Category: Press Releases

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
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., 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
202-337-0808

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SOURCE Encoded Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today announced the approval of its Clinical Trial Authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for the EXPEDITION Study of ETX101 as a potential treatment for children with SCN1A+ Dravet syndrome. Dravet syndrome is the most common developmental and epileptic encephalopathy and is characterized by a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation.

The UK CTA for EXPEDITION is part of a global regulatory strategy established by Encoded for clinical development of ETX101, which also includes regulatory clearance for an investigational new drug (IND) application in the United States and approval under the Clinical Trial Approval (CTA) scheme in Australia.

“This CTA marks the third regulatory approval for ETX101 as we advance our global clinical development program, POLARIS, to bring this potential one-time AAV gene regulation therapy to people affected by SCN1A+ Dravet syndrome around the world,” said Sal Rico, M.D., Ph.D., Chief Medical Officer of Encoded. “We believe ETX101 has the potential to deliver transformative benefit in multiple disease domains and have designed our clinical program to efficiently assess its impact on affected infants and young children. We are committed to working closely with our highly experienced investigators to expeditiously enroll affected children in an effort to advance therapeutic options for people living with Dravet syndrome.”

Professor Andreas Brunklaus, M.D., Ph.D., EXPEDITION Principal Investigator, and Consultant Paediatric Neurologist at the Royal Hospital for Children, Glasgow, UK, said, “In launching the EXPEDITION trial, we aim to confront the urgent need to develop disease modification strategies for children battling Dravet syndrome in the UK. The relentless and devastating nature of this condition underscores the critical importance of developing therapies that not only alleviate symptoms but also target the underlying mechanisms. EXPEDITION represents a significant stride forward in our collective pursuit of new treatments that can truly make a difference in the lives of people affected by Dravet syndrome.”

About EXPEDITION

EXPEDITION is part of Encoded’s global clinical development program, POLARIS, and is a Phase 1/2 dose escalation study of ETX101 in children aged 6 months to <4 years with SCN1A+ Dravet syndrome in the UK. 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 evaluate preliminary efficacy, and to contribute to therapeutic dose selection. EXPEDITION is expected to commence in the UK in mid-2024 and will run concurrently with clinical trials in the US (ENDEAVOR) and Australia (WAYFINDER).

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, including the ENDEAVOR (US), WAYFINDER (Australia) and EXPEDITION (UK) studies, to assess the safety and preliminary efficacy of ETX101 in infants and young children with SCN1A+ Dravet syndrome.

About ETX101

ETX101 is 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.

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/20240226523636/en/

Jennifer Gorzelany

communications@encoded.com

650-515-9695

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today unveiled multiple pipeline programs ahead of the 27^th ASGCT Annual meeting on May 7 – 11, 2024 in Baltimore, MD. Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.

“Our ASGCT presentations showcase the depth and versatility of our vector engineering platform to develop highly specific precision therapies for CNS disorders with high unmet need,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded. “Combining our novel regulatory elements and transgenes provides control of expression in target cells, potentially enabling treatment of devastating, intractable diseases. Building on our strong foundation in neuroscience, we are leveraging the experience and infrastructure established for our Dravet syndrome program, ETX101, to rapidly advance our promising pipeline of potentially best-in-class gene therapies.”

Platform Innovations

Encoded is engineering precision and selectivity into gene therapy constructs by combining highly selective and potent regulatory elements (REs) with novel transgenes to customize expression and functionality while minimizing off-target effects. These modular elements are compatible with multiple capsids and gene delivery systems.

Novel REs developed by Encoded include those for GABA selectivity to target expression to GABAergic (inhibitory) interneurons, utilized by the clinical-stage program ETX101 for Dravet syndrome and the preclinical program for LGS. For STXBP1-RD, regulatory element engineering has been harnessed to drive potent, neuron-specific expression in the brain, while de-targeting expression in dorsal root ganglia (DRG).

Engineered transgenes include transcription factors (eTFs) that upregulate the expression of endogenous genes as well miRNA sequences derived from Encoded’s miRNA discovery platform. A miRNA approach is utilized by the Angelman syndrome, Alzheimer’s disease and neuropathic pain programs, each of which demonstrate robust gene silencing or knockdown of their respective targets.

