Using RNA to interfere with a gene’s expression of disease-causing proteins is a validated therapeutic approach, but so far, the products in this drug class only address liver proteins. Reducing levels of certain proteins in the central nervous system could offer a new way to treat neurological disorders.

。降低中枢神经系统中某些蛋白质的水平可能为治疗神经系统疾病提供一种新方法。

The challenge facing drug developers is getting these therapies deep into the brain..

药物开发人员面临的挑战是将这些疗法深入大脑。。

Atalanta Therapeutics has technology that delivers RNA interference therapies into the CNS. The startup has spent the past four years quietly working on its technology and potential drugs. On Tuesday, Atalanta revealed two lead programs for rare neurological disorders that currently have no FDA-approved therapies.

Atalanta Therapeutics拥有将RNA干扰疗法传递到中枢神经系统的技术。这家初创公司在过去四年里一直在默默研究其技术和潜在药物。周二，亚特兰大公布了两项针对罕见神经系统疾病的领先项目，目前尚无FDA批准的疗法。

The Boston-based startup also announced .

这家总部位于波士顿的初创公司也宣布了这一消息。

$97 million in financing

9700万美元融资

to advance those programs to the clinic and support other CNS therapies in its pipeline.

将这些计划推广到诊所，并支持其他中枢神经系统治疗。

An RNA interference (RNAi) therapy uses small pieces of non-coding RNA to block how certain genes are expressed. In 2018, Alnylam Pharmaceuticals’ Onpattro became the

RNA干扰（RNAi）疗法使用小块非编码RNA来阻断某些基因的表达。2018年，Alnylam Pharmaceuticals的Onpattro成为

first FDA-approved RNAi drug

首个FDA批准的RNAi药物

. This therapy is delivered inside a lipid nanoparticle, which preferentially goes to liver cells. Alnylam and others have been pursuing ways to bring RNAi to the CNS.

该疗法在脂质纳米颗粒内递送，脂质纳米颗粒优先进入肝细胞。Alnylam和其他人一直在寻求将RNAi引入中枢神经系统的方法。

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是什么让医疗保健首席信息官夜不能寐：平衡技术投资与消费者期望

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。

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Atalanta, named for the mythical Greek huntress, makes its therapies with oligonucleotides, short pieces of synthetic RNA. Early efforts in oligo and RNAi drugs could not get beyond the outer layer of the brain, Atalanta CEO Alicia Secor said. One of Atalanta’s founders is Craig Mello, a professor in the RNA Therapeutics Institute at the University of Massachusetts Medical School who was awarded the .

亚特兰大以神话中的希腊女猎手命名，用寡核苷酸（一种合成RNA的短片段）进行治疗。亚特兰大首席执行官艾丽西亚·塞科（AliciaSecor）说，早期对寡核苷酸和RNAi药物的研究无法超越大脑的外层。亚特兰大的创始人之一是克雷格·梅洛（CraigMello），他是麻省大学医学院RNA治疗研究所的教授，获得了该奖项。

2006 Nobel Prize in Physiology or Medicine

2006年诺贝尔生理学或医学奖

for his RNAi discoveries. Mello’s research includes ways to deliver oligo therapies into the brain.

因为他的RNAi发现。梅洛的研究包括将寡核苷酸疗法导入大脑的方法。

Atalanta does not use lipid nanoparticles or other delivery technologies, Secor said. Instead, the startup’s drugs employ what she described as a divalent structure in which two small-interfering RNA (siRNA) duplexes are joined by a linker. This structure enable the therapy get into the CNS and it imbues the therapy with properties such as potency and durability..

塞科说，亚特兰大不使用脂质纳米粒或其他递送技术。相反，这家初创公司的药物采用了她所描述的二价结构，其中两个小干扰RNA（siRNA）双链体通过接头连接。这种结构使治疗能够进入中枢神经系统，并使治疗具有效力和耐久性等特性。。

“These molecules are very special and have demonstrated the ability to achieve really broad brain distribution in all regions, [and] importantly, deep brain penetration,” Secor said.

