Encoded Therapeutics

Creating disease-modifying gene therapies for CNS disorders with our cell-selective targeting and regulation platform. Guidelines:

[Encoded Therapeutics @EncodedTx]

June is #DravetAwarenessMonth — an opportunity to raise awareness and recognize the experiences of individuals and families affected by Dravet syndrome.

Dravet syndrome is often described as a “severe epilepsy,” but its effects can extend beyond seizures, impacting development, behavior, sleep, mobility, and temperature regulation.

This month, we stand with the Dravet community and reaffirm our commitment to advancing science that brings real hope closer to reality.

Learn more at the Dravet Syndrome Foundation website: https://t.co/ja1aa6Q5Rf

#curedravet #advocatefordravet @curedravet

[Encoded Therapeutics @EncodedTx]

Great article from Endpoints News today highlighting our latest ETX101 data in #DravetSyndrome presented at #ASGCT2026.
Check out the article here: https://t.co/LcLFmr2HLX
https://t.co/LcLFmr2HLX

[Encoded Therapeutics @EncodedTx]

Appreciating the coverage from @BiotechTV on our latest ETX101 data in #DravetSyndrome presented at #ASGCT2026.
Thanks for helping share the story.

[BiotechTV @BiotechTV]

𝐀𝐒𝐆𝐂𝐓 𝟐𝟎𝟐𝟔: @EncodedTx presented updated clinical data today for its gene therapy program targeting Dravet Syndrome with 52 weeks of follow up. #ASGCT2026

Full video: https://t.co/GeMzfKTQnX

[Encoded Therapeutics @EncodedTx]

Big moment at #ASGCT2026 today! Read our release for details on ETX101 data in #dravetsyndrome being featured today in the Presidential Symposium presentation: https://t.co/0cAnQldELX

[Encoded Therapeutics @EncodedTx]

Today’s release shares preclinical data from #ASGCT2026 showing the potential of our precision vector engineering platform to enable one time gene therapies. https://t.co/41u60E6Gph

[Encoded Therapeutics @EncodedTx]

Announcing strong pipeline momentum with new milestones for ETX101 in Dravet syndrome and ETX301, our candidate for post-amputation neuroma pain.
Learn more: https://t.co/AmJjp6HaFa

[Encoded Therapeutics @EncodedTx]

Don’t miss Dr. Joe Sullivan, VP Clinical Development, speaking in Session 12 at #EILATXVIII on Developing Gene Therapies for DEEs on May 6.

[Encoded Therapeutics @EncodedTx]

Encoded will present data and insights from our POLARIS program for ETX101 for Dravet syndrome at #EILATXVIII on 5/ 4 from 4:15 – 4:35 CET. Learn more: https://t.co/nfWNFdGMCC

[Encoded Therapeutics @EncodedTx]

We’re back from Epilepsy Awareness Day presented by Sofie's Journey.

We’re grateful for the opportunity to connect with the epilepsy community in a meaningful way and for the insights that directly shape how we show up as partners.

Thank you to everyone – patients, physicians, and other collaborators – who spent time and shared their stories with us.

We’re inspired to carry this momentum forward — grounded in real experiences and focused on what matters most.

[Encoded Therapeutics @EncodedTx]

Encoded is headed to #ASGCT2026, where we’ll present three abstracts — an oral presentation in the Presidential Symposium highlighting continued progress with ETX101, and two scientific posters showcasing our vector engineering approach and its potential to deliver precision one time genetic medicines for neurological disorders. Details are below and in the link: https://t.co/tDQjbdwiXW

Oral Presentation: Safety and Efficacy of ETX101, an Investigational AAV9-based Gene Therapy for SCN1A+ Dravet Syndrome: Interim Results from the POLARIS Phase 1/2 Clinical Trials
• Session: General Session: Presidential Symposium
• Location: Exhibit Hall B1 (Exhibit Level)
• Date and Time: Wednesday, May 13, 2026; 2:26 PM – 2:37 PM ET

