临床项目与研发进展 - 公司预计到2025年底将有四个活跃的临床项目[4] - ENTR-601-44的临床研究已在英国、欧盟和美国获得授权[4] - ENTR-601-45的临床研究已在英国和欧盟获得授权[4] - ENTR-601-50计划在2025年下半年提交全球监管申请[4] - 公司计划在2025年第二季度启动ENTR-601-44的Phase 1/2多次递增剂量临床研究[36] - ENTR-601-44和ENTR-601-45的多次递增剂量（MAD）研究预计分别在2025年第二季度和第三季度启动[65] - ENTR-601-50的全球Phase 1/2 MAD监管申请预计在2025年下半年提交，研究启动预计在2025年第四季度[65] 财务状况 - 截至2025年3月31日，公司现金、现金等价物和可市场证券总额为3.825亿美元，预计现金资源可持续到2027年第二季度[5] - 公司预计到2027年第二季度有足够的现金流[65] 药物安全性与疗效 - ENTR-601-44的Phase 1研究显示，6 mg/kg剂量下无治疗相关不良事件，且药物安全性良好[20] - ENTR-601-44在6 mg/kg剂量下显示出显著的外显子跳跃效果，与安慰剂相比具有统计学意义[31] - ENTR-601-44的药物浓度在剂量增加时呈现出剂量依赖性，支持在相对低剂量下的疗效潜力[24] - ENTR-601-44的尿液代谢物排泄量在剂量翻倍时超过翻倍，表明在增加疗效的同时毒性风险不成比例增加[26] - ENTR-601-44的剂量范围为每公斤6毫克至18毫克，预计持续6个月，参与者约24名[49] - ENTR-601-45的剂量范围为每公斤5毫克至15毫克，预计持续6个月，参与者约24名[56] - ENTR-601-44在小鼠模型中观察到的外显子44跳跃率在12周内保持显著[73] - ENTR-601-45在小鼠模型中显示出剂量依赖性外显子跳跃和肌萎缩蛋白恢复[79] - ENTR-601-50在小鼠模型中显示出高水平的外显子50跳跃，且在6周后仍然持续[85] 监管授权与市场扩张 - 公司在英国和欧盟获得了ENTR-601-44和ENTR-601-45的监管授权[58] - 预临床数据显示，ENTR-601系列产品在小鼠模型中显示出高且持久的肌萎缩蛋白恢复潜力[62]

Entrada Therapeutics (TRDA) FY Conference June 09, 2025 08:00 AM ET Speaker0 Conference. My name is Paul Choi, and I cover the biotechnology sector here at the firm. And it's my pleasure to welcome Entrada for our first session to kick off the conference. Before we begin, I'm required to make certain disclosures regarding Goldman Sachs' relationships with certain companies that may be attending or presenting here at the conference. Those relationships include investment banking, 1% or more ownership of the ...