塞科说：“这些分子非常特殊，已经证明能够在所有区域实现真正广泛的大脑分布，重要的是，能够深入大脑。”。

Mello was particularly interested in developing therapies for Huntington’s disease, a rare neurological disorder that originates deep in the brain. UMass research

梅洛对开发亨廷顿舞蹈病的治疗方法特别感兴趣，亨廷顿舞蹈病是一种罕见的神经系统疾病，起源于大脑深处。麻省大学研究院

published in Nature Biotechnology

发表于Nature Biotechnology

in 2019 described mouse and monkey studies that showed a single injection of divalent siRNA led to silencing of the gene that causes Huntington’s. This silencing lasted at least six months.

2019年描述的小鼠和猴子研究表明，单次注射二价siRNA导致导致亨廷顿舞蹈症的基因沉默。这种沉默至少持续了六个月。

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人工智能（AI）的进步正在从各个方面重塑患者护理，从促进更快的出院到策划治疗计划并建议改变生活方式。

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Soon after the research was published, F-Prime Capital licensed the UMass technology and formed Atalanta with Secor as its first employee. When the startup emerged from stealth in 2021 with $110 million in

这项研究发表后不久，F-Prime Capital授予了麻省大学的技术许可，并与Secor一起组建了亚特兰大大学，成为其第一名员工。当这家初创公司在2021年以1.1亿美元的收入悄然兴起时

Series A financing

A系列融资

, it also revealed R&D alliances with Biogen and Genentech. Secor acknowledged that it’s unusual for a startup to land two big pharma partnerships at such an early stage, but she said the deals speak to the industry interest in bringing RNAi to the brain.

，它还揭示了与Biogen和Genentech的研发联盟。塞科承认，一家初创公司在如此早期就与两家大型制药公司建立合作关系是不寻常的，但她表示，这些交易表明了业界对将RNAi带入大脑的兴趣。

The alliances provided Atalanta with non-dilutive capital that enabled the company to embark on additional preclinical research that further de-risked its technology platform, Secor said. CNS targets of the Genentech partnership remain undisclosed. Huntington’s was part of the Biogen alliance, but Secor said that agreement is unwinding “for business reasons.” Atalanta now has full control of the Huntington’s programs, one of which the startup aims to advance to human testing later this year..

塞科说，这些联盟为亚特兰大提供了非稀释性资本，使该公司能够开展额外的临床前研究，进一步降低其技术平台的风险。基因泰克合作伙伴关系的中枢神经系统目标仍未披露。。。

Other companies are further along in development with Huntington’s drug candidates. Last month,

其他公司正在与亨廷顿的候选药物一起进一步发展。上个月，

Novartis agreed to pay $1 billion for rights to a PTC Therapeutics small molecule on track for pivotal testing

诺华同意斥资10亿美元购买PTC治疗小分子的关键测试权利

. Roche and Ionis Pharmaceuticals are partners in the development of tominersen, an antisense oligonucleotide (ASO) that has weathered

罗氏和Ionis制药公司是tominersen开发的合作伙伴，tominersen是一种已经风化的反义寡核苷酸（ASO）

clinical trial setbacks

临床试验挫折

. Secor describes Atalanta’s technology as a more efficient way of selectively delivering a potent Huntington’s therapy into the brain.

塞科（Secor）将亚特兰大的技术描述为一种更有效的方法，可以选择性地将有效的亨廷顿疗法送入大脑。

“There is no other oligonucleotide that has been able to achieve what we have in terms of knockdown and durability,” Secor said. “I think most people would argue ASO chemistry is good, but RNAi is the next generation.”