“The data we’re sharing at ASGCT highlights our platform’s ability to achieve precise, targeted expression of transgenes with unique functionality, addressing technical limitations that exist today. This enables us to potentially unlock first-in-class gene therapies in monogenic disorders, like Dravet and Angelman syndromes, while also advancing potentially best-in-class gene therapies into more common diseases such as LGS, Alzheimer’s disease and neuropathic pain,” said Encoded CEO Kartik Ramamoorthi, Ph.D. “This is a significant maturation of our pipeline, and future success in these indications has the potential to transform the lives of many people living with severe CNS diseases.”

ASGCT Annual Meeting Presentations

Oral Presentation Details

Title: GABA Selective AAV-mediated Gene Therapy Provides Durable Seizure Protection in Multiple Refractory Epilepsy Models (Abstract #19)

Session: AAV Vectors – Preclinical and Proof-of-Concept: Therapy Focus

Date & Time: Tuesday, May 7, 1:45-2:00 p.m. ET

Location: Ballroom 2

Poster Presentation Details

Title: Advancing Gene Therapy for STXBP1-related Disorders Through Targeted Vector Engineering: Efficacy and Safety Results in Mice and Non-Human Primates (Abstract #508)

Date & Time: Wednesday, May 8, 5:30-7:00 p.m. ET

Location: Exhibit Hall

Title: A Vectorized miRNA-based Approach to Unsilence UBE3A in Angelman Syndrome (Abstract #1125)

Date & Time: Thursday, May 9, 5:30-7:00 p.m. ET

Location: Exhibit Hall

Title: Identification of Potent and Selective AAV-miRNA Candidates to Knockdown Non-Monogenic Neurological Targets SCN9A (Pain) and MAPT (Tauopathies) (Abstract #1601)

Date & Time: Friday, May 10, 5:30-7:00 p.m. ET

Location: Exhibit Hall

Scientific Symposium Details

Title: Searching for Goldilocks – Scaling for AAV Clinical Dose Prediction

Presentation: Dose Scaling from Preclinical Models to First-In-Human (FIH) for Local CNS AAV Gene Therapies

Date & Time: Thursday, May 9, 8:00-8:25 a.m. ET

Location: Ballroom 4

Presenter: Stephanie Tagliatela

Full abstracts are available on the ASGCT meeting website.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially disease-modifying therapies to improve the lives of people with severe CNS disorders. Our proprietary vector engineering approach combines novel regulatory elements and payloads with AAV vectors to unlock innovative solutions for debilitating, intractable CNS conditions. At the forefront is our flagship clinical program, ETX101 for Dravet syndrome, which targets the underlying cause of the disorder to enable highly selective upregulation of SCN1A for potentially long-lasting benefit. In parallel, we are advancing a pipeline of potentially best-in-class programs to address significant unmet needs across both monogenic and prevalent CNS conditions. Harnessing our proprietary technology platform and expertise, we are able to efficiently advance programs from discovery through clinical development. Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit www.encoded.com.

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Jennifer Gorzelany

communications@encoded.com

650-515-9695

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today highlights oral and poster presentations on the Company’s preclinical gene therapy programs and vector engineering platform at the 27^th ASGCT Annual Meeting being held May 7 – 11, 2024 in Baltimore, MD.

“We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts. These data underscore our platform’s potential to deliver precision therapies for a range of CNS diseases,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded. “Alongside clinical advancement of our lead program, ETX101 for SCN1A+ Dravet syndrome, we are poised to nominate multiple programs to development in 2025, marking yet another significant milestone.”

Oral Presentation

Title: GABA Selective AAV-mediated Gene Therapy Provides Durable Seizure Protection in Multiple Refractory Epilepsy Models (Abstract #19)

Session: AAV Vectors – Preclinical and Proof-of-Concept: Therapy Focus

Date & Time: Tuesday, May 7, 1:45-2:00 p.m. ET

Location: Ballroom 2

Epilepsy affects approximately 1% of the population, with nearly one third of cases failing to respond to first- and second-line treatments. Encoded’s vector engineering platform has enabled the development of GABA-selective AAV-mediated gene therapies designed to potentiate GABAergic neurotransmission and modulate the circuit dysfunction underlying refractory epilepsies. Our lead candidate reduced seizure incidence and severity in two biologically distinct seizure assays with no adverse impact on body weight, motor function or locomotion. Furthermore, the top candidate is well-tolerated in non-human primates (NHP) even at high doses. These data support further development of our candidate gene therapy for the treatment for refractory epilepsies.

