Encoded Therapeutics, Inc.

Venrock has added half a billion dollars to its healthcare investing strategy, the firm confirmed to Endpoints News on Wednesday. It comes on top of its $650 million 10th fund — which goes toward both tech and healthcare — that the firm revealed during the 2024 JPM Healthcare Conference in January. A regulatory document outlining a $500 million fund was filed on Wednesday. “We can confirm this $500M fund is separate from the $650M fund announced in January,” a Venrock spokesperson said in an email to Endpoints. “This is a new pool of capital, though not a new strategy; it will invest in certain investment opportunities alongside existing Venrock Healthcare Capital Partner funds.” The filing lists Venrock investors Bong Koh and Nimish Shah, both of whom are focused on crossover and public investing in biotech and pharma, according to the firm’s website. Koh jokingly refers to himself as “team bag carrier” on his LinkedIn profile: “While their bags are heavy and have odd odors, [it] is a real priviledge [sic] that they trust me to be a good steward of their bags.” Venrock Healthcare has been active recently. Alongside Wellington Management, it led the $215 million Series B for obesity biotech Metsera last week. It’s also an investor in Jade Biosciences, a Paragon Therapeutics spinout that disclosed a reverse merger on Oct. 31. Venrock is also an investor in a preclinical growth factor startup called Cavalry Biosciences , ADC startup MBrace Therapeutics, CNS gene therapy maker Encoded Therapeutics and multiple other startups.

ORLANDO, Fla.--( BUSINESS WIRE )--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 .

Four presentations highlight expansion of the company’s CNS pipeline and vector engineering platform Preclinical data presented supports potential to advance programs in Angelman Syndrome and Lennox-Gastaut Syndrome Proof-of-concept data demonstrating knock-down of neuropathic pain (SCN9A) and Alzheimer’s disease (MAPT) targets showcase advancements in the company’s vector engineering platform SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- 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 27th 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 NaV1.7 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 NaV1.7. 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 . View source version on businesswire.com: Contacts Jennifer Gorzelany communications@encoded.com 650-515-9695 Source: Encoded Therapeutics Inc. View this news release online at: