A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy

X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
The protein myotubularin is needed for muscle development, movement and breathing. A gene called MTM1 tells the body to make myotubularin. XLMTM is caused by changes, or mutations, in the MTM1 gene. Changes in the MTM1 gene cause low or no levels of myotubularin to be made, so the muscles do not work properly.
Gene therapy is a way of getting a healthy copy of a gene into the body. This allows the body's cells to make a normal protein that may reduce disease symptoms. Researchers have developed ASP2957 to get a healthy MTM1 gene into the body. This could help improve muscle development and function in young children with XLMTM.
In this study, ASP2957 will be given to humans for the first time.
ASP2957 has the healthy MTM1 gene inside a type of empty (killed) virus. The virus delivers the healthy MTM1 gene directly into cells in the body. It's possible that some boys may have antibodies to the virus if they have previously been infected with a similar virus. The antibodies could stop ASP2957 from working properly and cause an immune reaction to ASP2957. To prevent this, the boys will also be given medicines to lower the immune system.
The main aims of this study are to check the safety of ASP2957, how well it is tolerated, and to find a suitable dose of ASP2957.
The study was designed in 2 phases. In Phase 1, different small groups of boys will receive lower to higher doses of ASP2957. Each boy will receive a single infusion of ASP2957. Any medical problems will be recorded for each dose. This is done to find a suitable dose of ASP2957 to use in Phase 2.
In Phase 2, another small group of young boys will receive a single infusion of ASP2957. The most suitable dose of ASP2957 worked out from Phase 1 will be used.
The boys will be checked for up to 1 year after their single infusion of ASP2957. After this, there will be the option for the boys to join another study so they will continue to be checked longer term.

开始日期2025-12-15

申办/合作机构

Astellas Gene Therapies, Inc.

NCT06581146

/ RecruitingN/A

A Non-interventional, Epidemiologic Study of XLMTM and Clinical Expression in the Liver

XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition.
Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM.
There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study.
This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas).
In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study.
This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.

开始日期2025-05-19

申办/合作机构

Astellas Gene Therapies, Inc.

NCT06483802

/ Withdrawn临床1期

A Multicenter, Open-Label, Dose Escalation, Phase 1b Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia Cardiomyopathy

Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene.
At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia.
In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems.
The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016.
Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion.
For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise.
After the 12-month visit, people will visit the clinic every few months for up to a few years.

开始日期2024-11-08

申办/合作机构

Astellas Gene Therapies, Inc.

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2025-01-01·Journal of Toxicologic Pathology

Time-course of changes in fibrous components in a thioacetamide-induced liver fibrosis model in cynomolgus monkeys

Article

作者: Takahashi, Miwa ; Mitori, Hikaru ; Kajikawa, Satoru ; Tsubota, Kenjiro ; Ono, Mihoko

In liver fibrosis, the possible causes of irreversibility include the accumulation of collagen I during extracellular matrix remodeling, together with the deposition of elastic fibers in later stages. Drug development targeting liver fibrosis should preferably employ models that closely mimic human diseases. To better understand the progress of fibrosis in a cynomolgus monkey liver fibrosis model, we evaluated the time-course of changes in the fibrosis score, collagens, and elastic fibers. The animals were subcutaneously administered thioacetamide twice a week (experiment 1) or once every 2 weeks (experiment 2). Liver tissues were collected at 8 and 16 (experiment 1) or 10 and 20 (experiment 2) weeks of administration, and 12 weeks after withdrawal (experiments 1 and 2). The fibrosis score was evaluated by Masson's trichrome staining. Immunohistochemistry for collagen Ia1, III, and IV, and Elastica van Gieson staining were also performed. Fibrosis was observed from week 8 (experiment 1) or 10 (experiment 2), and in most animals, it progressed during the administration period. After withdrawal, the fibrosis scores tended to decrease. Collagen IV was predominant in the early stage but was replaced by collagen I after 20 weeks in both experiments. Collagen III distributed mostly along with collagen I throughout the study period. The elastic fibers deposition was markedly limited throughout the experiment. Fibrous component examination showed that the main collagen type contributing to fibrosis shifted from collagen IV to I after 20 weeks or later and revealed that the fibrosis status is not fully reflected in the fibrosis score.

2024-03-01·Molecular therapy. Methods & clinical development

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia

Article

作者: Fonck, Carlos ; Urbinati, Fabrizia ; Medicherla, Bala ; Bolan, Fria ; Liu, Su ; Ryan, Molly R ; Halawani, Sarah ; Purushothaman, Pravinkumar ; Lynch, David R ; Davis, Crystal ; Lawlor, Michael W ; Champe, Mark ; Gonzalo-Gil, Elena ; Ngaba, Lucie V ; Meng, Hui ; Chang, Joshua C ; Johnson, Caitlin A ; Lutz, Cathleen ; Stark, Marie C

Friedreich's ataxia (FRDA) is an autosomal-recessive disorder primarily attributed to biallelic GAA repeat expansions that reduce expression of the mitochondrial protein frataxin (FXN). FRDA is characterized by progressive neurodegeneration, with many patients developing cardiomyopathy that progresses to heart failure and death. The potential to reverse or prevent progression of the cardiac phenotype of FRDA was investigated in a mouse model of FRDA, using an adeno-associated viral vector (AAV8) containing the coding sequence of the FXN gene. The Fxn^flox/null::MCK-Cre conditional knockout mouse (FXN-MCK) has an FXN gene ablation that prevents FXN expression in cardiac and skeletal muscle, leading to cardiac insufficiency, weight loss, and morbidity. FXN-MCK mice received a single intravenous injection of an AAV8 vector containing human (hFXN) or mouse (mFXN) FXN genes under the control of a phosphoglycerate kinase promoter. Compared to vehicle-treated FXN-MCK control mice, AAV-treated FXN-MCK mice displayed increases in body weight, reversal of cardiac deficits, and increases in survival without apparent toxicity in the heart or liver for up to 12 weeks postdose. FXN protein expression in heart tissue was detected in a dose-dependent manner, exhibiting wide distribution throughout the heart similar to wild type, but more speckled. These results support an AAV8-based approach to treat FRDA-associated cardiomyopathy.