Poster Presentations

Title: Advancing Gene Therapy for STXBP1-related Disorders Through Targeted Vector Engineering: Efficacy and Safety Results in Mice and Non-Human Primates (Abstract #508)

Date & Time: Wednesday, May 8, 5:30-7:00 p.m. ET

Location: Exhibit Hall

STXBP1-related disorders (STXBP1-RD) result from loss-of-function variants in STXBP1, causing severe developmental delay and treatment-resistant epilepsy. Encoded has engineered AAV9 vectors that drive potent expression within CNS tissue while reducing off-target expression in dorsal root ganglia (DRG) and non-neuronal tissues. These engineered AAV9-STXBP1 vectors achieved robust, dose-dependent improvements in seizure, cognitive, and motor phenotypes in Stxbp1^+/- mice, with no observed dose-limiting toxicity. In NHPs, the engineered vectors showed strong CNS expression and were well-tolerated. Furthermore, the inclusion of a DRG de-targeting element reduced expression within the DRG and spinal cord by >10-fold, ameliorating microscopic DRG-related findings. These findings establish proof-of-concept for an AAV9-based gene therapy for STXBP1-RD and support the use of modular regulatory element engineering to drive desired expression profiles within the CNS.

Title: A Vectorized miRNA-based Approach to Unsilence UBE3A in Angelman Syndrome (Abstract #1125)

Date & Time: Thursday, May 9, 5:30-7:00 p.m. ET

Location: Exhibit Hall

Angelman syndrome (AS) is a severe neurodevelopmental disorder characterized by intellectual disability, ataxia, and seizures. AS is caused by loss-of-function of the maternally-inherited UBE3A gene. Unsilencing of the paternal copy of UBE3A has the potential address the underlying cause of the disease while mitigating the risk of UBE3A overexpression-related toxicity. We demonstrated that an AAV-based miRNA vector was capable of upregulating paternal UBE3A and correcting multiple phenotypes in the AS mouse model. Encoded’s lead miRNA candidate was well-tolerated in NHPs and demonstrated potent unsilencing of paternal UBE3A in critical brain regions. Collectively, these data support further development an AAV-based miRNA treatment approach for Angelman Syndrome.

Title: Identification of Potent and Selective AAV-miRNA Candidates to Knockdown Non-Monogenic Neurological Targets SCN9A (Pain) and MAPT (Tauopathies) (Abstract #1601)

Date & Time: Friday, May 10, 5:30-7:00 p.m. ET

Location: Exhibit Hall

Encoded’s innovative AAV-mediated miRNA platform offers a promising strategy for long-term knockdown of target genes in the central and peripheral nervous systems. We identified potent and target-specific miRNA candidates to effectively knock down two clinically relevant, non-monogenic neuronal targets: SCN9A and MAPT. SCN9A encodes the Na_V1.7 sodium channel, which is associated with pain sensation, while MAPT encodes the microtubule protein, Tau, which is implicated in Alzheimer’s disease neuropathology. Encoded’s miRNA candidates demonstrated potent, dose-dependent, and selective knockdown of SCN9A in human iPSC sensory neurons and in mouse dorsal root ganglion sensory neurons, establishing proof-of-concept for miRNA-based inhibition of Na_V1.7. Similarly, MAPT candidates significantly reduced Tau protein levels in vitro and in vivo. These findings support advancement of SCN9A and MAPT candidates to NHP studies and showcase the potential of vectorized miRNA knockdown as a promising treatment avenue for various neurological disorders.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially disease-modifying therapies to improve the lives of people with severe CNS disorders. Our proprietary vector engineering approach combines novel regulatory elements and payloads with AAV vectors to unlock innovative solutions for debilitating, intractable CNS conditions. At the forefront is our flagship clinical program, ETX101 for Dravet syndrome, which targets the underlying cause of the disorder to enable highly selective upregulation of SCN1A for potentially long-lasting benefit. In parallel, we are advancing a pipeline of potentially best-in-class programs to address significant unmet needs across both monogenic and prevalent CNS conditions. For more information, please visit www.encoded.com.

