Lead gene therapy candidate significantly reduced seizures and reversed early mortality in preclinical model of Dravet Syndrome
Company aims to maximize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy
Financing syndicate comprised of premier life science investors, including Venrock, ARCH Venture Partners and Matrix Capital Management
SOUTH SAN FRANCISCO, Calif. – June 26, 2019 – 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.”
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 22nd 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 devastating diseases.”
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.
Glover Park Group
Glover Park Group