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2026-05-26

·征战罕见D研习室

#079：手松不开、眼先模糊、心跳也会掉拍：肌强直性营养不良，把家族密码写进全身

Bringing medical advances from the lab to the clinic. （👆，征战罕见病，我们一起努力。）根据卫健委公布的罕见病目录（共两批，207种），美国Healsan恒祥医学科技将持续对所有罕见病进行解读和大数据分析，以其能够为从事罕见病研究的生物制药公司、医生科学家及患者提供一些有用的信息。 👇对于罕见病患者来说，最难的就是第一时间找到合适的医生确诊和治疗；本专辑通过大数据分析找到对罕见病有研究的医生及医院的信息。（点击👆，获取研究罕见病的医生信息）导语有些病会在家族里留下一串暗号：外公年轻时白内障，母亲握拳后松不开，舅舅心脏传导异常，孩子出生后肌张力低、吃奶弱、呼吸费力。很多年后，答案才浮出水面: 肌强直性营养不良，英文Myotonic Dystrophy，简称DM。它像一封被延迟投递的遗传信，拆开时，肌肉、心脏、眼睛、内分泌、呼吸和大脑都可能写着同一个落款。一、一个“松不开”的动作，牵出全身疾病地图肌强直性营养不良是成人最常见的肌营养不良类型之一，中国《第一批罕见病目录》第78项收录为“肌强直性营养不良 / Myotonic Dystrophy”。临床上主要分为DM1和DM2：DM1来自DMPK基因3’非编码区CTG重复扩增，DM2来自CNBP基因内含子1区CCTG重复扩增；二者都属于常染色体显性遗传，父母一方携带致病扩增时，每个孩子有约50%机会遗传。它最有辨识度的表情，是“肌强直”: 握拳、拧门把、拿筷子后，肌肉像慢半拍，迟迟放松不了；随后可能出现面部、颈部、远端肢体或近端肌无力、肌萎缩、白内障、心律失常、传导阻滞、睡眠呼吸障碍、胃肠动力不足、糖尿病、甲状腺异常、脱发、不孕、认知和行为改变。机制层面，问题并非只停在肌肉：异常重复序列转录出的毒性RNA滞留在细胞核里，抓住MBNL、CELF等剪接调控蛋白，导致一串mRNA剪接错误，身体多个系统一起被卷入。全球患病率差异很大，2022年系统综述给出的合并估计约为9.99/10万人，DM1约9.27/10万人，DM2约2.29/10万人；中国全国患病率仍缺高质量人群数据，罕见病诊疗指南引用约1/8000的估计，近年中国多中心队列和DM1患者登记研究正在补齐临床与遗传图谱。研究肌强直性营养不良Myotonic Dystrophy的大数据分析（2026) 我们针对该疾病做了基于SCI论文的大数据分析报告，通过分析SCI论文，找到该疾病临床科研中的中国专家。特别需要说明的是，该报告只是提供了针对特定疾病的临床科研水平突出的医生，欲进一步确定该医生的临床水平，请结合该医生所在医院同事、或者其他医院小同行的评价。另外，该报告仅限于使用的检索词范围内，具有局限性；且排序与临床水平无关。特别声明：本期为第一版，仅作抛砖引玉用；如果您发现推文有疏漏或者任何错误，请随时与文末的科研助手联系，我们会在第二版中更新。检索数据库：Medline 检索工具：Healsan™大数据检索及分析机构：Healsan LLC（美国恒祥医学科技）检索词: (((((((((((((((((((((((((((((((((Myotonic Dystrophy[MeSH Terms]) OR (Myotonic Dystrophy[Title/Abstract])) OR (myotonic dystrophy[Title/Abstract])) OR (myotonic dystrophies[Title/Abstract])) OR (Dystrophia Myotonica[Title/Abstract])) OR (dystrophia myotonica[Title/Abstract])) OR (congenital myotonic dystrophy[Title/Abstract])) OR (myotonic dystrophy type 1[Title/Abstract])) OR (myotonic dystrophy type I[Title/Abstract])) OR (Steinert disease[Title/Abstract])) OR (Steinert's disease[Title/Abstract])) OR (Steinert syndrome[Title/Abstract])) OR (Steinert's syndrome[Title/Abstract])) OR (DMPK-related myotonic dystrophy[Title/Abstract])) OR (DMPK related myotonic dystrophy[Title/Abstract])) OR (myotonic dystrophy type 2[Title/Abstract])) OR (myotonic dystrophy type II[Title/Abstract])) OR (proximal myotonic myopathy[Title/Abstract])) OR (proximal myotonic dystrophy[Title/Abstract])) OR (CNBP-related myotonic dystrophy[Title/Abstract])) OR (CNBP related myotonic dystrophy[Title/Abstract])) NOT (cardiovascular[Title])) NOT (coronary[Title])) NOT (stroke[Title])) NOT (hypertension[Title])) NOT (diabetes[Title])) NOT (pregnancy[Title])) NOT (renal[Title])) NOT (dialysis[Title])) NOT (vitamin B12 deficiency[Title])) NOT (folate[Title])) NOT (folic acid[Title]))) AND (("2021/01/01"[Date - Publication] : "3000"[Date - Publication])) 从2021年1月1日起截止目前，全球共发表该疾病研究论文1109篇，其中中国学者发表的论文数量为60篇。接下来我们对中国学者在该领域发表的论文（包括合作论文）进行了大数据分析。（SCI论文的时间）研究最活跃的中国医院如果按照发表的SCI论文累计影响因子的次序，在该领域发表SCI论文累计影响因子最高的是中南大学湘雅医院，发表了4篇该领域的研究论文；其他位居前列的医院依次为：中南大学湘雅医院。浙江大学医学院附属儿童医院四川大学华西医院。福建医科大学附属第一医院。中南大学湘雅二医院。上海交通大学医学院附属瑞金医院。复旦大学附属华山医院。山东第一医科大学第一附属医院（山东省千佛山医院）。吉林大学第一医院。上海市浦东新区公利医院。 (单位统计。备注：只统计第一作者的论文所在单位；即，论文检索下载后，每篇论文只保留第一作者的单位，然后统计每个单位的论文数。当同一单位有不同拼写时，PubMed会按照两个不同单位处理。) 最活跃的中国医学专家如果按照发表的SCI论文累计影响因子的次序，在该疾病研究中发表SCI论文累计影响因子最高的中国医生是：黄飞鹤（Huang, Feihe）单位：浙江大学化学系、斯托达特分子科学研究所、浙江大学杭州国际科创中心—杭州之江有机硅化工有限公司联合实验室、浙江-以色列自组装功能材料联合实验室、浙江大学杭州国际科创中心。刘艳霞（Liu, Yanxia）单位：电子科技大学材料与能源学院。黄锟（Huang, Kun）单位：中南大学湘雅医院神经内科、国家老年疾病临床医学研究中心。许军（Xu, Jun）单位：浙江大学医学院附属儿童医院遗传与代谢科、国家儿童健康与疾病临床医学研究中心、浙江大学医学院转化医学研究院、浙江省癌症代谢前沿医学重点实验室、浙江大学肿瘤中心。赵玮琦（Zhao, Weiqi）单位：浙江大学医学院附属儿童医院遗传与代谢科、国家儿童健康与疾病临床医学研究中心、浙江大学医学院转化医学研究院、浙江省癌症代谢前沿医学重点实验室。 Koon, Alex Chun 单位：香港中文大学理学院生命科学学院。王芳（Wang, Fang）单位：中国农业科学院畜禽营养与饲养全国重点实验室、中国农业科学院北京畜牧兽医研究所、瓦赫宁根大学动物营养组。卜登攀（Bu, Dengpan）单位：中国农业科学院畜禽营养与饲养全国重点实验室、中国农业科学院北京畜牧兽医研究所、中国农业科学院—埃塞俄比亚农业研究所—世界农用林业中心作物—林木—畜牧综合系统联合实验室、青岛农业大学动物科学与技术学院。谭洋（Tan, Yang）单位：南京大学生命科学学院、医药生物技术全国重点实验室。吴兴新（Wu, Xingxin）单位：南京大学生命科学学院、医药生物技术全国重点实验室。（该领域最活跃的研究者。备注：只统计第一作者和最后一位作者署名发表的论文数。如果作者的名字有不同拼写时，会被PubMed当成不同作者。如张三丰的拼写可能会有：Zhang,Sanfeng、Zhang,San-feng、或者Zhang,San Feng等不同拼写方式；部分期刊可能只会写Zhang,S，PubMed检索平台就会按照不同作者处理。）中国专注该疾病的医生学术圈如下所示。（中国医生科学家及其学术网）二、在中国，患者能把求助清单写给谁？在中国，肌强直性营养不良的政策入口首先是五部门发布的《第一批罕见病目录》：国家卫生健康委员会、科学技术部、工业和信息化部、国家药品监督管理局、国家中医药管理局共同把它纳入目录。