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Raised liver enzymes were a factor during Intellia Therapeutics\' phase 1 trial of nex-z in ATTR.\n Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but analysts are keeping faith in the gene therapy’s wider safety profile.The biotech used a Securities and Exchange Commission filing May 28 to provide an update on a trio of late-stage trials of its candidate nex-z. This included a study in transthyretin amyloid cardiomyopathy (ATTR-CM), which has seen more than 200 patients dosed to date.So far, the reported adverse events have been “similar in nature” to a phase 1 trial of the drug in ATTR-CM, Intellia said. These events have included infusion-related reactions and asymptomatic liver transaminase elevations, a term for raised levels of the ALT and AST enzymes that can be a sign of liver stress.One of the cases of raised liver transaminase was classed as grade 4—the most serious level of nonfatal adverse event. This recent event was asymptomatic and was identified by laboratory tests, according to Intellia. It then appeared to resolve without the need for hospitalization or medical intervention, and the ALT and AST enzyme levels of this individual are currently classified as grade 3 and 2, respectively.Still, the details appeared to trigger a sell-off of Intellia’s stock, which was 23% in the red in premarket trading Thursday morning. William Blair analysts branded the grade 4 event “obviously disappointing” and suggested the stock drop meant “there will be outstanding question about safety moving forward.”“However, we believe the asymptomatic nature of these elevations and low incidence still plays out for a favorable risk/benefit for nex-z in TTR amyloidosis,” the analysts added in a May 29 note. “Yes, there are other effective treatment options here with TTR stabilizers and silencers, so safety will be a consideration moving forward in this indication, but … we continue to see nex-z’s TTR knockdown profile as impressive and de-risking a positive clinical outcome.” As Intellia alluded to in yesterday’s filing, raised enzyme levels were a factor during its phase 1 trial of nex-z in ATTR, leading the company to add a lower fixed dose level to the polyneuropathy arm of that study back in 2022.There are a host of drugs on the market for several versions of ATTR including Alnylam’s Onpattro and Amvuttra, Ionis Pharmaceuticals\' Tegsedi and its AstraZeneca-partnered Wainua, which are used against polyneuropathy (damage to nerves) associated with ATTR. Pfizer’s Vyndaqel is also approved to help stabilize the transthyretin protein for cardiomyopathy associated with TTR.Intellia’s plan remains to submit an approval application to the FDA for nex-z in transthyretin amyloid polyneuropathy (ATTRv-PN) in 2028 with an anticipated U.S. commercial launch in 2029, the biotech said in its filing.  Before that, the company hopes to submit a separate FDA approval application next year for the gene therapy in hereditary angioedema. The company is currently running phase 3 trials in both those indications, in addition to the ATTR-CM study.

近日，BioBAY园内企业星锐医药科学创始人程强博士凭借在mRNA靶向递送领域的贡献，荣膺首届“Wiley新锐科学家奖”。 该奖项重点聚焦生命科学领域，由中国科学院文献情报中心期刊分区代表团队严格遴选，并经行业权威专家历时3个月评审，最终仅有5位杰出的新锐科学家脱颖而出。程强博士作为BioBAY青年科学家的杰出代表，正以先锋之姿，为科研事业注入源源不断的新动力。据了解，程强博士是北京大学未来技术学院研究员、核糖核酸北京研究中心研究员，入选北京大学博雅青年学者等。主持国家重点研发计划、国创中心“揭榜挂帅”、国家自然科学基金等科研项目。长期从事新型mRNA靶向药物研发和疾病治疗，相关研究成果在Nature Nanotech，Nature Materials， Nature Commun 等杂志上发表50余篇（含5篇ESI高被引论文），申请授权20多项国际专利。相关一系列专利独家许可生物公司完成超4亿美元融资进行临床转化，并推动至少2项管线进入到临床1期阶段。程强博士的科研之路始于2011年到2016年期间，他在北京大学分子医学研究所攻读博士学位，师从国内小核酸（siRNA）制药的领军人物梁子才教授。彼时，LNPs作为递送siRNA的主流技术，第一款问世的Onpattro药物正是采用了MC3 LNPs载体。程强博士在读博期间，便接触到了众多前沿的siRNA药物研发项目，也正是在那时，他开始深入研究LNPs载体技术。程强博士联合创办的星锐医药于2021年8月落户BioBAY，公司聚焦肝外靶向递送技术，致力于研发以mRNA为核心的创新药物。公司建立了近4000平米的研发和中试生产基地，打造了围绕化学、mRNA和LNP制剂的核心产业链，切实攻克mRNA药物研发的核心难点。累计合成筛选了3000多个新型阳离子脂质分子库和上万种脂质纳米颗粒(LNPs)，并在小动物和非人灵长类动物中实现了多种肝外组织和细胞特异性靶向递送。公司已在mRNA预防性疫苗、治疗性肿瘤疫苗和在体生成细胞疗法（in vivo CarT）布局丰富管线，其中呼吸道合胞病毒疫苗（STR-V003）已获得FDA临床试验许可，个性化肿瘤疫苗（STR-V005）和in vivo CarT技术（STR-P004）先后进入IIT临床试验阶段。值得一提的是，新型递送平台是BioBAY重点布局方向。已吸引了包括星锐医药、炫景生物、艾博生物、圣因生物等多家企业的率先布局和深入研究。BioBAY的科学家们年轻且充满活力，他们以高效执行力和坚定信念，专注于攻克科研难题，推动新型递送平台在药物开发中的不断拓展。展望未来，我们有理由相信，在程强博士等青年科学家的带领下，BioBAY的科研力量将持续壮大，新型递送技术也将不断拓展，为人类的健康事业贡献更多创新成果。关于Wiley新锐科学家奖“新锐科学家奖”重点聚焦生命科学领域，由Wiley于2024年创立，邀请生命科学领域中国知名科学家组成评奖委员会，并委托中国科学院文献情报中心期刊分区表团队支撑奖项遴选，旨在更好地推动与支持中国青年科研人才发展，并为他们的科研事业注入动力。本次评选共有500余名学者参加评选，经过严谨、公正且多维度的评审流程，专家委员会从众多卓越的候选人中遴选出5位获奖者。奖项评选专家委员会（排名不分先后，按照姓氏首字母排序）：曹晓风 院 士陈 坚 院 士种 康 院 士董尔丹 院 士高 福 院 士贾伟平 院 士康 乐 院 士时松海 院 士宋 保 院 士王红阳 院 士朱永官 院 士傅向东 研究员高彩霞 研究员高 歌 教 授刘秀云 教 授杨立英 研究员周 军 教 授张 勇 研究员朱 冰 研究员撰写：宋佳铭责编：于嘉敏审核：任旭推荐阅读研发动态丨爱科诺：2期研究获积极数据！口服高选择性TYK2/JAK1抑制剂AC-201治疗中重度斑块型银屑病研发动态丨兴盟生物鸡尾酒单抗克瑞毕®在中国澳门获批上市研发动态丨全球首创！宜联生物VEGF ADC申报IND