Category: Press Releases

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics today announced the upcoming presentation of initial data from the ENVISION prospective natural history study of children with SCN1A+ Dravet syndrome, a rare developmental and epileptic encephalopathy, at the American Epilepsy Society 2021 Annual Meeting, taking place in Chicago from December 3-7, 2021. Additionally, Encoded will present on its ongoing DRAVET ENGAGE initiative, which aims to capture the Dravet syndrome patient community’s experiences and perspectives, and a summary of molecular diagnostic and phenotypic findings in people with SCN1A+ Dravet syndrome identified through genetic testing.

Title: ENVISION: An International, Prospective Natural History Study in Young Children With SCN1A+ Dravet Syndrome

Abstract number: 2.222

Date: Sunday, December 5th

Platform presentation details: #B.07 | 10-10:15 a.m. CT | W475, Level 4

Poster session details: 12-2 p.m. CT | Hall F1, Level 3

Presenting author: Andreas Brunklaus, M.D., Consultant Pediatric Neurologist at the Royal Hospital for Children, Glasgow, United Kingdom

Title: A Patient-Oriented Approach to Integrating Families’ Experiences in Clinical Trials Design and Clinical Care for Dravet Syndrome

Abstract number: 3.073

Date/time: Monday, December 6th | 12-1:45 p.m. CT

Location: Hall F1, Level 3

Presenting author: Emma James, Ph.D., M.F.P.M. (Hon), VP, Medical & Patient Affairs at Encoded Therapeutics

Title: Molecular Diagnostic and Phenotypic Findings in Individuals With SCN1A-Related Disease Identified Through a Sponsored, No-Charge Epilepsy Genetic Testing Program

Abstract number: 3.351

Date/time: Monday, December 6th | 12-1:45 p.m. CT

Location: Hall F1, Level 3

Co-presenting author: Jennifer Gorzelany, Head of Strategy at Encoded Therapeutics

About ETX101

Encoded is developing ETX101 as a potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome in hopes of addressing the full range of disease manifestations. In ETX101, a cell-selective regulatory element and a transgene coding for an engineered transcription factor are delivered in a clinically validated adeno-associated virus (AAV) capsid to upregulate, or increase, the expression of the endogenous SCN1A gene in the brain. This approach is expected to increase production of Na_V1.1 protein and density of sodium channels in target neurons, leading to restored function. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. Encoded anticipates treating the first participant in the ENDEAVOR interventional trial in 2022.

About the ENVISION Natural History Study

ENVISION is an ongoing observational study of infants and children with SCN1A+ Dravet syndrome. This prospective natural history study is designed to further define the seizure, neurodevelopmental, motor and behavioral manifestations of SCN1A+ Dravet syndrome in children aged 6 to 60 months with SCN1A mutations. The study will examine these characteristics over two years using standardized assessments and will also explore the impact of the disease on parents/caregivers and healthcare resource utilization. These findings will help identify appropriate scales and endpoints to assess ETX101 safety and efficacy in clinical trials. For more information about ENVISION (NCT04537832), visit clinicaltrials.gov and search for “Encoded Therapeutics”.

About Dravet Syndrome

Dravet syndrome is a severe, lifelong disorder of the central nervous system that occurs in approximately 1 in 16,000 births worldwide, with the majority of cases resulting from loss-of-function mutations in the SCN1A gene. This rare developmental and epileptic encephalopathy equally affects people of both sexes and all races, manifesting in a wide array of symptoms. Frequent, prolonged and difficult-to-treat seizures typically begin in the first year of life of a previously healthy baby. Additional symptoms, such as cognitive delays, sleep abnormalities, motor impairment and behavioral difficulties, compound the effects of the disease when they appear, often in the second or third year of a child’s life. More information about Dravet syndrome can be found at www.dravetfoundation.org.

About Encoded Therapeutics

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com, and follow us on LinkedIn, Twitter @EncodedTx and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211123005526/en/

Lynnea Olivarez

lolivarez@encoded.com

956-330-1917

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics today announced initial findings from its ENVISION natural history study of children living with SCN1A+ Dravet syndrome, a rare developmental and epileptic encephalopathy. Along with a substantial seizure burden, preliminary findings suggest the trajectory and timing by which children with Dravet syndrome deviate from neurotypical peers, showing that these children experience early and substantial delay in adaptive functioning, communication and socialization, worsening with age. These data and others from 46 study participants are being presented this weekend in Chicago at the American Epilepsy Society (AES) 2021 Annual Meeting.

“The primary goal of ENVISION is to elucidate Dravet syndrome progression during a critical period of child development by further defining the full range and evolution of disease manifestations, as well as their impact on young children and families living with this disease,” said Andreas Brunklaus, M.D., Consultant Pediatric Neurologist at the Royal Hospital for Children in Glasgow, United Kingdom. Dr. Brunklaus is a leading Dravet syndrome expert and presenting author of the ENVISION study data at the AES 2021 Annual Meeting. “ENVISION is generating a comprehensive contemporary dataset that evidences the profound neurodevelopmental stagnation in young children with Dravet syndrome. We hope that data over time will enable the community to refine our understanding of Dravet syndrome pathophysiology, paving the way for potential new disease-modifying medicines to be administered at a time point in young children’s lives that could change the course of disease as we know it.”

One of the key findings to date is that, regardless of SCN1A variant type, there was global evidence of developmental stagnation as young as two years of age across domains of communication, language, socialization, and motor and adaptive functioning compared with neurotypical peers. Researchers also observed that seizure frequency continues to increase with age, despite the use of multiple and newer antiseizure medications.

“We have heard directly from families in the Dravet syndrome community that seizures and communication significantly impact quality of life, and that preservation of neurodevelopment is a key outcome sought from a gene therapy,” said Salvador Rico, M.D., Chief Medical Officer at Encoded. “These preliminary ENVISION data reinforce the ongoing impact that Dravet syndrome has on the lives of people and their families, and the need for a therapy that addresses the underlying cause of disease to positively affect both seizure and neurocognitive symptoms. Though we will gain further insights through longitudinal data as ENVISION advances, we believe that early administration of a potential one-time disease-modifying therapy such as ETX101 could yield the maximum benefit.”

“We are thankful to the children and families participating in ENVISION and to all the experts and health professionals involved in the study for their ongoing contributions that are critically important to understand the progression of Dravet syndrome,” Dr. Rico continued.

Encoded will share additional learnings from its ongoing DRAVET ENGAGE initiative, which aims to capture the Dravet syndrome patient community’s experiences and perspectives, in a separate presentation at the AES 2021 Annual Meeting. For a full list of Encoded’s presentations, click here. All abstracts are available on the AES website.

About ETX101

Encoded is developing ETX101 as a potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome, in hopes of addressing the full range of disease manifestations. In ETX101, a cell-selective regulatory element and a transgene coding for an engineered transcription factor are delivered in a clinically validated adeno-associated virus (AAV) capsid to upregulate, or increase, the expression of the endogenous SCN1A gene in the brain. This approach is expected to increase production of Na_V1.1 protein and density of sodium channels in target neurons, leading to restored function. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. Encoded anticipates treating the first participant in the ENDEAVOR interventional trial in 2022.

About the ENVISION Natural History Study

ENVISION is an ongoing observational study of infants and children with SCN1A+ Dravet syndrome. This prospective natural history study is designed to further define the seizure, neurodevelopmental, motor and behavioral manifestations of SCN1A+ Dravet syndrome in children aged 6 to 60 months with SCN1A mutations. The study will examine these characteristics over two years using standardized assessments and will also explore the impact of the disease on parents/caregivers and healthcare resource utilization. These findings will help identify appropriate scales and endpoints to assess ETX101 safety and efficacy in clinical trials. For more information about ENVISION (NCT04537832), visit clinicaltrials.gov and search for “Encoded Therapeutics”.

About Dravet Syndrome

Dravet syndrome is a severe, lifelong disorder of the central nervous system that occurs in approximately 1 in 16,000 births worldwide, with the majority of cases resulting from loss-of-function mutations in the SCN1A gene. This rare developmental and epileptic encephalopathy equally affects people of both sexes and all races, manifesting in a wide array of symptoms. Frequent, prolonged and difficult-to-treat seizures typically begin in the first year of life of a previously healthy baby. Additional symptoms, such as cognitive delays, sleep abnormalities, motor impairment and behavioral difficulties, compound the effects of the disease when they appear, often in the second or third year of a child’s life. More information about Dravet syndrome can be found at www.dravetfoundation.org.

About Encoded Therapeutics

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com, and follow us on LinkedIn, Twitter @EncodedTx and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211203005088/en/

Lynnea Olivarez

lolivarez@encoded.com

956-330-1917

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics announced nonclinical data being presented today at the American Society for Cell and Gene Therapy 25^th Annual Meeting showing how its proprietary human genomic regulatory element (RE) engineering platform has been used to develop cell-selective expression vectors for targeted gene therapy. The company efficiently discovered a spectrum of sequence-encoded genetic elements, including enhancers, promoters and UTR elements, that drive selective expression profiles in mice. These REs function within the size constraints and episomal architecture of adeno-associated viruses (AAVs) and are compatible with multiple capsids and gene delivery systems.

“By combining human genomic regulatory elements to customize expression profiles and minimize off-target effects, we aim to improve the safety and efficacy of gene therapies for a broad range of monogenic and non-monogenic diseases in the future,” said Encoded CEO Kartik Ramamoorthi, Ph.D. “Our regulatory elements engineering approach increases cell-selective expression, reducing toxicity concerns in tissues like the liver. The data we’re sharing today at ASGCT are exemplary of the Encoded platform’s ability to achieve appropriately targeted transgene expression across many other central nervous system (CNS) cell types, like dorsal root ganglia (DRG) neurons, as well as non-CNS cell types .”

Using Encoded’s genomic medicine platform, researchers applied both expression-based functional screening and computational modeling to simultaneously test thousands of genomic elements in vivo. They uncovered sequence elements that selectively decrease liver expression while maintaining CNS expression in mice. Additionally, the research team constructed predictive models to rapidly and iteratively continue to improve the discovery rate and distribution of activity profiles, resulting in further reduction of liver expression in mice, with unchanged expression in the brain.

Click here for the abstract of Encoded’s ASGCT presentation, “Optimized Human Regulatory Sequences Achieve Targeted Expression in CNS and Decreased Liver Expression in Mice.”

About Encoded Therapeutics

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com, and follow us on LinkedIn, Twitter @EncodedTx and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220518005469/en/

Lynnea Olivarez

lolivarez@encoded.com

956-330-1917

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics announced today the presentation of non-human primate (NHP) validation of the company’s technology to drive desired gene and protein expression patterns at the American Society of Gene and Cell Therapy (ASGCT) 26^th Annual Meeting. An important challenge of developing gene therapy in many central nervous system (CNS) disorders is the toxicity concern associated with high transgene expression in the dorsal root ganglion (DRG). By incorporating a DRG de-targeting regulatory element (RE) derived from Encoded’s research platform into a CNS-targeted gene therapy construct, the company achieved a significant reduction in DRG protein expression to endogenous baseline. This resulted in a greater than 10-fold selective reduction in DRG transcriptional activity without impacting brain expression.

“Off-target expression in the DRG and other organs has created limitations on gene therapy efficacy optimization,” said Encoded CEO Kartik Ramamoorthi, Ph.D. “In addition to de-targeting the DRG and other organ systems, we believe that Encoded REs have the potential to drive high levels of cell-type selectivity, potency and specificity, irrespective of the gene modulation approach at hand. We’re incorporating this technology into our pipeline to unlock novel indications, and we hope to have an even greater impact for people living with CNS diseases by collaborating with other gene therapy developers in the future.”

Encoded tested over 10,000 REs in vivo with its high-throughput NGS-based platform to screen for human genomic sequences that can modulate transgene expression and produce a more highly targeted gene therapy expression profile in mice. The research team analyzed top candidates in NHPs, showing successful translation to higher species while still achieving precise expression in the CNS.

Click here for the abstract of Encoded’s 2023 ASGCT presentation, “High Throughput Discovery of Optimized Human Genomic Regulatory Elements That Selectively Decrease Off-Target Expression in Dorsal Root Ganglion in Mice and Non-Human Primates” (Abstract No. 1481).

About Encoded Therapeutics

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230519005038/en/

communications@encoded.com

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome. Individuals with Dravet syndrome, the most common developmental and epileptic encephalopathy, experience a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation. Dravet syndrome is primarily caused by loss-of-function variants in the SCN1A gene, accounting for over 85% of cases. ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons and potentially address the underlying cause of the disease.

ETX101 Global Clinical Development

Encoded has received clearance for its Investigational New Drug (IND) application from the US Food and Drug Administration and approval under the Clinical Trial Approval (CTA) scheme from the Australia Therapeutic Goods Administration to initiate clinical trials of its gene therapy candidate, ETX101. The company plans to initiate ENDEAVOR, a two-part, Phase 1/2 clinical trial in US patients 6 months to <3 years of age with SCN1A+ Dravet syndrome in the first half of 2024. WAYFINDER, a Phase 1/2 trial in patients 3 to <7 years of age in Australia, will be conducted concurrently with ENDEAVOR.

“Dravet syndrome is a devastating disorder that necessitates a paradigm shift from conventional symptomatic management towards precise and targeted interventions at the genetic level. I’m hopeful that gene therapy will not just alleviate symptoms but address the root cause of Dravet syndrome, marking a potentially transformative approach in our quest to address the ongoing medical burden of families living with Dravet syndrome,” said Joseph Sullivan, M.D., Murphy Parker Endowed Professor in Pediatric Epilepsy, Professor of Neurology & Pediatrics at UCSF Pediatric Epilepsy Center of Excellence and Principal Investigator on the ENDEAVOR study.

The ENDEAVOR and WAYFINDER clinical trials are part of Encoded’s global clinical development program, POLARIS, which aims to assess safety and efficacy of ascending doses of ETX101 in infants and young children. Importantly, POLARIS will evaluate the initial effects of ETX101 on seizure burden, as well as potential long-term improvements in neurodevelopment. Furthermore, Encoded has aligned with FDA on the design of ENDEAVOR as a two-part study, which creates an opportunity to seamlessly transition to a potentially confirmatory trial following demonstration of initial safety and efficacy.

