One raise, three uses: the pivotal program, in-house manufacturing, and pipeline work including ETX301's path to an IND
Encoded raised $275 million to take its Dravet gene therapy through a pivotal study.
The same round pays to scale up its own commercial manufacturing while that study is still running.
Encoded Therapeutics announced the close of a $275 million Series F on September 9. The money will support pivotal and expansion studies of ETX101, an investigational AAV9-based gene regulation therapy for SCN1A+ Dravet syndrome designed as a one-time treatment. It will also support commercial scale-up of in-house GMP manufacturing and pipeline advancement, including ETX301 toward a 2027 IND submission.
What was announced
Encoded Therapeutics is a privately held biotech in South San Francisco. On September 9 it announced the close of a $275 million Series F, co-led by GV and a second healthcare fund the company did not name.
Eleven more investors took part, among them ARCH Venture Partners, Farallon Capital Management, RTW Investments, SoftBank Vision Fund 2 and Venrock. The full list is in the FAQ below.
For a therapy whose pivotal study is still running, the notable part of this round is that it buys more than trials.
Where the money goes, per Encoded
- ETX101 pivotal study: infants and young children with SCN1A+ Dravet syndrome. The pivotal ENDEAVOR Part 2 study, evaluating children aged 6 months to 4 years, is ongoing.
- ETX101 expansion study: children and adolescents up to 18 years of age.
- Manufacturing: commercial scale-up of internal GMP manufacturing capabilities.
- Pipeline: pipeline advancement, including ETX301 toward a 2027 IND submission for post-amputation neuroma pain.
- Split between these uses: not disclosed.
CEO Kartik Ramamoorthi said the financing gives Encoded "the resources to advance ETX101 toward registration" with pivotal development already underway.
The data Encoded released the day before the round
The raise was announced a day after Encoded's September 8 release on updated Phase 1/2 POLARIS data, presented at the 16th European Epilepsy Congress in Athens. The analysis uses an August 3, 2026 data cutoff.
From week 5 through week 52, or each participant's latest visit, the median reduction in monthly countable seizure frequency was about 76% at dose level 3 (n=5). At dose level 4 (n=9) it was about 60%. Among participants who completed 52 weeks, the month-12 median was about 79% at dose level 3 (n=3) and 89% at dose level 4 (n=5).
At dose level 4, about 60% median seizure reduction across weeks 5 to 52 (n=9), and about 89% at month 12 (n=5).
These are interim Phase 1/2 results from small, open-label cohorts, and the figures are the sponsor's own. The month-12 numbers rest on eight participants in total; the reported week 5 to 52 figures cover 14, and the safety data 21.
Dose level 4 sits below dose level 3 in the cumulative window and above it at month 12. The month-12 figures cover only participants who completed 52 weeks: five of nine at dose level 4 and three of five at dose level 3. The two windows differ in both people and weeks, so neither ordering yet says much about dose response.
Encoded also reports gains in cognition and adaptive behaviour. In participants treated before age two, Bayley-4 cognitive scores diverged from the ENVISION natural history study with up to 76 weeks of follow-up. VABS-3 scores improved across all evaluated domains, which Encoded describes as clinically meaningful.
On safety, Encoded reports no treatment- or procedure-related serious adverse events across all four dose levels, with up to 117 weeks of follow-up. Treatment-related adverse events included transaminase elevations in 7 of 21 participants and thrombocytopenia in 3 of 21, both clinically asymptomatic and resolved in all participants.
What ETX101 is
ETX101 is an investigational AAV9-based gene regulation therapy, designed to increase expression of the SCN1A gene and restore sodium channel function in inhibitory interneurons. It is given once, by intracerebroventricular injection.
It holds FDA Breakthrough Therapy, RMAT, Fast Track, Rare Pediatric Disease and Orphan Drug designations, plus EMA Orphan designation. It was also selected for the FDA's CMC Development and Readiness Pilot, or CDRP.
Of that list, the CDRP selection bears most on this round. It is an FDA pilot on chemistry, manufacturing and controls (CMC) readiness, and this round will support manufacturing scale-up as well as trials.
What this means for a CGT program
GTC analysis: on CMC, the choice is when to pay. Encoded is paying for commercial manufacturing scale-up now, from this Series F, rather than leaving it to a raise after the readout.
GTC analysis: for a sponsor that uses a CDMO, the same decision arrives as a vendor question. When is the commercial site committed, and do pivotal lots come from it?
If you have planned a commercial manufacturing site around a pivotal study, GTC's Expert Network matches CMC experts like you to paid consulting work: join free.
GTC analysis: the two age ranges set the regulatory order. The pivotal study evaluates children aged 6 months to 4 years, while the expansion study runs up to age 18. If ETX101 is approved on the pivotal study, the youngest patients are the ones its evidence covers first, and older patients depend on what the expansion study adds.
GTC analysis: three figures would make this round a usable benchmark, and neither release gives them. They are the split of the round, a pivotal readout date and manufacturing capacity.
Frequently asked questions
How much did Encoded Therapeutics raise, and who led the round?
Encoded Therapeutics announced the close of a $275 million Series F financing on September 9, 2026. GV and another healthcare fund, not named in the announcement, co-led the round. ARCH Venture Partners, Braidwell LP, Farallon Capital Management, Illumina Ventures, Invus, Janus Henderson Investors, Matrix Capital Management, Nolan Capital, RTW Investments, SoftBank Vision Fund 2 and Venrock also took part.
