What Encoded Therapeutics does

Encoded Therapeutics, Inc. is a clinical-stage biotechnology company founded in 2014 and headquartered in South San Francisco, California, with a secondary Research Triangle Park facility in Morrisville, North Carolina. The company develops one-time, AAV-based gene regulation therapies for severe neurological disorders, with a lead clinical program ETX101 for SCN1A+ Dravet syndrome delivered as a single intracerebroventricular infusion. Its core technology is the Genetic Medicines Product Engine, a proprietary platform that combines high-throughput next-generation sequencing (NGS) screening of human regulatory elements (promoters, enhancers, 3'UTRs) with machine learning to design cell-type-selective AAV cassettes, including REGABA (GABAergic interneuron-selective), NociPro (nociceptor-selective), and DRG de-targeting 3'UTR elements. Encoded also operates an internal GMP manufacturing ecosystem supplemented by preferred CDMO relationships, and pairs its in-house vector engineering with externally licensed BBB-crossing AAV variants where appropriate. Beyond ETX101, the pipeline includes ETX301 (preclinical AAV9-miRNA targeting SCN9A/NaV1.7 in dorsal root ganglion nociceptors for post-amputation neuroma pain), an Angelman syndrome UBE3A-unsilencing program, and an Alzheimer's/tauopathies MAPT-knockdown program.

Encoded is pre-revenue and does not currently offer pre-approval access anywhere in the world; its long-term revenue model is expected to be one-time, single-administration gene therapy product sales (with possible partnering income from co-development and licensing). Current go-to-market is exclusively via clinical trial distribution at specialized centers of excellence in the US (ENDEAVOR), UK (EXPEDITION), and Australia (WAYFINDER), supported by Medical Affairs outreach to healthcare professionals, a Patient Advocacy team engaged with the Dravet, Angelman, and broader pediatric CNS communities, and a Business Development channel for inbound partnering inquiries. The company has raised approximately $239M in disclosed venture funding (Series C of $104M in June 2019 and Series D of $135M in July 2020) from investors including ARCH Venture Partners, Venrock, Matrix Capital, SoftBank Vision Fund, GV, Illumina Ventures, Alexandria, Boxer Capital, Menlo Ventures, RTW, and others; no subsequent financing round is disclosed.

Name

Encoded Therapeutics

Legal name

Encoded Therapeutics, Inc.

Website

https://encoded.com

Company type

Private

Founded year

2014

Operating status

Operating

Headcount range

101–250 employees

Short description

Encoded Therapeutics is a clinical-stage biotechnology company developing one-time AAV-based gene regulation therapies for severe neurological disorders, with lead program ETX101 in pivotal trials for SCN1A+ Dravet syndrome.

Ownership category

akta.pro rank

Product category

Gene therapy for neurological disorders

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Gene Therapy Analytical Testing & QC (potency, vg/ddPCR, infectivity, RCL/RCA) (HLAAACAK)

Keywords

• AAV gene therapy
• Neurological disease treatment
• Precision genetic medicines
• Regulatory genome platform
• Cell-selective gene regulation

Headquarters

HQ city

South San Francisco

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Infrastructure, Operations, Supply Chain, Marketing or Sales

Revenue model

1. Pre-revenue / Future one-time gene therapy product sales: Encoded is a clinical-stage biotechnology company with no products currently approved or generating revenue. ETX101, ETX301 and pipeline programs are investigational. The expected long-term revenue model is one-time (single-administration) sales of AAV gene therapy products targeting monogenic and common neurological disorders. Encoded does not currently offer pre-approval access anywhere in the world. Future revenue may also include partnering income from co-development and licensing opportunities.

Pricing tiers

Go-to-market motion3 records

Distribution channels4 records

Core offering

Encoded Therapeutics is a clinical-stage biotechnology company developing one-time, AAV-based precision gene therapies that target the underlying genetic cause of severe neurological disorders. Its proprietary Genetic Medicines Product Engine uses high-throughput NGS screening of human regulatory elements combined with machine learning to design cell-selective cassettes (e.g., REGABA promoter, NociPro promoter, DRG de-targeting 3'UTRs) delivered via clinically validated AAV9 capsids. The lead investigational product, ETX101, is in pivotal development for SCN1A+ Dravet syndrome with five FDA designations (Breakthrough Therapy, RMAT, Fast Track, Rare Pediatric Disease, Orphan Drug) plus EMA Orphan designation.