The POLARIS program is based on preceding patient-focused drug development initiatives (Dravet ENGAGE), a multipronged biomarker discovery project (ELUCIDATE) and a recently completed natural history study (ENVISION), the largest longitudinal natural history study in Dravet syndrome to date. These initiatives have informed the optimal clinical design for POLARIS and emphasize the urgent unmet need for disease-modifying therapies.

“ETX101 represents a groundbreaking advancement in the therapeutic landscape for Dravet syndrome, with potential not only for seizure management but also for addressing the broader spectrum of non-seizure manifestations. ENDEAVOR and WAYFINDER are the first step in bringing a potentially one-time, disease-modifying gene therapy to the Dravet community, and we are excited to be partnering with leading experts in the care of patients with Dravet syndrome to begin clinical trials in the US and Australia in the coming months,” said Sal Rico, M.D., Ph.D., Chief Medical Officer.

Encoded will be providing additional updates on its research pipeline, which is focused on both rare genetic and common disease programs, in addition to clinical progress with ETX101 for Dravet syndrome later in 2024.

“Encoded is committed to creating innovative genetic medicines that bring renewed hope and possibilities to patients living with severe CNS disorders. The initiation of clinical trials in Dravet syndrome and the advancement of our CNS pipeline strategically positions Encoded to achieve pivotal program milestones in 2024. We look forward to sharing additional company updates in the coming months,” said Kartik Ramamoorthi, Ph.D., Co-Founder and Chief Executive Officer.

About ETX101

Encoded is developing ETX101 as a potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome. In ETX101, a transgene encoding an engineered transcription factor under the control of a cell-selective regulatory element is delivered within a clinically-validated capsid (AAV9) to upregulate, or increase, the expression of the endogenous SCN1A gene. This approach is expected to increase production of Na_V1.1 protein sodium channels in target neurons in the brain, leading to restored function. By targeting the underlying mechanism, ETX101 has the potential to address the full range of symptoms associated with Dravet syndrome. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the US Food and Drug Administration and Orphan Designation by the European Medicines Agency.

About Encoded’s ETX101 Clinical Development Program POLARIS

POLARIS is built upon Encoded’s comprehensive preclinical research, and incorporates a multipronged biomarker discovery program (ELUCIDATE), patient-focused drug development initiatives (Dravet ENGAGE), and the recently completed natural history study (ENVISION), the largest prospective, longitudinal natural history study of Dravet patients to date. This foundational work has enabled optimization of POLARIS’ rigorous clinical design, beginning with ENDEAVOR and WAYFINDER, to assess the safety and efficacy of ETX101 in infants and young children with SCN1A+ Dravet syndrome.

ENDEAVOR Clinical Study (United States)

ENDEAVOR is a two-part Phase 1/2 dose escalation study of ETX101 in infants and young children aged 6 months to <3 years. In Part 1, up to two doses of ETX101 will be evaluated in 4 participants. The primary aims of the study are to evaluate the safety and tolerability of ETX101, to assess preliminary efficacy, and to contribute to therapeutic dose selection. ETX101 has received IND clearance, and ENDEAVOR Part 1 is expected to begin in the first half of 2024. Part 2 is planned following demonstration of safety and efficacy in ENDEAVOR Part 1.

WAYFINDER Clinical Study (Australia)

WAYFINDER is a Phase 1/2 dose escalation study of ETX101 in young children aged 3 to <7 years with SCN1A+ Dravet syndrome. Up to two doses of ETX101 will be evaluated in 4 participants. The primary aims of the study are to evaluate the safety and tolerability of ETX101, to assess preliminary efficacy, and to contribute to therapeutic dose selection. WAYFINDER has received TGA approval and is expected to begin in the first half of 2024.

About Dravet Syndrome

Dravet syndrome is a severe, lifelong disorder of the central nervous system that occurs in approximately 1 in 16,000 births worldwide, with the majority of cases resulting from loss-of-function variants in the SCN1A gene. This developmental and epileptic encephalopathy equally affects people of both sexes and all races, manifesting in a wide array of symptoms. Frequent, prolonged, and treatment-resistant seizures primarily begin in the first year of life of a typically developing infant. Severe cognitive and developmental stagnation, sleep abnormalities, motor impairment and behavioral difficulties usually manifest by the second or third year of a child’s life. More information about Dravet syndrome can be found at www.dravetfoundation.org.