塞科说：“在击倒和耐久性方面，没有其他寡核苷酸能够达到我们所拥有的水平。”。“我认为大多数人会认为ASO的化学反应很好，但RNAi是下一代。”

The other Atalanta program on track to the clinic is a potential treatment for a rare form of epilepsy driven by gain-of-function variants in the KCNT1 gene. Patients who have this disease can experience 50 to 100 seizures daily that aren’t treatable with existing anti-seizure drugs, Secor said. Atalanta’s therapy is designed to reduce levels of KCNT1 protein.

另一个即将进入临床的亚特兰大计划是一种潜在的治疗方法，用于治疗由KCNT1基因功能获得性变异驱动的罕见癫痫。塞科说，患有这种疾病的患者每天可能会出现50到100次癫痫发作，而这些发作是现有抗癫痫药物无法治疗的。亚特兰大的治疗旨在降低KCNT1蛋白的水平。

In mouse studies, a 50% reduction in protein led to a 70% reduction in seizures, Secor said..

Secor说，在小鼠研究中，蛋白质减少50%会导致癫痫发作减少70%。。

Evaluating safety is the main goal of the planned Phase 1 test of the KCNT1 therapy, but this study will also enable Atalanta to quickly demonstrate its technology can work in humans. That’s because seizure activity can also be measured. Reducing seizures in the trial will provide some clinical proof of concept for the drug, and for Atalanta’s technology, Secor said..

评估安全性是KCNT1疗法计划的第一阶段测试的主要目标，但这项研究也将使亚特兰大能够快速证明其技术可以在人类中发挥作用。这是因为癫痫发作活动也可以测量。塞科说，减少试验中的癫痫发作将为该药物和亚特兰大的技术提供一些临床概念证明。。

Atalanta’s latest financing is a Series B round co-led by EQT Life Sciences and Sanofi Ventures. Other participants include new investors RiverVest Venture Partners, Novartis Venture Fund, funds managed by abrdn Inc, Pictet Alternative Advisors, Mirae Asset Financial Group, and GHR Foundation alongside earlier investor F-Prime Capital..

亚特兰大的最新融资是由EQT生命科学公司和赛诺菲风险投资公司共同领导的B轮融资。其他参与者包括新投资者RiverVest Venture Partners、诺华风险基金、由 abrdn Inc、Pictet Alternative Advisors、Mirae Asset Financial Group和GHR Foundation以及早期投资者F-Prime Capital。。

Besides the Huntington’s and KCNT1 programs, the Atalanta pipeline currently spans Alzheimer’s disease, pain, and unspecified CNS disorders driven by multiple undisclosed targets. Atalanta is not actively looking for more partners right now, but Secor is leaving the door open.

除了亨廷顿舞蹈病和KCNT1项目外，亚特兰大的管道目前还涵盖阿尔茨海默氏病，疼痛和由多个未公开目标驱动的未指明的中枢神经系统疾病。亚特兰大目前并没有积极寻找更多的合作伙伴，但塞科尔正在敞开大门。

“We’ve got a pretty powerful platform that can basically knock down any transcript in the brain where there’s genetic validation that it’s disease causing,” she said. “There’s a whole universe of targets and we are open to inbound interest. Right now, we’re focused on filing our [investigational new drug applications].

她说：“我们有一个非常强大的平台，基本上可以敲除大脑中的任何转录本，在那里有基因验证它是致病的。”。“有一个完整的目标范围，我们对入境利益持开放态度。目前，我们专注于提交[研究性新药申请]。

But you know, we might be open [to partnerships] in 2025.”.

但你知道，我们可能会在2025年（对伙伴关系）持开放态度。”。

Image by Atalanta Therapeutics

亚特兰大治疗公司图片

Topics

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Alicia Secor

Alicia Secor 的

Atalanta Therapeutics

亚特兰大治疗学

Clinical Trials

临床试验

Epilepsy

癫痫

Huntington's disease

亨廷顿舞蹈病

INVEST 2025

投资2025

RNA interference

RNA干扰

Startups

创业公司

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