 2026
// BUSINESSWIRE
https://www.businesswire.com/news/home/20260105836296/en/Encoded-Therapeutics-to-Present-at-the-44th-Annual-J.P.-Morgan-Healthcare-Conference-on-January-12-2026
16 Dec 2025
// BUSINESSWIRE
https://www.businesswire.com/news/home/20251216682441/en/Encoded-Therapeutics-Strengthens-Clinical-Leadership-Team-with-Appointment-of-Professor-Joseph-Sullivan-M.D.-as-Vice-President-of-Clinical-Development
05 Dec 2025
// FIERCE BIOTECH
https://www.fiercebiotech.com/biotech/encoded-deciphers-path-pivotal-trial-phase-12-dravet-gene-therapy-data
Drugs in Development
Details:
An undisclosed cell & gene therapy targeting SCN9A (NaV1.7) shows promise in treating chronic pain disorders.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable October 09, 2025
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Encoded Therapeutics Shows Non-opioid Gene Therapy Potential for Chronic Pain
Details : An undisclosed cell & gene therapy targeting SCN9A (NaV1.7) shows promise in treating chronic pain disorders.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
October 09, 2025
Details:
ETX101 is an AAV9-mediated gene regulation therapy designed to upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons. It is being evaluated for the treatment of Dravet syndrome.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable February 13, 2025
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Encoded Updates on ETX101 for Dravet, Highlights 2024 Wins, Shares 2025 Plan
Details : ETX101 is an AAV9-mediated gene regulation therapy designed to upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons. It is being evaluated for the treatment of Dravet syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 13, 2025
Details:
ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable February 28, 2024
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 28, 2024
Details:
ETX101, an AAV9-mediated gene therapy, selectively upregulates SCN1A gene expression in GABAergic neurons. It is being studied for treating SCN1A-positive Dravet syndrome in young children.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable February 26, 2024
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Encoded Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101
Details : ETX101, an AAV9-mediated gene therapy, selectively upregulates SCN1A gene expression in GABAergic neurons. It is being studied for treating SCN1A-positive Dravet syndrome in young children.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 26, 2024
Details:
ETX101 is an AAV9-mediated gene therapy designed to upregulate SCN1A expression in GABAergic neurons, currently evaluated in phase 1/2 trials for SCN1A-positive Dravet syndrome.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable February 06, 2024
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Encoded Receives US IND Clearance and Australian Approval for ETX101
Details : ETX101 is an AAV9-mediated gene therapy designed to upregulate SCN1A expression in GABAergic neurons, currently evaluated in phase 1/2 trials for SCN1A-positive Dravet syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 06, 2024
Details:
ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable November 01, 2023
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
November 01, 2023
Details:
ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: Phase I/ Phase IIProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable June 15, 2022
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ETX101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Epilepsies, Myoclonic.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 15, 2022
Details:
Multiple adaptable DNA sequence-encoded human genetic elements are modality-agnostic and can be combined to customize expression profiles to optimize therapeutic approaches in and beyond the brain.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Undisclosed
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable May 18, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Multiple adaptable DNA sequence-encoded human genetic elements are modality-agnostic and can be combined to customize expression profiles to optimize therapeutic approaches in and beyond the brain.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
May 18, 2022
Details:
ETX101, Encoded's lead asset, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by FDA for the treatment of SCN1A+ Dravet Syndrome.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Cell & Gene Therapy
Sponsor: GV
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Series D Financing July 22, 2020
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Partner/Sponsor/Collaborator : GV
Deal Size : Undisclosed
Deal Type : Series D Financing
Details : ETX101, Encoded's lead asset, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by FDA for the treatment of SCN1A+ Dravet Syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
July 22, 2020
Details:
ETX101, a gene therapy developed by Encoded Therapeutics to restore the levels of NaV1.1-alpha specifically in GABAergic neurons, is currently progressing toward clinical development for SCN1A-positive Dravet patients.
Lead Product(s): ETX101
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Cell & Gene Therapy
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable May 19, 2020
Lead Product(s) : ETX101
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Gene Therapy Shows Promise in Mouse Model of Dravet Syndrome
Details : ETX101, a gene therapy developed by Encoded Therapeutics to restore the levels of NaV1.1-alpha specifically in GABAergic neurons, is currently progressing toward clinical development for SCN1A-positive Dravet patients.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 19, 2020
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