About Encoded Therapeutics

Encoded Therapeutics is creating one-time, potentially disease-modifying gene therapies to improve the lives of people with severe CNS disorders. Our cell-selective targeting and regulation platform offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. We are overcoming key limitations of current gene therapies by incorporating platform innovations into therapeutic development and advancing potentially best-in-class programs in Dravet syndrome and additional CNS disorders. For more information, please visit www.encoded.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240206685753/en/

Jennifer Gorzelany

communications@encoded.com

650-515-9695

SOUTH SAN FRANCISCO, Calif., June 26, 2019 /PRNewswire/ — Encoded Therapeutics, Inc. (Encoded), a precision gene therapy company, today announced a $104 million Series C financing which will be used to develop and commercialize innovative therapeutics with the potential to transform the treatment paradigm for severe genetic disorders. Incubated by Illumina Accelerator and seeded by Venrock and ARCH Venture Partners, Encoded unveiled its precision gene therapy platform, which has the potential to enable the development of gene therapies with greater cell-type selectivity, increased potency, and the ability to modulate the expression of endogenous genes. Encoded’s approach addresses key limitations of current gene therapy technology, unlocking new treatment opportunities.

The Series C financing includes investments from existing shareholders Venrock, ARCH Venture Partners, Matrix Capital Management, Illumina Ventures, and Altitude Life Science Ventures. New investors include Menlo Ventures, RTW Investments, Boxer Capital of Tavistock Group, and Alexandria Venture Investments. Encoded will use the funds to advance its lead program in Dravet Syndrome and its preclinical pipeline, as well as to leverage its platform to develop new therapeutics to treat severe genetic disorders.

“Our mission is to develop and commercialize life-changing therapeutics for severe genetic disorders that are not addressable with existing gene therapy approaches,” said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D. “The support we have received from this visionary group of investors will allow us to develop into a fully-integrated therapeutics company and establish Encoded as a leading innovator in gene therapy.”

Encoded’s approach to gene therapy may enable entirely new treatment paradigms for individuals with severe genetic disorders. In certain disorders, it is desirable to target gene expression to certain cell types, improve the efficiency of gene delivery, or modulate the expression of large or complex genes. Encoded uses genomics and computational technologies to identify and optimize DNA sequences in the human genome, known as regulatory elements, to control gene expression. These regulatory elements are packaged into gene therapy viral vectors to precisely recapitulate natural patterns of gene expression, addressing key limitations with existing approaches. This gene regulation platform creates opportunities to advance gene therapies for previously untreatable disorders.

“By moving gene therapy beyond its current limitations, Encoded is poised to become a leader in the field,” said Bryan Roberts, Ph.D., partner at Venrock. “We are pleased to join this distinguished group of investors in supporting Encoded’s efforts to bring new gene therapies to diseases that can’t be reached with existing therapeutics.”

Therapeutic Focus

Encoded is focused on four core areas of research: neurocircuitry disorders, liver and metabolic disease, neurodegeneration and cardiovascular disease. Initial programs use clinically-validated adeno-associated viral vectors (AAV), and offer the potential to address disorders outside the reach of current gene therapy technology or significantly improve the benefits of gene therapy in established targets.

Lead Program in Dravet Syndrome

Encoded’s lead program is in Dravet syndrome, a severe genetic disorder that occurs in approximately 1 in 16,000 births worldwide. The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays and an increased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. Current treatments reduce seizures but do not address the underlying cause of the disorder-SCN1A haploinsufficiency. More information about Dravet Syndrome can be found at www.dravetfoundation.org.

A gene therapy approach has the potential to impact the full spectrum of the disorder by targeting the underlying mechanism, yet Dravet Syndrome has been recognized by the field as an exceptionally challenging target. This is because the SCN1A gene exceeds the packaging capacity of AAV and targeting of a specific neuronal cell type, GABAergic inhibitory interneurons, is required. Encoded’s technology platform has enabled the design of an AAV therapeutic that targets the relevant cell type and upregulates endogenous SCN1A expression. Encoded has demonstrated an ability to achieve both cell-selective targeting and upregulation, and is therefore uniquely positioned to advance gene therapies in diseases where a specific cell type is implicated and/or a target gene exceeds the packaging capacity of AAV.

Encoded presented preclinical data from the Dravet Syndrome program at the 22^nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, D.C. in May. These data demonstrated that a single dose of Encoded’s gene therapy is capable of up-regulating SCN1A expression in GABAergic inhibitory interneurons. Furthermore, Dravet mice treated with Encoded’s gene therapy exhibited a significant improvement in sensitivity to hyperthermic seizures. When observed for sudden unexpected death in epilepsy (SUDEP) over a 10 month period, gene therapy-treated Dravet mice were indistinguishable from wild-type mice, whereas control-treated Dravet mice experienced ~50% mortality in that time.

An abstract for this presentation is available at the website of the American Society of Gene and Cell Therapy, annualmeeting.asgct.org.