诊疗端，国家卫健委发布《罕见病诊疗指南（2019年版）》，由国家卫生健康委罕见病诊疗与保障专家委员会办公室、中国医学科学院北京协和医院牵头制定；指南中“强直性肌营养不良”章节提示需关注肌强直、肌无力、多系统受累、基因检测、心电图、眼科、内分泌、呼吸和遗传咨询。就医网络方面，全国罕见病诊疗协作网已从2019年的324家成员医院扩展到419家，协作网承担相对集中诊疗、双向转诊、病例登记、规范诊疗和临床研究等任务。公益与非营利支持方面，可关注中国罕见病联盟及其实体运营机构北京罕见病诊疗与保障学会、北京病痛挑战公益基金会、中华社会救助基金会、蔻德罕见病中心（CORD）、罕见病信息网、中国人口福利基金会罕见病关爱基金、瓷娃娃罕见病关爱中心等。病痛挑战基金会的“罕见病医疗援助工程”按《第一批罕见病目录》覆盖病种，提供医疗资源转介、医保信息、药物进展和个案资金援助。单病种层面，国内公开资料中尚少见全国性DM专门基金会，患者通常借助罕见病平台、神经肌肉病社群、遗传咨询门诊和协作网医院形成支持链条；海外可参考Myotonic Dystrophy Foundation、Muscular Dystrophy Association、英国Myotonic Dystrophy Support Group及其线上互助社群。三、新药战场：科学家正在拆那串“重复密码” 今天的DM治疗仍以症状管理和多学科随访为主，Myotonic Dystrophy Foundation提示目前还没有能够减缓疾病进展的治疗，常见管理包括心脏监测、呼吸评估、白内障处理、睡眠管理、康复训练、遗传咨询，以及针对肌强直的药物研究。真正让行业心跳加速的，是RNA药物、AAV、反义寡核苷酸和基因调控平台。Avidity的delpacibart etedesiran（del-desiran，原AOC 1001）靶向DMPK，已进入全球Ⅲ期HARBOR研究；Novartis在2026年宣布完成对Avidity的收购，del-desiran成为其神经科学后期管线的重要资产。Dyne Therapeutics的DYNE-101/zeleceiment basivarsen采用TfR1靶向递送反义寡核苷酸，在ACHIEVE研究中评估安全性、药效学和功能指标。Vertex与Entrada合作推进VX-670，面向DM1开展Ⅰ/Ⅱ期临床。ARTHEx Biotech的ATX-01通过抑制miR-23b提高MBNL蛋白表达，处于ArthemiR Ⅰ/Ⅱa研究，并在2026年获得FDA快速通道资格。Sanofi的SAR446268是一种一次性AAV基因疗法，设计目标是沉默DMPK表达，2025年获得FDA快速通道资格。PepGen的PGN-EDODM1、AMO Pharma的tideglusib、Sarepta的SRP-1003、Lupin的缓释美西律PR，也分别从核酸递送、先天/儿童起病DM1、siRNA和肌强直症状控制切入。MDF 2026年管线图还列出Design Therapeutics、Arrakis、Astellas Gene Therapies、LoQus23、Modalis、Rgenta等早期项目。这个领域仍在等待第一款真正改写病程的药。结尾肌强直性营养不良最值得警惕的地方，是它常常披着“小毛病”的外衣出现：手松不开、眼睛早早长白内障、走路容易绊、白天总困、心电图异常、家族里好几个人“体质相似”。这些碎片放在一起，可能就是一张遗传病拼图。早做基因检测，早做心脏和呼吸管理，早把家庭成员纳入遗传咨询，很多风险可以提前被看见。对患者来说，名字带来方向；对家庭来说，诊断带来选择；对科研来说，那串重复扩增的密码，正等待被更精准地改写。声明： A, 本报告为PubMed检索平台仅以检索式报告的结果进行的可视化报告。 B, 大数据分析无任何排名意义。 C，本推文很可能存在谬误；也请各位同仁多多指正，以促进我们提供越来越高质量的推文。 D，如果您是从事该疾病临床诊治的医生，并希望更多人知道您，请把您的姓名、单位、出诊时间、及挂号方式发给科研助手；我们会集中收集后统一发布。请联系微信号Healsan E，本文仅为学术交流，不构成任何临床建议。 ▼ 本公众号由美国Healsan (恒祥医学科技)运营。投稿及商务联系Jason老师；微信号：hanson-006；提供特惠码：MELT99，可以获得优惠。美国Healsan专长于Healsan医学大数据分析（Healsan™）、及基于大数据的Hanson临床科研服务（HansonCR™）和医学编辑服务（MedEditing™）。主要为医生科学家、生物制药公司和医院科研处等提供文献计量分析和SCI论文润色、编辑、选刊等服务，成为诸多机构的“临床科研外挂”。网址： https://healsan.com/ （👆，征战罕见病，我们在行动！） ▼ Healsan医路成长更多免费资源：（点击👆图片，进入自己感兴趣的专辑。或点击“资源”，浏览本公众号所有资源）

