Intellia Therapeutics has said it will reduce its headcount by 27% this year and shelve early-stage R&D programmes as it puts its resources behind two priority drug candidates.

Intellia Therapeutics表示，今年将裁员27%，并搁置早期研发计划，因为它将资源用于两个优先候选药物。

The specialist in CRISPR-based gene editing – co-founded by Nobel Prize winner Jennifer Doudna – said it is focusing on NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for ATTR amyloidosis as it anticipates its first commercial launch in 2027.

由诺贝尔奖获得者詹妮弗·杜德（JenniferDoudna）共同创立的基于CRISPR的基因编辑专家表示，该公司正在专注于NTLA-2002治疗遗传性血管性水肿（HAE）和nexiguran-ziclumeran（nex-z）治疗ATTR淀粉样变性病，因为它预计将于2027年首次商业推出。

The discontinued programmes are headed by NTLA-3001, an

停止的项目由NTLA-3001领导

in vivo

体内

CRISPR drug developed as a one-short treatment for lung disease associated with alpha-1 antitrypsin deficiency (AATD). The inherited disorder leads to the accumulation of abnormal AAT protein and its primary symptom is pulmonary emphysema, which can be life-threatening.

CRISPR药物被开发为与α-1抗胰蛋白酶缺乏症（AATD）相关的肺部疾病的一种短期治疗方法。遗传性疾病导致异常AAT蛋白的积累，其主要症状是肺气肿，可能危及生命。

Intellia's drug was designed to correct a mutation in the SERPINA1 gene that codes for AAT and has been discontinued along with other 'select research-stage programmes,' according to the company, which had been in preparations for a phase 1/2 trial. It is expecting to take an $8 million charge in the first quarter in connection with the layoffs..

Intellia公司表示，Intellia的药物旨在纠正编码AAT的SERPINA1基因突变，该药物已与其他“选择研究阶段计划”一起停止使用，该公司正在准备进行1/2期试验。预计第一季度将收取800万美元的裁员费用。。

'Within a challenging market environment, [we] 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,' commented Intellia's chief executive John Leonard.

Intellia首席执行官约翰·伦纳德（JohnLeonard）评论道：“在一个充满挑战的市场环境中，[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。”。

The company is still cash-rich, ending 2024 with around $862 million in hand, which will be enough to fund three ongoing phase 3 clinical trials – HAELO for NTLA-2002 in HAE and the MAGNITUDE and MAGNITUDE-2 studies of nex-z in polyneuropathy and cardiomyopathy associated with ATTR, respectively.

该公司仍然现金充裕，截至2024年，手头有约8.62亿美元，足以资助三项正在进行的3期临床试验-HAE中NTLA-2002的HAELO以及nex-z在多发性神经病和与ATTR相关的心肌病中的大小和大小2研究。

In October, Intellia reported

10月，Intellia报道

phase 2 results

第二阶段结果

with NTLA-2002 – which is pitching to become the first approved

随着NTLA-2002的推出，它将成为第一个获得批准的

in vivo

体内

CRISPR therapy – saying the results suggested it could offer a 'functional cure' for HAE by switching off a gene involved in the potentially life-threatening inflammatory attacks that characterise the disease.

CRISPR疗法-表示研究结果表明，它可以通过关闭一种与潜在威胁生命的炎症发作有关的基因，从而为HAE提供“功能性治愈”。

The first dosing in HAELO, which is expected to test a one-off 50mg dose of NTLA-2002 in around 60 HAE patients, will begin later this quarter with results and a regulatory filing expected next year.

HAELO的第一次给药预计将在大约60名HAE患者中测试一次性50mg剂量的NTLA-2002，将于本季度晚些时候开始，结果和监管文件预计将于明年提交。

Meanwhile, Intellia is also recruiting patients into the MAGNITUDE study of nex-z and intends to dose its first patients in MAGNITUDE-2 before the end of March.

与此同时，Intellia也在招募患者参加nex-z的Magnite研究，并打算在3月底之前为其第一批Magnite-2患者提供剂量。

In other news, Intellia announced that chief scientific officer Laura Sepp-Lorenazo will retire at the end of the year, to be replaced by Birgit Schultes, who has been leading the company's immunology and cell therapy operations since 2017.

在其他新闻中，Intellia宣布首席科学官LauraSepp Lorenazo将于年底退休，由Birgit Schultes接替，Birgit Schultes自2017年以来一直领导公司的免疫学和细胞治疗业务。