Board of Directors

Encoded’s board members are world-class innovators, investors, and business leaders with significant experience in gene therapy and genomics, including:

• Sean P. Nolan, Chairman, Former CEO at AveXis Inc.
• Kartik Ramamoorthi, Ph.D., Chief Executive Officer and co-founder
• Stephanie Tagliatela, Chief Scientific Officer and co-founder
• Bryan Roberts, Ph.D., partner at Venrock
• Robert Nelsen, managing director at ARCH Venture Partners
• Karan Takhar, managing director at Matrix Capital Management
• John R. Stuelpnagel, MBA, D.V.M., co-founder of Illumina, Inc., chairman of 10x Genomics and Inscripta, Inc.

“I am thrilled to lead Encoded’s board and accelerate the company’s efforts to deliver breakthrough therapeutics,” said Encoded chairman and former president and CEO of AveXis Inc. Sean P. Nolan. “Encoded represents an incredibly exciting addition to the gene therapy field and I look forward to working with this talented team to further develop and commercialize the pipeline of potentially transformative therapeutics for patients and families suffering from devasting diseases.”

About Encoded
Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. For more information, please visit www.Encoded.com.

Media Contacts
Sarah Sutton
Glover Park Group
ssutton@gpg.com
202-337-0808

Morgan Warners
Glover Park Group
mwarners@gpg.com
202-337-0808

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SOURCE Encoded Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif., Sept. 17, 2019 /PRNewswire/ — Encoded Therapeutics, Inc. (Encoded), a precision gene therapy company, today announced a significant expansion of its leadership team, strengthening its capabilities in corporate development and gene therapy manufacturing.

Andrew Stober, who previously led gene therapy manufacturing for Novartis, will serve as Encoded’s chief manufacturing officer. He brings more than two decades of experience in biologics manufacturing and will lead Encoded’s efforts to develop a strategy for supporting the global needs of its gene therapy pipeline. David McNinch, who was previously a consultant to Encoded, has been appointed chief business officer. He will leverage his deep experience in commercialization and partnering strategy for orphan and rare diseases to lead corporate development.

“We are delighted to add Andrew and David to our leadership team, and look forward to drawing on their collective experience across the biotech industry as we advance our lead program in Dravet syndrome and deepen our gene therapy pipeline,” said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D. “These appointments will accelerate our efforts to become not just an innovative leader in gene therapy research, but an integrated therapeutics company that can independently develop, manufacture, and commercialize gene therapies for patients with severe genetic disorders not addressable with existing approaches.”

“There has never been a more exciting time in gene therapy than now,” said Stober. “I am thrilled at the opportunity to leverage my experience in gene therapy manufacturing to support Encoded’s innovative platform and make manufacturing a core capability of the company.”

“I am pleased to join Encoded as it takes on new frontiers in gene therapy research and therapeutics development,” said McNinch. “I look forward to continuing to support the company’s efforts to scale research, move our lead product into the clinic, and implement go-to-market strategies for the portfolio.”

New Leadership Team Appointments

• Andrew Stober most recently led gene therapy manufacturing at Novartis, where he significantly expanded capacity, sought cost-effective solutions, and made gene therapy manufacturing a core company competency. In a series of leadership roles at AveXis, he supported the evolution of manufacturing capabilities for the gene therapy portfolio as it moved from clinical development through product launch. Prior to AveXis, Stober served in multiple senior manufacturing roles at Biogen, culminating as senior director of manufacturing. He also held positions at Amgen and Merck & Co. He earned his B.S. in chemical engineering at Lehigh University.
• David McNinch has 25 years of experience in biotech and has successfully scaled up multiple companies to evolve their capabilities. He previously served as chief commercial officer of Prothena, Corp., where he led the development of commercial strategy for its immunotherapy pipeline. Prior to Prothena, he was senior vice president of commercial operations at InterMune, Inc., where he built the commercial organization for successful launch of the first treatment for IPF and the company’s eventual acquisition by Roche. Previously, David held various leadership roles of increasing responsibility at Ipsen, Genentech, Novartis and AstraZeneca. He currently serves as vice chair for the Pulmonary Fibrosis Foundation. He earned his B.S. in marketing from the Darla Moore School of Business at the University of South Carolina.

About Encoded
Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. For more information, please visit www.Encoded.com.

Media Contacts
Sarah Sutton
Glover Park Group
ssutton@gpg.com
202-337-0808

Danielle Cantey
Glover Park Group
dcantey@gpg.com
202-337-0808

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SOURCE Encoded Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif., Dec. 18, 2019 /PRNewswire/ — Encoded Therapeutics, Inc. (Encoded), a precision gene therapy company, today announced the continued expansion of its senior leadership team. Nancy Boman, M.D., Ph.D., has joined Encoded as chief regulatory officer, and Monique Bobadilla as chief people and culture officer.

“These two senior leaders add to our diverse and experienced team, further enhancing our ability to create and deliver innovative gene therapies to patients,” said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D. “Monique has spent most of her career in the biotech sector, and has an eye for attracting talent and building a strong corporate culture. Nancy played a major role in collaborating with the FDA to get the first pediatric neuromuscular gene therapy, Zolgensma^®, approved when the regulatory path for gene therapy was new for both the FDA and the industry. We welcome them both to Encoded and look forward to drawing on their expertise.”

“Encoded has a tremendous opportunity to bring potentially life-changing treatments to patients with Dravet Syndrome and other serious genetic diseases,” said Dr. Boman. “I am thrilled to bring my experience in gene therapy development to Encoded as we advance ETX101 to the clinic.”

“I am delighted to join Encoded at this significant moment of growth for the company,” said Bobadilla. “I look forward to collaborating with the entire team to scale the organization on the foundation of our values-based culture, in support of our efforts to develop precision therapies for patients in need.”

New Leadership Team Appointments:

• Nancy Boman, M.D., Ph.D., has 25 years of experience in regulatory affairs and clinical development, and has led the filing of more than 15 drug marketing applications. She joins Encoded from AveXis, Inc., where she was chief regulatory officer and led the successful Biologics License Application (BLA) for Zolgensma. She previously served as senior vice president, regulatory affairs and pharmacovigilance at Alder BioPharmaceuticals. Before that, she held positions in regulatory affairs and clinical development at Acucela, Inc., Genentech, Inc., and Amgen, Inc. She earned her B.Sc. and M.D., as well as her Ph.D. in biochemistry from the University of British Columbia.
• Monique Bobadilla brings significant experience leading human resources functions within the biotech industry. She previously spent more than a decade at Genentech, Inc., where she held a series of human resources roles with increasing responsibility, culminating in her most recent position as site lead for the global product development organization. Before that, she held human resource related roles at Biogen, Inc., Digirad Imaging Systems, Inc., and Edwards Lifescience Cardiovascular Resources, Inc. She earned a B.A. in political science from the University of Massachusetts.

About Encoded

Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positive Dravet Syndrome. For more information, please visit www.Encoded.com.

Media Contacts

Sarah Sutton
Glover Park Group
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202-337-0808

Morgan Warners
Glover Park Group
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SOURCE Encoded Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif., Feb. 11, 2020 /PRNewswire/ — Encoded Therapeutics, Inc. (Encoded), a precision gene therapy company, today announced the appointment of Salvador Rico, M.D., Ph.D., as chief medical officer and the promotion of Martin Moorhead, Ph.D., to chief technology officer. Dr. Rico joins Encoded from Audentes Therapeutics, where he led clinical development of the company’s pipeline of gene therapies for neuromuscular disorders. In his three years at Encoded, Dr. Moorhead has guided the development of the company’s technology platform for creating innovative AAV-based gene therapies. He previously led the development of clonoSEQ^®, the FDA-approved next-generation sequencing assay for detecting minimal residual disease in lymphoid malignancies, at Adaptive Biotechnologies.

“Sal is an accomplished physician-scientist with deep experience advancing novel therapeutics through clinical development, and Martin is a strong leader who brings a genomics mindset to all aspects of gene therapy development,” said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D. “With these appointments, we now have some of the most qualified gene therapy experts in the industry with a proven track record of delivering for patients in need. Their collective experience includes bringing multiple AAV-based gene therapies through clinical development, FDA filings, and approval. I am more confident than ever that our novel gene therapies can make a major impact on patients suffering from debilitating diseases, starting with Dravet Syndrome.”

At Encoded, Dr. Rico will lead medical strategy and clinical development of ETX101, which is being developed for patients with SCN1A+ Dravet Syndrome. Dr. Moorhead will lead the technical team that enables Encoded’s innovative research platform.

“I am delighted to join an organization that is so committed to transforming patients’ lives with the development of next-generation gene therapies,” said Dr. Rico. “I look forward to working closely with both the team at Encoded, and with the Dravet Syndrome community, to advance ETX101 through clinical development and ultimately, deliver it to patients in need.”

“In building a technology platform that combines the power of genomics and computation with AAV-based gene therapy, Encoded is forging the path for the next generation of precision genetic medicines,” said Dr. Moorhead. “I am very proud of what we have accomplished to date and am thrilled at the opportunity to help advance multiple programs for diseases where no treatment options currently exist.”