Poster Presentation: NociPro: A Novel Modality-Agnostic Promoter Platform for Precise Cellular Targeting of Nociceptor Sensory Neurons in Gene Therapy for Chronic Pain
• Abstract Number: 1312
• Location: Poster Hall
• Date and Time: Tuesday, May 12, 2026; 5:00 PM – 6:30 PM ET

Poster Presentation: An Experimental Intravenous AAV-miRNA-based Approach Achieves Broad Neuronal Transduction and UBE3A Unsilencing for the Treatment of Angelman Syndrome
• Abstract Number: 1468
• Location: Poster Hall
• Date and Time: Tuesday, May 12, 2026; 5:00 PM – 6:30 PM ET

[Encoded Therapeutics @EncodedTx]

Thank you, @neurology_live, for featuring our POLARIS program for ETX101 for Dravet syndrome in this month’s Clinical Trial in Focus. Every opportunity to elevate the science—and the needs of this community—helps move us closer to meaningful, lasting change for patients and caregivers. https://t.co/8RmtPvO09v

[Encoded Therapeutics @EncodedTx]

Today, we announced the successful completion of our Initial Comprehensive Multidisciplinary Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA, confirming the pivotal study design in 30 patients with Dravet syndrome and aligning on expansion of the Phase 1/2 open label study to include older children and adolescents. The initiation of both studies is already underway.

In addition, new data from the POLARIS clinical program of ETX101 will be presented during the Presidential Symposium at the #ASGCT2026 Annual Meeting taking place in Boston, from May 11 – 15, 2026.

We look forward to advancing ETX101 toward its next milestones and continuing our work on behalf of the Dravet community.

Learn more: https://t.co/0X3r8G1wry

[Encoded Therapeutics @EncodedTx]

This International Epilepsy Day, we’re proud to support efforts to raise visibility for epilepsy and deepen understanding for those affected.

We know it takes a community to advance new therapies, and we’re committed to working alongside patients and their families every step of the way — so that together we can transform hope into possibilities.

#epilepsyday

[Encoded Therapeutics @EncodedTx]

New insights from up to two-year ENVISION natural history data, published in Epilepsia, shed light on the early emergence and progression of behavioral and social–emotional challenges in young children with SCN1A+ Dravet syndrome: Problematic behaviors often begin before age 3 years and intensify through early childhood, spanning inattention, externalizing behaviors (including aggression), social withdrawal, and autistic features. Notably, poorer communication and adaptive skills were associated with more severe behavioral difficulties—underscoring the importance of early diagnosis to enable targeted management.

Scheffer IE, et al. Characterizing early behavioral and social-emotional problems in young children with SCN1A+ Dravet syndrome: Findings from the ENVISION prospective natural history study. Epilepsia. 2026 Feb 2. doi: 10.1002/epi.70127. Epub ahead of print. PMID: 41627953.

You can access the published article here: https://t.co/H8UkeO9I2D

#dravet, #dravetsyndrome, #naturalhistorystudy, #envision, #SCN1A+

[Encoded Therapeutics @EncodedTx]

Exciting news: The FDA granted Breakthrough Therapy Designation to ETX101 based on clinical evidence from the POLARIS Phase 1/2 trials.

This designation is reserved for therapies that show early signs of meaningful improvement and is intended to accelerate development and regulatory review. This marks an important milestone in advancing ETX101 with urgency and care for patients and families living with Dravet syndrome.

Read today’s release for more information: https://t.co/2rHGqiD1aa

#DravetSyndrome

[Encoded Therapeutics @EncodedTx]

Today, we provided a summary of the significant progress made in 2025 with our Dravet syndrome candidate, ETX101, including positive interim Phase 1/2 results presented at the American Epilepsy Society Annual Meeting, advances across our gene therapy pipeline, and anticipated 2026 milestones.

Read today’s press release for details: https://t.co/l3CARGIohm

#JPM2026 #DravetSyndrome #AES2026

[Encoded Therapeutics @EncodedTx]

Today, we announced that our CEO, Dr. Kartik Ramamoorthi, will present a corporate overview and 2026 outlook at the 44th Annual J.P. Morgan Healthcare Conference.

We hope to see you there!

Read today’s press release for details: https://t.co/UxEHODAMYD

#JPM2026