Product overview

Encoded Therapeutics is a clinical-stage biotechnology company whose offering is organized as a single integrated R&D engine — the Genetic Medicines Product Engine — that generates and advances a wholly owned pipeline of one-time, precision AAV-based genetic medicines for severe neurological disorders. The platform combines high-throughput NGS screening with machine-learning-driven regulatory element optimization to engineer cell-type-selective AAV cassettes (e.g., REGABA for GABAergic neurons; NociPro for nociceptors; 3'UTR DRG de-targeting elements) and supports an internal GMP manufacturing ecosystem. The engine currently powers four named pipeline programs: the lead clinical-stage product ETX101 (AAV9 gene regulation therapy for SCN1A+ Dravet syndrome, advanced under the integrated POLARIS clinical program), the preclinical ETX301 (AAV9-miRNA for post-amputation neuroma pain, IND planned for 2027), a preclinical Angelman syndrome UBE3A-unsilencing program, and a preclinical Alzheimer's/tauopathies MAPT-knockdown program. Programs span monogenic (Dravet, Angelman) and common (chronic pain, tauopathies) neurological indications.

Differentiator

Problem solved

Functional benefit

Products and services

• ETX101 (for SCN1A+ Dravet Syndrome) Investigational AAV9-based, cell-selective gene regulation therapy for SCN1A+ Dravet syndrome. Delivered as a single intracerebroventricular (ICV) infusion, ETX101 uses an engineered transcription factor (eTF) under the GABAergic-selective REGABA promoter to upregulate endogenous SCN1A expression in inhibitory interneurons, restoring NaV1.1 sodium channel density. Designed for infants and young children aged 6 months to less than 4 years (pivotal Part 2) and ages 4 to 18 (Part 1B expansion). FDA Breakthrough Therapy, RMAT, Fast Track, Rare Pediatric Disease, and Orphan Drug designations plus EMA Orphan designation.
• ETX301 (for post-amputation neuroma pain) Investigational AAV9-based vectorized microRNA (miRNA) gene therapy for chronic neuropathic pain caused by post-amputation neuroma. Delivered via a single intrathecal injection using the NociPro nociceptor-selective promoter to knock down SCN9A (NaV1.7) in dorsal root ganglion nociceptive neurons. Targets the estimated 100,000 US patients with painful neuromas following lower limb amputation (~5,000 new cases annually). IND submission planned for 2027.
• Angelman Syndrome Program (UBE3A Unsilencing) Preclinical one-time, vectorized miRNA-based gene therapy designed to unsilence the paternal UBE3A gene by targeting the UBE3A antisense transcript (UBE3A-ATS). Recent preclinical data showed ~57% increase in UBE3A expression and ~24% increase in UBE3A protein across cortex, hippocampus, and thalamus in non-human primates using a blood-brain-barrier-crossing AAV variant, with 60-day tolerability.
• Alzheimer's Disease / Tauopathies Program Preclinical program applying Encoded's cell-selective AAV vector engineering to deliver miRNA-based knockdown of intracellular tau (MAPT), targeting Alzheimer's disease and related tauopathies. Addresses ~6 million people in the U.S. with Alzheimer's disease.
• Genetic Medicines Product Engine End-to-end platform encompassing preclinical development, GMP manufacturing, multi-modal delivery, and clinical expertise for AAV-based gene therapies. Uses high-throughput NGS screening and machine-learning-driven regulatory element optimization to engineer cell-type-selective cassettes (REGABA for GABAergic neurons; NociPro for nociceptors; 3'UTR DRG de-targeting elements). Internal GMP manufacturing facility complemented by preferred CDMO relationships. Supports a wholly owned, internally developed pipeline of neurological gene therapies.
• POLARIS Clinical Program (ENDEAVOR, EXPEDITION, WAYFINDER, ENVISION) Comprehensive ETX101 clinical development program comprising Phase 1/2 dose-escalation studies (ENDEAVOR Parts 1A and 1B, EXPEDITION, WAYFINDER), pivotal ENDEAVOR Part 2 (NCT05419492), the ENVISION natural history study (NCT04537832), the Dravet ENGAGE patient-focused initiative, and the ELUCIDATE biomarker discovery program. Conducted across the United States, United Kingdom, and Australia under FDA, MHRA, and TGA oversight.

Quantifiable outcome

• ~76% median monthly countable seizure frequency (MCSF) reduction at DL3 (n=3) sustained from Week 5 through Week 52
• +6 more outcomes

Named customers1 record

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability2 records

Feature7 records

Partnerships

Three partnerships are on record, tiered research collaboration, operational support and future potential.

