SOUTH SAN FRANCISCO, Calif.--(

加利福尼亚州南旧金山--(

BUSINESS WIRE

商业热线

)--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..

)--Encoded Therapeutics Inc.是一家临床阶段的生物技术公司，开发用于严重中枢神经系统（CNS）疾病的遗传药物，今天宣布其领先的临床项目ETX101继续取得进展，并强调了公司取得的成就，这些成就推进了其研究渠道，并进一步开发了基础设施，以充分整合公司的基因治疗能力。。

“

“

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,”

2024年，我们启动了正在进行的北极星计划，评估SCN1A+Dravet综合征中的ETX101。随着多剂量水平的给药，我们预计将在今年下半年分享初步疗效数据。”

said

赛义德

Kartik Ramamoorthi, Ph.D., co-founder and Chief Executive Officer.

Kartik Ramamoorthi博士，联合创始人兼首席执行官。

“

“

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.

这一快速进展，加上我们的Angelman综合征计划ETX201的进步以及我们更广泛的中枢神经系统管道，突显了我们组织发现和开发创新中枢神经系统疗法的潜力。鉴于我们投资组合的巨大潜力，Encoded将资源集中在ETX101和我们建立的项目上，在这些项目中，我们有最大的机会创造近期价值。

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.”.

与此同时，我们做出了一个艰难的决定，即缩小技术规模和早期研发功能。我非常感谢团队的贡献，并相信2025年将是ENCODE变革的一年。”。

ETX101 for Dravet Syndrome, POLARIS Program

ETX101用于Dravet综合征，POLARIS计划

ETX101 is an AAV9-based gene regulation therapy designed to upregulate expression of the

ETX101是一种基于AAV9的基因调控疗法，旨在上调

SCN1A

SCN1A

gene in inhibitory neurons and is the first potential one-time therapy to address the underlying genetic cause of Dravet syndrome.

抑制性神经元中的基因，是解决Dravet综合征潜在遗传原因的第一种潜在的一次性疗法。

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.

POLARIS是美国、英国和澳大利亚的一项全球临床计划，正在招募患有SCN1A+Dravet综合征的婴儿和儿童（6个月至7岁）；八名患者接受了多剂量治疗。

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

截至2月12日，ETX101的耐受性良好；在任何剂量水平上均未报告与治疗相关的严重不良事件。

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

计划在下半年完成额外剂量水平并报告初步安全性和有效性数据。

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

ENCODE与FDA和MHRA就假控制，延迟治疗，潜在验证性试验的设计达成一致。

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

ETX101具有来自FDA的快速通道，罕见儿科和孤儿药物名称以及来自EMA的孤儿药物名称。

ETX201 for Angelman Syndrome

ETX201治疗Angelman综合征

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

ETX201是一种基于AAV9的矢量化microRNA（miRNA），旨在降低UBE3A反义转录本（UBE3A-ATS）和非沉默父本的表达

UBE3A

UBE3A

expression.

表达式。

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

。数据表明，ETX201耐受性良好，能够广泛传播父系

UBE3A

UBE3A

upregulation across critical, disease-relevant brain regions.

跨关键的，与疾病相关的大脑区域上调。

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

根据FDA的指导，已启动IND授权研究，以支持2026年潜在的ETX201 IND申请。

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

ETX201有可能成为解决Angelman综合征根本原因的一次性治疗方法。

Research Pipeline

研究管道

Chronic pain candidate, an AAV9-based vectorized miRNA designed to knock down expression of

慢性疼痛候选物，一种基于AAV9的矢量化miRNA，旨在敲低

SCN9A

SCN9A

(Na

（不适用

V

五

1.7), 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.

1.7），在已建立的啮齿动物疼痛模型中证明了对多种疼痛表型的强大而持久的纠正。NHP的研究正在进行中，有可能在下半年提名一名发展候选人。

Alzheimer’s disease candidate, an AAV9-based vectorized miRNA, demonstrated robust and durable knockdown (up to 32%) of

阿尔茨海默病候选者是一种基于AAV9的矢量化miRNA，表现出强大而持久的击倒（高达32%）

MAPT

地图

(tau) across disease relevant brain regions in an NHP study.

（tau）在NHP研究中跨越疾病相关的大脑区域。

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

疼痛和阿尔茨海默病项目的数据将在2025年年中的科学会议上公布。

Strategic Reorganization

战略重组

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

Encoded实现了劳动力减少29%，将现金跑道延长至第三季度。

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

受影响的职能主要包括技术和早期研发。

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.

增强型跑道支持我们实现关键里程碑的目标，包括ETX101的初步临床安全性和有效性，ETX201的IND/CTA，以及我们共同中枢神经系统项目的开发候选人提名。

Additional Corporate Updates

其他公司更新

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.

2024年，ENCODE启动了GMP设施的建设，以支持ETX101的下一阶段开发和更广泛的投资组合；该设施将于2015年第1季度全面投入运营。展望未来，ETX101，ETX201和管道计划的所有原料药材料将在内部生产。

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.

礼来公司的全资子公司Encoded and Prevail Therapeutics于2024年5月签署了一项合作协议，让礼来基因治疗公司使用Encoded的新型调控元件。这些调控元件旨在增强或减少特定组织和器官中的转基因表达。

Encoded received an upfront payment and is eligible to receive preclinical, development, regulatory and commercial milestone payments..

Encoded收到了预付款，有资格获得临床前、开发、监管和商业里程碑付款。。

About Encoded Therapeutics

关于编码疗法

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.

Encoded Therapeutics是一家临床阶段的遗传药物公司，正在开发针对严重中枢神经系统疾病的潜在一次性疾病缓解疗法。我们专有的矢量工程技术将新型监管元件和有效载荷与AAV矢量相结合，为棘手的中枢神经系统疾病解锁了创新解决方案。

Our lead clinical-stage program, ETX101 for Dravet syndrome, targets the underlying cause of the disorder through selective upregulation of .

我们的主要临床阶段计划ETX101针对Dravet综合征，通过选择性上调靶向疾病的根本原因。

SCN1A

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

为了潜在的长期利益。Encoded的第二个程序ETX201是一种开发阶段的基于miRNA的基因疗法，旨在恢复

UBE3A

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.

患有Angelman综合征的个体。与此同时，我们正在推进针对常见中枢神经系统疾病（包括慢性疼痛和阿尔茨海默病）的潜在最佳课程。利用我们的专有技术平台和专业知识，我们可以有效地推进从发现到临床开发的项目。

Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit .

Encoded致力于开创中枢神经系统疾病的突破性治疗。有关更多信息，请访问。

www.encoded.com

www.encoded.com

.

.