New Leadership Team Appointments

• Salvador Rico, M.D. Ph.D.: Dr. Rico brings over 15 years of clinical research experience in the biopharmaceutical industry. Prior to joining Encoded, he served as senior vice president of clinical development at Audentes Therapeutics, an innovator in neuromuscular gene therapies that was recently acquired by Astellas, Inc. While there, Dr. Rico led the clinical development of AT132, an AAV-based gene therapy for X-Linked Myotubular Myopathy, participated in the design, conduct, and analysis of Phase I-IV clinical trials in multiple therapeutic areas, and successfully contributed to the FDA approval of multiple drugs, biological products, and medical devices including the INTERCEPT Blood System®, Intermezzo®, Hibor®, Champix®, Prolia,® Kerydin®, and others. Prior to joining Audentes, he led the clinical development teams at Cerus Corporation and Transcept Pharmaceuticals and was an investigator at the Centre for Drug Research, Hospital de la Santa Creu i Sant Pau in Barcelona, Spain. Dr. Rico earned his Doctor of Medicine and Surgery degree from the National Autonomous University of Mexico and holds an M.S. and a Ph.D. (Summa Cum Laude) in pharmacology from the Universitat Autonoma de Barcelona. He did his postdoctoral training in clinical pharmacology and transfusion medicine at the National Autonomous University of Mexico, and in pharmaceutical medicine at Universitat Autonoma de Barcelona.
• Martin Moorhead, Ph.D.: Dr. Moorhead is a seasoned biotechnology executive with expertise that spans biology, assay development, machine learning, algorithm and software development. Prior to joining Encoded, he served as senior vice president of research and development at Adaptive Biotechnologies, and before that, as vice president of computational biology and informatics at Sequenta, Inc. Earlier in his career, he held positions at Affymetrix, ParAllele Bioscience, Viaken, and Synomics. He earned his Ph.D. in elementary particle physics from the University of Oxford, holds an M.Sc. in computing from the Imperial College of London, and earned his B.Sc. in physics from the University of Manchester. He also completed a postdoctoral research fellowship at the Lawrence Berkeley National Library, where he developed machine learning algorithms for data analysis.

About Encoded

Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positive Dravet Syndrome. For more information, please visit www.Encoded.com.

Media Contacts

Sarah Sutton
Glover Park Group
ssutton@gpg.com
202-337-0808

Danielle Cantey
Glover Park Group
dcantey@gpg.com
202-337-0808

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SOURCE Encoded Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif., July 22, 2020 /PRNewswire/ — Encoded Therapeutics, Inc. (Encoded), a precision gene therapy company, today announced it has raised $135 million in an oversubscribed Series D financing. The company also announced that its lead asset, ETX101, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

GV (formerly Google Ventures) led the Series D financing with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 2¹, and additional unnamed investors.

“I am incredibly proud of the progress our organization has made over the past year. We have built an outstanding team, further advanced ETX101 towards the clinic, applied our technology to a series of pipeline programs, and expanded our capabilities to become a fully integrated organization. We are grateful to our investors for supporting our vision to transform patients’ lives with cell type-selective genetic medicines,” said Encoded co-founder and chief executive officer, Kartik Ramamoorthi, Ph.D.

“Since 2019, our company has attracted some of gene therapy’s most experienced executive leaders to prepare ETX101 for the clinic and beyond. This well-rounded team has made significant advances in manufacturing, clinical development, and regulatory affairs for ETX101. By coupling these with an innovative discovery engine, Encoded is advancing its preclinical pipeline of gene therapies to patients suffering from devastating pediatric CNS diseases,” said Encoded’s board chairman, Sean Nolan.

Encoded’s discovery engine combines biological and computational approaches to identify and screen human DNA sequences known as regulatory elements at a high throughput scale. The resulting multi-dimensional, large scale datasets are leveraged to design optimal gene therapy expression cassettes which more precisely control transgene expression. By recapitulating natural patterns of gene expression, the resulting gene therapy vectors can provide maximal therapeutic benefit, minimize off-target expression, and address genetic disorders that have previously been considered untreatable.

David Schenkein, M.D., general partner and co-leader of GV’s life sciences team, will join the Encoded Board of Directors as an observer. “Encoded’s cutting edge platform has the potential to impact a broad range of diseases across different organ systems. Importantly, Encoded has translated this innovation into a one-time, first-in-class precision gene therapy candidate for the treatment of SCN1A+ Dravet Syndrome, a devastating pediatric disease with significant unmet medical need. I am enthusiastic to work with this top-tier team in their mission to treat these underserved patients and advance a portfolio of innovative medicines,” said Dr. Schenkein.

The company also announced that ETX101 was granted Orphan Disease and Rare Pediatric Disease Designation by FDA. Both programs offer incentives for the development of therapeutics for underserved populations.

Proceeds from the Series D will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. Additionally, the funds will support progression of the company’s pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

About Encoded
Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Seeded by Venrock, ARCH Venture Partners, Illumina Ventures, and Matrix Capital Mangament, our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome. For more information, please visit www.Encoded.com.

About Dravet Syndrome
Dravet syndrome is a rare, severe genetic disorder that occurs in approximately 1 in 16,000 births worldwide. The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays, and an increased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. Current treatments reduce seizures but do not address the underlying cause of the disorder-SCN1A haploinsufficiency. More information about Dravet Syndrome can be found at www.dravetfoundation.org.

Media Contacts

Sarah Sutton
Glover Park Group
ssutton@gpg.com
202-337-0808

Danielle Cantey
Glover Park Group
dcantey@gpg.com
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SOURCE Encoded Therapeutics, Inc.