• External AAV capsid/BBB-crossing variant licensor (Angelman program)research collaborationOEM/ Whitelabel/ Licensing PartnerFor the Angelman syndrome program, Encoded paired its optimized miRNA candidate with an externally-developed blood-brain-barrier-crossing AAV variant to achieve widespread miRNA expression across disease-relevant brain regions in non-human primates (~57% increase in UBE3A expression). The specific external licensor is not named in the source material.
• Preferred CDMO partnersoperational supportImplementation/ SI/ Consulting PartnerEncoded's GMP manufacturing ecosystem includes preferred CDMO relationships to complement its in-house GMP manufacturing facility. Encoded has invested in its own manufacturing capabilities since inception, with process development meeting current AAV commercial-scale requirements and infrastructure that meets GMP-scale requirements for multiple programs.
• Industry co-development and licensing partnersfuture potentialStrategic or Co-development PartnerEncoded states it aims to continue developing its pipeline utilizing end-to-end capabilities and leveraging its research platform to broaden therapeutic reach "independently as well as through industry partnerships". Company is actively evaluating co-development and licensing opportunities via [email protected].

Scale indicators14 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Stoke Therapeutics: Direct competitor in SCN1A+ Dravet syndrome. Stoke is developing STK-001, an antisense oligonucleotide that upregulates SCN1A expression — the same target as Encoded's ETX101 — using a different modality (ASO vs. AAV gene therapy). Stoke is the most clinically advanced competitor in Dravet syndrome and serves the same patient population and physician community.
• Taysha Gene Therapies: Clinical-stage AAV gene therapy company developing CNS-targeted genetic medicines. Notably, Encoded's Board Chairman Sean P. Nolan is also CEO of Taysha, creating direct leadership overlap. Both companies pursue AAV-based gene regulation therapies for severe neurological diseases with similar regulatory and manufacturing challenges.
• Voyager Therapeutics: AAV gene therapy company developing CNS-targeted treatments for neurodegenerative diseases. Encoded's CEO Kartik Ramamoorthi previously served on the team that launched Voyager. Both companies use AAV capsids to deliver gene regulation payloads to specific CNS cell types, and compete for similar scientific talent and clinical sites.
• Solid Biosciences: Clinical-stage AAV gene therapy company focused on Duchenne muscular dystrophy and CNS disorders. Similar to Encoded, Solid has faced safety setbacks, restructured operations, and rebuilt clinical programs — providing a comparable trajectory of a clinical-stage AAV gene therapy company navigating manufacturing scale-up and clinical development.
• Passage Bio: Clinical-stage AAV gene therapy company developing CNS-targeted treatments for rare monogenic neurological diseases. Passage Bio's portfolio (e.g., GM1 gangliosidosis, Krabbe disease, frontotemporal dementia) overlaps with Encoded's CNS focus and shares similar scientific, manufacturing, and regulatory challenges in delivering AAV payloads to the brain.

Broad incumbents

• AveXis (Novartis Gene Therapies): Established AAV gene therapy franchise within Novartis that developed and commercialized Zolgensma for spinal muscular atrophy — the benchmark one-time AAV therapy. Several Encoded executives (Sean Nolan, Andrew Stober) led AveXis through Zolgensma development, and AveXis represents the proven commercial and regulatory template for one-time AAV therapies in rare pediatric neurological disease.
• Audentes Therapeutics (Astellas Gene Therapies): AAV gene therapy unit of Astellas developing treatments for rare neuromuscular diseases (e.g., AT132 for X-linked myotubular myopathy). Encoded CMO Salvador Rico led AT132 clinical development at Audentes. Operates the same AAV gene therapy model — internal manufacturing, rare disease focus, complex regulatory pathway — though with some high-profile safety setbacks.
• Regenxbio: Established AAV gene therapy platform company that develops and licenses AAV capsid technology (including AAV9, used by Encoded for ETX101 and ETX301). Regenxbio is both a potential licensor/collaborator for Encoded and a competitor pursuing CNS and rare disease gene therapies using its proprietary NAV technology platform.
• uniQure: Established gene therapy company with commercial AAV product (Hemgenix for hemophilia B) and CNS-directed AAV programs. Provides a comparable template for an AAV gene therapy company advancing from clinical development through commercial launch, including pricing, manufacturing, and reimbursement strategy for one-time therapies.
• Pfizer (Gene Therapy Division): Large pharmaceutical company with a gene therapy portfolio that includes Beqvez (an AAV gene therapy for hemophilia B) and prior investments in AAV CNS programs. Represents a potential strategic acquirer or partner for Encoded's platform and pipeline, and a benchmark for large-pharma AAV manufacturing and commercialization capabilities.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/EncodedGenomics
• YouTubeyoutube.com/playlist?list=PLsPi6j2TogJVG4WIprDhxRf2IRG_Jzwqo
• LinkedInlinkedin.com/company/encodedtherapeutics

Compliance8 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Funding overview

Funding rounds6 records

Investors20 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Encoded Therapeutics