Dive Brief:

潜水简介：

Intellia Therapeutics will stop work on one of its principal drug research programs and lay off more than one-quarter of its staff in a

Intellia Therapeutics将停止其主要药物研究项目之一的工作，并在一年内解雇超过四分之一的员工

restructuring

重组

meant to prioritize resources around its two most advanced experimental medicines.

旨在围绕其两种最先进的实验药物优先考虑资源。

The discontinued program, dubbed NTLA-3001, targets a rare lung disease known as alpha-1 antitrypsin deficiency. Intellia had begun a Phase 1/2 clinical trial testing the therapy in August. Alongside NTLA-3001, the company is also ending “select research-stage programs.”

该停止的计划被称为NTLA-3001，针对一种罕见的肺部疾病，称为α-1抗胰蛋白酶缺乏症。Intellia已于8月开始进行1/2期临床试验，测试该疗法。除了NTLA-3001之外，该公司还将结束“选择研究阶段计划”

Intellia will additionally prepare for a change in its scientific leadership as Laura Sepp-Lorenzino, the company’s chief scientific officer since May 2019, plans to retire at the end of the year. Birgit Schultes, who’s led Intellia’s immunology and cell therapy work since 2017, will take Sepp-Lorenzino’s place as CSO..

Intellia还将为其科学领导层的更迭做准备，自2019年5月起担任该公司首席科学官的劳拉·塞普·洛伦齐诺计划于年底退休。自2017年以来一直领导Intellia免疫学和细胞治疗工作的Birgit Schultes将接替Sepp Lorenzino担任CSO。。

Dive Insight:

潜水洞察：

For the second time in a row, Intellia is starting the new year with layoffs and research cuts.

Intellia连续第二次以裁员和裁员开始新的一年。

In January 2024, the CRISPR gene editing company said it would eliminate 15% of its workforce and pause some exploratory research. A few months prior, Intellia had

2024年1月，CRISPR基因编辑公司表示，将裁减15%的员工，并暂停一些探索性研究。几个月前，Intellia

nixed an earlier program

否决了先前的计划

for alpha-1 antitrypsin deficiency, also known as AATD.

用于α-1抗胰蛋白酶缺乏症，也称为AATD。

At the time, Intellia

当时，Intellia

said

赛义德

it intended to focus on enrolling pivotal studies of its two lead programs, launch new clinical trials and experiment with new gene editing and delivery tools.

它打算专注于注册其两个领先项目的关键研究，启动新的临床试验，并尝试新的基因编辑和传递工具。

This time around, Intellia’s primary concerns look a bit different. The company will prioritize clinical execution for those two programs, targeting the rare diseases

。该公司将优先考虑这两个项目的临床执行，针对罕见疾病

hereditary angioedema

遗传性血管性水肿

and

和

transthyretin amyloidosis

转甲状腺素蛋白淀粉样变性

, and “advance commercial readiness” to prepare for launching those medicines.

，以及“提前做好商业准备”，为推出这些药物做好准备。

Both of those programs, dubbed NTLA-2002 and nex-z respectively, are now in Phase 3, and Intellia hopes to complete or speed enrollment into those trials.

这两个分别被称为NTLA-2002和nex-z的项目目前都处于第三阶段，Intellia希望完成或加速这些试验的注册。

“Our early clinical data for both NTLA-2002 and nex-z support novel, highly differentiated product profiles that directly address the significant unmet needs of patients and prescribers in HAE and ATTR,” said Intellia CEO John Leonard in a

Intellia首席执行官约翰·伦纳德（JohnLeonard）在一份报告中说：“我们对NTLA-2002和nex-z的早期临床数据都支持新颖、高度分化的产品概况，这些产品概况直接解决了HAE和ATTR中患者和开处方者的重大未满足需求。”

Thursday statement

周四声明

. “We understand the significant potential of our late-stage programs, and within a challenging market environment, have made a difficult decision to focus our resources predominantly on NTLA-2002 and nex-z where we have the greatest opportunity to create significant, near-term value.”

“我们了解后期项目的巨大潜力，在充满挑战的市场环境下，我们做出了一个艰难的决定，将资源主要集中在NTLA-2002和nex-z上，在那里我们有最大的机会创造重大的近期价值。”

Intellia was one of a handful of companies targeting AATD with new drugmaking technologies like CRISPR.

Intellia是为数不多的针对AATD的公司之一，这些公司采用了CRISPR等新的制药技术。

Wave Life Sciences

波浪生命科学

,

,

Korro Bio

Corro生物

and

和

ProQr

ProQr

are all developing RNA medicines for the disease, which can damage the liver as well as lungs.

。

Beam Therapeutics, meanwhile,

同时，Beam Therapeutics，

expects to soon share initial trial data

预计不久将分享初步试验数据

for an AATD therapy that uses what’s known as base editing to correct the disease-causing mutation.

用于AATD疗法，该疗法使用所谓的碱基编辑来纠正致病突变。

The environment for gene editing companies has darkened over the past year or so.

在过去一年左右的时间里，基因编辑公司的环境已经变暗。

Several

几个

others

其他

have been forced to lay off staff and pare back their research plans in response to funding constraints or shifting market expectations.

为了应对资金限制或市场预期的变化，他们被迫解雇员工并削减研究计划。