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Jennifer Gorzelany

communications@encoded.com

650-515-9695

ORLANDO, Fla. – Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that it will present new preclinical data on ETX201, an AAV9-based vectorized miRNA approach designed to reduce the expression of the UBE3A-ATS and unsilence paternal UBE3A expression for the treatment of Angelman syndrome.

At the Foundation for Angelman Syndrome Therapeutics (FAST) 17^th Annual Global Science Summit in Orlando, FL, Encoded will present data showing that ETX201 was well tolerated in non-human primates (NHPs) with broad expression across multiple brain regions following a single intracerebroventricular administration. ETX201 miRNA transcript was also detectable in the cerebrospinal fluid. Knockdown of the UBE3A antisense transcript (UBE3A-ATS) was observed, in addition to widespread paternal UBE3A upregulation across the cortex and hippocampus — critical, disease-relevant brain regions important for learning, memory, and cognitive functioning.

“Our approach restores expression of the UBE3A gene by unsilencing the paternal allele and represents an important step towards addressing the underlying cause of Angelman syndrome with a potentially durable, one-time therapy,” said Stephanie Tagliatela, co-founder and Chief Scientific Officer of Encoded. “These data validate our vectorized miRNA technology and provide strong rationale for exploring its application for common CNS diseases.”

FAST Global Science Summit Presentation Details:

Title: ETX201: An experimental vectorized miRNA-based approach for the treatment of Angelman syndrome

Presenter: Sirika Pillay, Ph.D., Director of Molecular and Cellular Pharmacology, Encoded Therapeutics

Date & Time: Saturday, November 9, at 9:50-10:10 a.m. ET

About Angelman Syndrome

Angelman syndrome is a severe and lifelong neurodevelopmental disorder that is estimated to affect approximately 1 in 12,000 to 1 in 20,000 people globally. It is caused by loss of function in the maternally inherited copy of the UBE3A gene, which plays a critical role in the development and function of the nervous system. As a result of this deficiency, individuals with Angelman syndrome experience a range of challenges, including cognitive impairment, developmental delays, lack of speech, movement disorders (such as ataxia and tremors), seizures, as well as sleeping and feeding problems. While affected individuals typically have a normal lifespan, they are unable to live independently; they require substantial support throughout their lives, relying on caregivers and family for assistance with daily activities. Despite its significant impact, there are currently no approved therapies specifically designed to treat Angelman syndrome.

About ETX201

ETX201 is a vectorized microRNA (miRNA) designed to unsilence the paternal copy of UBE3A, which is naturally silenced in neurons by the UBE3A antisense transcript (UBE3A-ATS). ETX201 expresses a miRNA that targets the UBE3A-ATS to knock down its expression and is delivered using a clinically-validated adeno-associated virus serotype 9 (AAV9) vector. This potential one-time treatment approach aims to consistently and durably restore UBE3A expression and address the spectrum of symptoms associated with Angelman syndrome by targeting the underlying cause of the disease.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially disease-modifying therapies to improve the lives of people with severe CNS disorders. Our proprietary vector engineering approach combines novel regulatory elements and payloads with AAV vectors to unlock innovative solutions for debilitating, intractable CNS conditions. At the forefront is our flagship clinical program, ETX101 for Dravet syndrome, which targets the underlying cause of the disorder to enable highly selective upregulation of SCN1A for potentially long-lasting benefit. In parallel, we are advancing a pipeline of potentially best-in-class programs to address significant unmet needs across both rare and common CNS conditions. Harnessing our proprietary technology platform and expertise, we can efficiently advance programs from discovery through clinical development. Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit www.encoded.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241107185885/en/

Jennifer Gorzelany

communications@encoded.com

650-515-9695

SOUTH SAN FRANCISCO, Calif. – Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced continued progress in its lead clinical program, ETX101, and highlighted corporate achievements that advance its research pipeline and further develop infrastructure to fully integrate the Company’s gene therapy capabilities.

“In 2024, we launched our ongoing POLARIS program evaluating ETX101 in SCN1A+ Dravet syndrome. With multiple dose levels administered, we expect to share preliminary efficacy data in the second half of the year,” said Kartik Ramamoorthi, Ph.D., co-founder and Chief Executive Officer. “This rapid progress, coupled with advancement of our Angelman syndrome program, ETX201, and our broader CNS pipeline, underscores the potential for our organization to discover and develop innovative CNS therapies. Given the significant potential of our portfolio, Encoded is focusing resources on ETX101 and our established programs where we have the greatest opportunity to create near-term value. In parallel, we have made the difficult decision to reduce the size of our technology and early-stage research and development functions. I am exceptionally grateful for the team’s contributions and confident that 2025 will be a transformative year for Encoded.”