What will the $275 million fund?
Encoded says the proceeds will support the pivotal study of ETX101 in infants and young children with SCN1A+ Dravet syndrome. They will fund an expansion study in children and adolescents up to 18 years of age as well. The funding will also support commercial scale-up of its internal GMP manufacturing and pipeline advancement, including ETX301 toward a 2027 IND submission for post-amputation neuroma pain. The announcement does not say how the money is split between these uses.
What is ETX101?
ETX101 is an investigational AAV9-based gene regulation therapy designed to increase expression of the SCN1A gene and restore sodium channel function in inhibitory interneurons. It is given as a single intracerebroventricular injection. It holds FDA Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations, plus EMA Orphan designation. It was also selected for the FDA's CMC Development and Readiness Pilot program.
What did the latest POLARIS data show?
Encoded presented interim data at the 16th European Epilepsy Congress, with an August 3, 2026 cutoff. From week 5 through week 52 or the latest visit, the median reduction in monthly countable seizure frequency was about 76% at dose level 3 (n=5). At dose level 4 (n=9) it was about 60%. Among participants with 52 weeks of observation, the month-12 median reduction was about 79% at dose level 3 (n=3) and 89% at dose level 4 (n=5). Encoded reports no treatment- or procedure-related serious adverse events with up to 117 weeks of follow-up.
Where does the pivotal study stand?
The pivotal ENDEAVOR Part 2 study is ongoing, evaluating seizure and neurodevelopmental outcomes in children aged 6 months to 4 years. Encoded's CEO, Kartik Ramamoorthi, said the financing gives the company the resources to advance ETX101 toward registration. Neither of the two September releases reports pivotal results.
Sources
- Encoded Therapeutics Raises $275 Million in Series F Financing to Advance ETX101 Through Pivotal Development and Accelerate its Precision Genetic Medicines Neurology Pipeline – Encoded Therapeutics, press release, South San Francisco, September 9, 2026.
- 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 Therapeutics, press release, September 8, 2026.
- Encoded raises $275 million to push Dravet gene therapy into pivotal testing – The Pharma Letter, September 10, 2026.
Join the GTC Expert Network
Independent Experts
Independent experts in cell & gene therapy: share knowledge, help build a report, or contribute quietly. Build authority and get called for paid consulting work. Join free.
Early R&D Experts
Early R&D ExpertsContribute target validation, vector engineering, or transgene design expertise. Get matched to paid consulting work through content you help create.
Join FreeCMC Experts
CMC ExpertsContribute scale-up, tech transfer, or GMP manufacturing expertise. Get matched to paid consulting work through content you help create.
Join FreeRegulatory Affairs Experts
Regulatory Affairs ExpertsContribute IND/BLA strategy or regulatory liaison expertise. Get matched to paid consulting work through content you help create.
Join FreeCommercial & Strategy Experts
Commercial & Strategy ExpertsContribute due diligence, licensing, or market access expertise. Get matched to paid consulting work through content you help create.
Join FreeEnabling Technology Experts
Enabling Technology ExpertsContribute bioprocessing hardware, analytical tooling, or supply-chain expertise. Get matched to paid consulting work through content you help create.
Join FreeAI Transformation Experts
AI Transformation ExpertsContribute agentic workflow, LLM, or AI-driven vector design expertise. Get matched to paid consulting work through content you help create.
Join FreeIndustry Representatives
Industry professionals in cell & gene therapy: de-risk your pipeline with expert input on demand, exactly when your program needs it. Join free.
Drug Developers
Drug DevelopersFounders, CSOs, VPs of R&D
Practical guides spanning vector design through commercialization, plus direct access to the expert network that wrote them.
Join FreeInvestors / VCs
Investors / VCsVC Fund Managers, Family Offices, Angels
Due diligence frameworks and strategic intelligence for evaluating CGT assets, from experts who do this diligence for a living.
Join FreeTechnology Developers
Technology DevelopersFounders, Licensing & Commercial Leads
IP protection and platform strategy guides for enabling technologies, plus the network that built them.
Join FreeCDMOs / CROs
CDMOs / CROsBD, Operations & Technical Leads
Partner selection, vendor evaluation, and operational excellence guides, sourced from the CGT organisations that use them.
Join FreeVendors / Suppliers
Vendors / SuppliersCommercial & Technical Leads
Market intelligence and partnership strategies for supply chain companies serving cell and gene therapy.
Join FreeStrategy Consultants
Strategy ConsultantsConsultants, Market Access Advisors
Spot the problems before they cost your clients — join the network that shapes what GTC covers next.
Join FreeAI Transformation Partners
AI Transformation PartnersAI/ML Consultancies, Digital Transformation Firms
AI education, implementation, and consulting for operational excellence in cell and gene therapy — for companies building or buying AI capability.
Join FreeWhere the analysis comes from. Industry teams get expert input on demand; independent experts build authority and get called for paid consulting work.
See more of our work in Google Search
Google lets you pick the sites you want to see more often in Top Stories. Add Gene Therapy Consultancy to your list, and our daily gene therapy news will show up higher for you.