纪要涉及的公司 Entrada Therapeutics (TRDA)，Vertex 纪要提到的核心观点和论据 1. **临床项目进展** - 到2025年底预计有四个活跃临床项目，包括Duchenne肌肉萎缩症（DMD）的ENTR 601 - 44、ENTR 601 - 45、ENTR 601 - 50项目，以及与Vertex合作的强直性肌营养不良症1型（DM1）的VX - 670项目[2][4] - ENTR 601 - 44项目在英国、美国和欧盟获临床研究授权，正快速招募；ENTR 601 - 45项目在英国和欧盟获监管批准可进行临床试验招募；ENTR 601 - 50项目预计今年下半年提交监管申请[3][4] - VX - 670全球研究已启动26个临床站点，Vertex进展良好，双方合作有里程碑付款和特许权使用费[4][5] 2. **临床前管线** - 推进临床前管线，年底将分享眼部项目数据[6] 3. **财务状况** - 资产负债表约有3.83亿美元，现金可维持到2027年第二季度，有助于公司度过关键临床数据节点[6] 4. **治疗技术优势** - 75%致病靶点在细胞内，公司利用内体逃逸载体（EEV），即环状细胞穿透肽家族，增加细胞摄取、改善内体逃逸，靶向目标器官[8] - EEV有独特化学性质和结构，可提高摄取和内体逃逸，延长半衰期、增加稳定性，具有广泛生物分布和一致可预测的药代动力学，从鼠模型到非人灵长类动物再到人类的转化一致[9][10][11] 5. **临床研究结果** - ENTR 601 - 44项目一期健康志愿者研究显示无治疗相关不良反应，目标暴露和参与情况良好，有潜力每六周给药一次，数据可用于其他DMD外显子研究[14][15][16] - 剂量与血浆浓度呈线性关系，剂量翻倍代谢物增加超一倍，有潜力在不增加毒性情况下提高疗效，有广泛治疗窗口[20][21] 6. **临床研究计划** - 44和45项目设计相似，为全球两部分研究，包括多剂量递增研究和2b期研究，44项目2b期研究有望成为美国加速批准途径基础，主要目标是安全性和耐受性，次要目标包括评估药代动力学、肌营养不良蛋白产生和功能变化[28][29][30] - 44项目剂量为6 - 18mg/kg，45项目为5、10和15mg/kg[28][31] - 2026年将持续发布数据，美国和欧洲约4万人患有DMD，有巨大未满足临床需求[27] 其他重要但是可能被忽略的内容 - 公司使命是用细胞内疗法治疗毁灭性疾病，与DMD患儿家长合作设计临床研究[7] - 除DMD和DM1外，公司有眼部项目和平台扩展计划，思考第二代、第三代EEV及其他匹配项目[12][13] - 45项目有最强大的临床前数据集，在MDX小鼠模型中实现完全功能矫正[32] - 公司现金能否覆盖2b期研究需看初始阶段招募情况[39]

BOSTON, June 02, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) today announced that the Company granted an aggregate of 23,820 restricted stock units (“RSUs”) to six newly-hired non-executive employees under the Company’s 2025 Inducement Equity Plan (the “Inducement Plan”), effective as of June 1, 2025. The inducement grants were previously approved by the Compensation Committee of the Company’s Board of Directors, as a material inducement to the new employees’ entry into employment wit ...

About ENTR-601-45 ENTR-601-45, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the second product candidate within Entrada's Duchenne muscular dystrophy franchise from its growing pipeline of EEV-therapeutics. Each EEV-PMO therapeutic candidate has an oligonucleotide sequence designed and optimized for the specific subpopulation of interest. ENTR-601-45 is designed to address the underlying cause of Duchenne due to mutated or missing exons in the DMD ...

Entrada Therapeutics (TRDA) FY 2025 Conference May 20, 2025 02:00 PM ET Speaker0 Hello, and welcome to the latest in this series of fireside chats here at the H. C. Wainwright BioConnect Conference. Thank you very much all for joining us. My name is Ram Selvaraju, and I'm a Managing Director and Senior Healthcare Equity Research Analyst within Wainwright's Equity Research Department. I'm joined here today by the next in our series of presenting companies, Entrada Therapeutics, represented by Nathan Dowden, ...

Entrada Therapeutics, Inc. (TRDA) came out with a quarterly loss of $0.42 per share versus the Zacks Consensus Estimate of a loss of $0.78. This compares to earnings of $0.68 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 46.15%. A quarter ago, it was expected that this company would post a loss of $0.66 per share when it actually produced earnings of $0.03, delivering a surprise of 104.55%.Over the last four quarters, the co ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ____________________________ FORM 10-Q ____________________________ (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____________ to ____________ Commission File Number: 001-40969 ___________________ ...

– Receives regulatory authorization in the EU for ELEVATE-44-201, a Phase 1/2 MAD clinical study of ENTR-601-44 in patients living with Duchenne muscular dystrophy who are amenable to exon 44 skipping – – Company remains on track to initiate ELEVATE-44-201 and ELEVATE-45-201 in Q2 and Q3 2025, respectively – – Cash runway expected into Q2 2027 with $383 million in cash, cash equivalents and marketable securities as of March 31, 2025 – BOSTON, May 08, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nas ...

– Company on track to initiate ELEVATE-45-201 study in Q3 2025 – – ELEVATE-45 regulatory filings submitted in the EU, with regulatory review ongoing –  – ELEVATE-45 is the second of three novel exon skipping Duchenne programs the Company expects to progress into global clinical development in 2025 – BOSTON, March 24, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (NASDAQ:TRDA) today announced it has received authorization from the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MH ...