2026-05-22

·EndPoints

Amgen CFO to follow David Reese into retirement; Josh Smiley is out at Zai Lab

→ Peter Griffith will be the second high-profile exec to leave Amgen this year when he retires on Sept. 1. Thomas Dittrich will replace him as CFO and return to Amgen in July. Dittrich handled CFO responsibilities at Swiss dermatology biotech Galderma and had an eight-year stint with Amgen, where he was VP, finance corporate planning and chief accounting officer from 2010-14. Like Dittrich at Galderma, Griffith joined Amgen in October 2019. As Endpoints News previously reported , technology chief David Reese announced his retirement last month and will stay with Amgen until the end of June. Nike vet Sean Bruich , the AI and data leader since 2024, will take over as chief technology officer. Amgen makes the announcement in the midst of a firestorm over its rare disease drug Tavneos , which was approved in 2021 for patients with ANCA-associated vasculitis (AAV). The FDA is ramping up the pressure to withdraw Tavneos from the market after the regulator found eight deaths linked to the drug. The Wall Street Journal further tightened the screws by reporting 20 Tavneos-related deaths in Japan, but Amgen hasn’t wavered. “Based on all available data, Amgen continues to believe Tavneos demonstrates effectiveness and a favorable benefit–risk profile for patients living with AAV,” the company said in a statement. → The board of directors at Zai Lab has determined that Josh Smiley “will cease serving” as president and COO, according to an SEC filing . A press release was equally terse, saying the move was “aimed at strengthening operational performance and efficiency.” CEO Samantha Du will step in, along with other staffers, and there’s nothing that indicates a replacement is on the horizon. Zai Lab took a chance on Smiley in 2022 after he was ousted from Eli Lilly for having an “inappropriate personal relationship” with an employee. At the time of the hiring, Du said she was “very confident that his commitment to high ethical standards and operational excellence will be instrumental in our mission to become a leading global company.” Now we’re left to wonder if it was worth the gamble. → Jeffrey Brown has landed at Sanofi as head of neurology, according to a LinkedIn post . Brown just completed a three-year run as CSO of Boston-based CNS biotech Sensorium Therapeutics after stops at Deep Genomics (VP of preclinical research), Voyager Therapeutics (senior director of neuroscience) and Wave Life Sciences (director, CNS biology). As the Belén Garijo era begins, neurology is one of Sanofi’s four key therapeutic areas, along with immunology, vaccines and rare diseases. The French pharma makes the multiple sclerosis drug Aubagio and is developing other treatments for the condition. Its BTK inhibitor tolebrutinib has been plagued by clinical flops — missing the mark in primary progressive multiple sclerosis in December — and an FDA rejection the same month in non-relapsing secondary progressive multiple sclerosis. Sanofi is also testing an anti-CD40 candidate named frexalimab in MS and type 1 diabetes. And last year, it purchased an Alzheimer’s drug from Vigil Neuroscience that’s now known as SAR448851 . → Fred Chereau is taking the top spot at Sanofi-backed hearing loss biotech Sensorion . Chairman Amit Munshi , the former CEO of Arena Pharmaceuticals , had replaced Nawal Ouzren on an interim basis. It’s the first CEO job for Chereau since he ran LogicBio from 2016-22. AstraZeneca ’s rare disease subsidiary Alexion bought the gene editing company at a 667% premium and Chereau had led strategy and business development at Alexion ever since. “I think we are bringing something which is quite unique today to Alexion. It’s experience in genomic medicine that they don’t have, especially at preclinical and translational level, including clinical-stage,” Chereau told Endpoints when the deal was made. → Danish obesity player Antag Therapeutics has made it official with Philip Just Larsen as CEO. Larsen had chaired the board at Antag since 2018 and had been filling in since Jörg Möller ’s quick exit. You may know Larsen from his days as CEO of SixPeaks Bio , a Swiss-based obesity upstart that was acquired by AstraZeneca. He told Endpoints in November 2025 that Antag was having “lots of ongoing discussions with partners who recognize the value of the pipeline.” Meanwhile, Möller joined the board of directors at Sweden’s Alligator Bioscience this month. → Autoimmune cell therapy maker Artiva Biotherapeutics has brought Diego Miralles on board as president and head of R&D. He will help the San Diego biotech prepare for registrational testing in refractory rheumatoid arthritis, the company said. Miralles had been on the Artiva board for the past two years. Miralles was CEO of Vividion Therapeutics when Artiva CEO Fred Aslan was president at the small molecule company. Miralles also led Laronde , the Flagship Pioneering -incubated startup that ran into data integrity issues and later rebranded as Sail Biomedicines . Miralles also held roles as president of Adaptive Therapeutics and helped found Johnson & Johnson ’s JLABS program for biotech startups. → Orca Bio has tapped Bijan Nejadnik as CMO, while his predecessor Scott McClellan takes a different role as chief development officer. Nejadnik comes to Orca after nearly three years with Tanabe Pharma America as CMO and head of R&D and regulatory affairs. The