ETX101 for Dravet Syndrome, POLARIS Program

• ETX101 is an AAV9-based gene regulation therapy designed to upregulate expression of the SCN1A gene in inhibitory neurons and is the first potential one-time therapy to address the underlying genetic cause of Dravet syndrome.

• POLARIS, a global clinical program in the US, UK and Australia enrolling infants and children (6 months – 7 years of age) with SCN1A+ Dravet syndrome is ongoing; eight patients have been treated at multiple dose levels.

• As of February 12, ETX101 has been well-tolerated; no treatment-related serious adverse events have been reported at any dose level.

• Completion of additional dose levels and reporting of preliminary safety and efficacy data are planned for 2H25.

• Encoded has aligned with FDA and MHRA on the design of a sham-controlled, delayed-treatment, potentially confirmatory trial.

• ETX101 has Fast Track, Rare Pediatric, and Orphan Drug Designations from FDA and Orphan Designation from EMA.

ETX201 for Angelman Syndrome

• ETX201 is an AAV9-based vectorized microRNA (miRNA) designed to reduce the expression of the UBE3A antisense transcript (UBE3A-ATS) and unsilence paternal UBE3A expression.

• Encoded presented positive results of an NHP study at the Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in November. Data showed that ETX201 was well-tolerated and capable of widespread paternal UBE3A upregulation across critical, disease-relevant brain regions.

• Following guidance from FDA, IND-enabling studies have been initiated to support a potential ETX201 IND filing in 2026.

• ETX201 has the potential to be a one-time treatment approach to address the underlying cause of Angelman syndrome.

Research Pipeline

• Chronic pain candidate, an AAV9-based vectorized miRNA designed to knock down expression of SCN9A (Na_V1.7), demonstrated robust and durable correction of multiple pain phenotypes in an established rodent pain model. NHP studies are ongoing with the potential to nominate a development candidate in 2H25.

• Alzheimer’s disease candidate, an AAV9-based vectorized miRNA, demonstrated robust and durable knockdown (up to 32%) of MAPT (tau) across disease relevant brain regions in an NHP study.

• Data for both the pain and Alzheimer’s disease programs will be presented at scientific meetings in mid-2025.

Strategic Reorganization

• Encoded implemented a 29% reduction in workforce, extending cash runway through 3Q26.

• Impacted functions primarily include technology and early-stage research and development.

• Enhanced runway supports our goal of achieving key milestones, including preliminary clinical safety and efficacy for ETX101, IND/CTA for ETX201, and nomination of development candidates for our common CNS programs.

Additional Corporate Updates

• In 2024, Encoded initiated the build of a GMP facility to support the next stage of development for ETX101 and the broader portfolio; facility will be fully operational in 1Q25. Moving forward, all drug substance material for ETX101, ETX201 and pipeline programs will be manufactured in-house.

• Encoded and Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, signed a collaboration agreement in May 2024 for Lilly Gene Therapy to use Encoded’s novel regulatory elements. These regulatory elements are designed to enhance or reduce transgene expression in specific tissues and organs. Encoded received an upfront payment and is eligible to receive preclinical, development, regulatory and commercial milestone payments.

About Encoded Therapeutics

Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially one-time, disease-modifying therapies for severe CNS disorders. Our proprietary vector engineering technology combines novel regulatory elements and payloads with AAV vectors, unlocking innovative solutions for intractable CNS conditions. Our lead clinical-stage program, ETX101 for Dravet syndrome, targets the underlying cause of the disorder through selective upregulation of SCN1A for potentially long-lasting benefit. Encoded’s second program, ETX201, is a development-stage vectorized miRNA-based gene therapy designed to restore expression of UBE3A in individuals with Angelman syndrome. In parallel, we are advancing potentially best-in-class programs for common CNS conditions, including chronic pain and Alzheimer’s disease. Harnessing our proprietary technology platform and expertise, we can efficiently advance programs from discovery through clinical development. Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit www.encoded.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250213028144/en/

Jennifer Gorzelany

communications@encoded.com

650-515-9695