Dracaena’s owner, Dr Ana Mingorance, most recently co-authored a review of the current landscape and future directions in developing gene therapies for neurogenetic disorders.

Gene therapies for neurogenetic disorders.

Devinsky O, Coller J, Ahrens-Nicklas R, Liu XS, Ahituv N, Davidson BL, Bishop KM, Weiss Y, Mingorance A.

Trends Mol Med. September 2025

*NEW* Dravet Syndrome Pipeline and Opportunities Review 2024 provides an overview and analysis of the global therapeutic landscape of Dravet syndrome, as well as current and future opportunities of the Dravet syndrome market.

As of end 2024, there are three symptomatic treatments approved by both the FDA and the EMA for the treatment of seizures in Dravet syndrome: Diacomit (stiripentol), Epidiolex/Epidyolex (cannabidiol oral formulation) and Fintepla (fenfluramine). The first treatment specifically designed to increase SCN1A levels, zorevunersen (STK-001) from Stoke Therapeutics, is advancing to Phase 3 trials after positive Phase 2 data. The first viral gene therapy for SCN1A upregulation (ETX101, from Encoded Therapeutics) has recently initiated Phase 1/2 clinical studies. There are additional disease-modifying treatments in early and late preclinical development. Overall, the Dravet syndrome pipeline comprises 3 approved drugs and at least 25 additional treatments in development from preclinical stage to Phase 3 just counting industry-sponsored programs.

Our Hot Topics in Epilepsy Drug Development 2023 report provides an analysis of four major topics of relevance for drug development in epilepsy:

1. The transition from epilepsy trials to neurodevelopmental trials in DEEs.

2. The growing pipeline of Kv7 channel openers.

3. Advanced modalities in development for targeting drug-resistant focal epilepsies, with most of them targeting MTLE.

4. Growing challenges in clinical trial recruitment, with case studies for Dravet syndrome and focal onset seizures.

Dravet Syndrome Pipeline and Opportunities Review 2022 is a market intelligence publication that provides an overview of the global therapeutic landscape of Dravet syndrome, in addition to an analysis of the competitive landscape, unmet needs, and evaluates current and future opportunities of the Dravet syndrome market.

As of end 2022, the Dravet syndrome pipeline comprises 3 approved drugs, 16 drug candidates, and 15 different products have received orphan drug designations. Since the 2020 report, the pipeline has changed by, among others, the multi-billion acquisitions of GW Pharma and Zogenix by Jazz Pharma and UCB Pharma respectively, Takeda taking sole ownership of soticlestat (currently in Phase 3), and the publication of preliminary efficacy data for the first treatment specifically designed to target the cause of Dravet syndrome.

Dravet Syndrome Pipeline and Opportunities Review 2020 is a market intelligence publication that provides an overview of the global therapeutic landscape of Dravet syndrome, in addition to an analysis of the competitive landscape, unmet needs, and evaluates current and future opportunities of the Dravet syndrome market.

As of end 2020, the Dravet syndrome pipeline comprises 3 approved drugs, 11 drug candidates, and 12 different products have received orphan drug designations. Since the last report the pipeline has changed by, among others, the approval by FDA and EMA of Epidyolex (cannabidiol) and Fintepla (fenfluramine), the initiation of the first clinical trial with an antisense therapy, and progresses in different disease-targeting modalities including gene therapy approaches.

Dravet Syndrome Pipeline and Opportunities Review 2019 is a market intelligence publication that provides an overview of the global therapeutic landscape of Dravet syndrome, in addition to an analysis of the competitive landscape, unmet needs, and evaluates current and future opportunities of the Dravet syndrome market.

As of August 2019, the Dravet syndrome pipeline comprises 12 drug candidates, and 11 different products have received orphan drug designations. In the last 12 months the pipeline has changed by, among others, the approval by the FDA of Diacomit (stiripentol), the CHMP positive opinion on Epidyolex (cannabidiol) approval, the IPO from Stoke Therapeutics and the arrival of a new company pursuing a gene therapy approach for Dravet syndrome (Encoded Therapeutics).

Dravet Syndrome Pipeline and Opportunities Review 2018 is a market intelligence publication that provides an overview of the global therapeutic landscape of Dravet syndrome, in addition to an analysis of the competitive landscape, unmet needs, and evaluates current and future opportunities of the Dravet syndrome market.

As of July 2018, the Dravet syndrome pipeline comprises 14 drug candidates, and 9 different products have received orphan drug designations. In the last 12 months the pipeline has changed by, among others, the approval by the FDA of Epidiolex (cannabidiol) by GW Pharmaceuticals for the treatment of Dravet syndrome, the announcement of positive data from two Phase III clinical trials with ZX008 from Zogenix, and the arrival of a new company pursuing a disease-modifying antisense-based approach (Stoke Therapeutics). 

Dravet Syndrome Pipeline and Opportunities Review 2017 is a market intelligence publication that provides an overview of the global therapeutic landscape of Dravet syndrome, in addition to an analysis of the competitive landscape, unmet needs, and evaluates current and future opportunities of the Dravet syndrome market.

As of June 2017, 8 different products have received orphan drug designations for Dravet syndrome, there are 3 drug candidates in late-stage placebo-controlled Phase II or III studies, and 2 of the products in development are potentially disease-modifying treatments. The pipeline report includes the most recent updates on programs from GW Pharmaceuticals, Zogenix, PTC Therapeutics, Biscayne Neurotherapeutics, OPKO Health, Ovid Therapeutics, Takeda, Epygenix Therapeutics, INSYS Therapeutics, Xenon Pharmaceuticals, Sage Therapeutics and Lundbeck.