original PDUFA date for Orca’s cell therapy was April 6, but the FDA pushed back the decision by three months to digest additional manufacturing information. → Melissa Koomey revealed on LinkedIn that she’s the new North American president and general manager at Recordati Rare Diseases . Koomey had a number of roles over a 12-year career at Gilead , including VP of the US HIV business unit and head of global commercial operations. In 2024, she became chief commercial officer at Bluejay Therapeutics , a hepatitis biotech that Mirum Pharmaceuticals snapped up for $620 million upfront. → Before releasing an abstract for its Nectin-4 directed ADC CRB-701 and presenting the data in more detail at next week’s ASCO meeting, Corbus Pharmaceuticals introduced its first-ever business chief. Nishant Saxena is a longtime Evercore vet who moved to industry last year as CFO of Krystal Biotech sub Jeune Aesthetics . → Antengene has promoted Bing Hou to CSO, a step up from his days as a VP heading up discovery science and translational medicine. The move comes a few months after the Shanghai biotech linked arms with UCB on a T cell engager deal. It served as the backbone to the Belgian drugmaker’s growing TCE franchise, which will soon consume Candid Therapeutics in a $2 billion upfront acquisition . Prior to joining Antengene in 2019, Hou worked on ADCs and CAR-T programs at Abmart in China. → Genetic medicines shop Arbor Biotechnologies has named Mathew Pletcher as the successor to CSO John Murphy , a 30-year industry veteran who is retiring. The gene editing company plucked Pletcher from Weaver Biosciences , where he held the same post. He’s also been CSO at Kisbee Therapeutics and a division head for gene therapy work at Astellas Gene Therapies . The Cambridge, MA-based biotech is in early clinical testing with its experimental CRISPR therapy for primary hyperoxaluria type 1. → Pfizer ’s molecular glue partner Triana Biomedicines , which nabbed a $120 million Series B in December, has selected Mohan Bala as COO. Bala is a Tesaro alum who was chief development officer for Mersana Therapeutics when Day One Biopharmaceuticals bought it for a bargain price. (To complete this particular set of M&A Matryoshka dolls, Servier recently bought Day One for $2.5 billion.) He also served as COO of Constellation Pharmaceuticals until that too attracted a buyer . → We have reached peak Kyverna at Peer Review, as the cell therapy maker introduces Greg Martini as CFO. Martini held numerous positions at Ironwood Pharmaceuticals over a nine-year period, rising to CFO in January 2025. In the last three months, Kyverna has appointed a chief commercial officer , a technology chief and a pair of board members as miv-cel could become the first FDA-approved therapy for stiff person syndrome. → Amy Sullivan has joined Dutch lysosomal storage disorder biotech Azafaros as CFO after three and a half years at IO Biotech , which filed for bankruptcy in the wake of a narrow Phase 3 failure. Last year at this time, Azafaros raised $146 million in a Series B to bankroll late-stage trials for nizubaglustat in Niemann-Pick disease type C, as well as GM1 and GM2 gangliosidoses. → There’s a new medical chief at Passkey Therapeutics , which specializes in “synergistic drug combinations” that are identified through its Locksmith platform. Mandeep Kaur had been SVP of global medical affairs and outcomes research for rare disease at Amgen, and spent a year with Horizon before the buyout. Passkey first showed up on our doorstep in October 2024 with a $20 million seed round from Innovation Endeavors , Breakout Ventures and others. → CytomX has hired Mamata Gokhale as SVP of regulatory affairs. The Amgen vet led global regulatory affairs at Sierra Oncology before the sale to GSK , and worked for Actinium Pharmaceuticals and Perspective Therapeutics in the same capacity. CytomX’s stock $CTMX has recovered a bit since the South San Francisco biotech disclosed the death of a colorectal cancer patient who was taking an ADC that’s now known as varsetatug masetecan (or Varseta-M ) in a Phase 1 trial. → London-based NovalGen has welcomed Laurent Fischer as its chairman of the board. Fischer sold Adverum Biotechnologies to Eli Lilly last year and chairs the board at Shinobi Therapeutics . He also stays busy on the boards at Mirum Pharmaceuticals, AtaiBeckley and Lycia Therapeutics . → Pliant Therapeutics CEO Bernard Coulie has been named chairman of the board at Australian inflammatory disease biotech Atisama Therapeutics . Coulie sits on the board at Septerna and used to be chairman of Calypso Biotech , an autoimmune company that was purchased by Novartis for $250 million upfront. → Former Cubist Pharmaceuticals CEO Mike Bonney has permanently succeeded Duane Nash as chairman of Immunic , while interim chair Simona Skerjanec stays on the board. Bonney is juggling other chairman posts at Autolus Therapeutics , Dunad Therapeutics and Santa Ana Bio after stepping down from the board of directors at Alnylam . → The board at San Diego-based BlossomHill Therapeutics has grown (we’re not going to say blossomed) to eight members with the additions of ex- Gossamer Bio chief Sheila Gujrathi and former Auspex Pharmaceuticals CFO John Schmid . Gujrathi now runs another biotech called Prana Therapies , with Curie.Bio vet Ryan Sullivan as CSO. Schmid has board seats all over Southern California, including First Tracks Biotherapeutics and Design Therapeutics .

上市批准高管变更并购临床1期

2026-05-18

·BioSpace

Arbor Biotechnologies Appoints Mathew Pletcher, Ph.D., as Chief Scientific Officer

Gene therapy and rare disease leader with nearly two decades of drug development experience to focus on advancing the company’s next-generation gene editing pipeline CAMBRIDGE, Mass., May 18, 2026 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced the appointment of Mathew Pletcher, Ph.D., as Chief Scientific Officer, succeeding John Murphy, Ph.D., who is retiring after five years with the company and more than 30 years in the biopharma industry. In this role, Dr. Pletcher will lead Arbor’s scientific strategy and research organization as the company advances its wholly owned portfolio of novel gene editing therapeutics. “As we celebrate John's retirement and the foundation he helped build, including Arbor’s first IND, we are excited to welcome Mat, who brings a rare combination of scientific depth, operational expertise, and genuine passion for patients with rare and serious genetic diseases,” said Devyn Smith, Ph.D., Chief Executive Officer of Arbor Biotechnologies. “His experience leading large gene therapy research organizations, paired with his track record advancing programs from early discovery through regulatory approval, makes him the right scientific leader to help us accelerate our pipeline and realize the full potential of Arbor’s gene editing platform.” Dr. Pletcher joins Arbor from Weaver Biosciences, where he served as Chief Scientific Officer, building a platform of heterobifunctional molecules enabling targeted transcriptional regulation. Prior to Weaver, he served as Chief Scientific Officer at Kisbee Therapeutics and as SVP and Division Head for Gene Therapy Research and Technical Operations at Astellas Gene Therapies, where his team led the company’s preclinical gene therapy portfolio and manufacturing activities. Dr. Pletcher also served as Head of Rare Disease at Roche, directing early development and research activities and supporting the approval of Risdiplam (Evrysdi) for spinal muscular atrophy. Dr. Pletcher also held leadership roles at Pfizer, including Director of Medical Genetics for the Rare Disease Research Unit, and began his career at The Scripps Research Institute. He earned his Ph.D. in human genetics from the Johns Hopkins University School of Medicine and completed postdoctoral training at the Genomics Institute of the Novartis Research Foundation. “Arbor has built a differentiated suite of gene editors with the versatility and precision to address diseases that have long been out of reach,” said Dr. Pletcher. “I’ve spent my career working to bring transformative therapies to patients with serious genetic diseases, and Arbor’s platform and pipeline represent a remarkable opportunity to do exactly that. I’m thrilled to join this talented team and help advance these programs toward the patients who need them the most.” About Arbor Biotechnologies, Inc. Arbor Biotechnologies™, a clinical stage, next-generation gene editing company based in Cambridge, MA, is advancing a portfolio of first-in-class genomic medicines addressing serious diseases for which there are no existing functional cures, with the lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, in clinical studies and a CNS pipeline initially prioritizing multiple ALS targets. The company’s unique breadth of gene editing technologies goes beyond the limitations of early editing approaches to unlock access to new gene targets and expand the therapeutic reach of genomic medicines. For more information, please visit: arbor.bio. Media Contact: Peg Rusconi Deerfield Group prusconi@deerfieldgroup.com

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