Part A of this trial evaluated the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF (LY888891) and evaluated the Akouos Delivery Device, together with the Precision Delivery Mechanism (as applicable), to safely achieve the intended product performance.
Part B of the trial is an open-label, global expansion phase to confirm the efficacy and safety of one dose level of AAVAnc80-hOTOF administered in one or both ear(s). Part B will also evaluate the Intracochlear Delivery System (IDS), which consists of the Akouos Delivery Device, the Precision Delivery Mechanism, and an off-the-shelf syringe.
Part C is a region-specific expansion phase. Part C participants will receive the same dose level of AAVAnc80-hOTOF as participants in Part B, in one or both ear(s) utilizing the IDS.
Part D is a sequential administration phase that allows participants who received AAVAnc80-hOTOF previously in a single ear to receive AAVAnc80-hOTOF administration in the other ear utilizing the IDS.

开始日期2023-09-15

申办/合作机构

Eli Lilly & Co.

NCT05572073

/ RecruitingN/A

A Natural History Study in Individuals With Otoferlin Gene-mediated Hearing Loss

This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss.

开始日期2022-07-14

申办/合作机构

Akouos, Inc.

[+1]

100 项与 AK-OTOF 相关的临床结果

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100 项与 AK-OTOF 相关的转化医学

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100 项与 AK-OTOF 相关的专利（医药）

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104

项与 AK-OTOF 相关的新闻（医药）

2026-09-17

·PRNewswire

From GLP-1s to Gene Therapy: ENT's Next Breakthroughs Take Center Stage in Los Angeles

October's Annual Meeting of the American Academy of Otolaryngology–Head and Neck Surgery Foundation will showcase breaking science that spans widely used treatments and emerging innovations ALEXANDRIA, Va., Sept. 17, 2026 /PRNewswire/ -- The latest treatments and research advances in ear, nose, and throat care will take center stage in Los Angeles October 17-20, when more than 5,000 of the world's leading otolaryngologists gather for the 2026 Annual Meeting & OTO EXPO of the American Academy of Otolaryngology–Head and Neck Surgery Foundation. The event will highlight advances across conditions affecting hearing, breathing, sleep, speech, balance, swallowing, and head and neck health. Reporters are invited to register and review the full 2026 Annual Meeting Media Guide, featured studies, expert resources, and meeting information in the online press kit . The meeting will also examine the policy and economic pressures shaping access to care. The event comes as proposed Medicare cuts, rising practice costs, and workforce challenges put pressure on physicians' ability to invest in new technology and patient services. The Centers for Medicare & Medicaid Services has proposed 50% Medicare payment cuts for certain same-day services beginning in 2027, adding urgency to conversations at the event. "Otolaryngology is at the center of some of today's most important health and policy conversations," said Rahul K. Shah, MD, MBA, Academy CEO and executive vice president. "From gene therapy and GLP-1 medications to dementia prevention, sleep apnea, cancer, and hearing restoration, this research shows how far ENT care reaches. Our physicians need the freedom to keep innovating—and sound policies that help turn that innovation into better patient care." The Academy's 2026 media guide highlights six research frontiers: The Ozempic era comes for ENT care. As GLP-1 and GIP/GLP-1 medications become more common, ENT specialists are examining questions involving thyroid cancer risk, cancer aggressiveness, obesity-related airway disease, surgical outcomes, and patient counseling. Hidden connections across mental and physical health. Researchers are investigating whether treatment of nasal nerves may reduce migraine-related disability in patients with rhinitis, or chronically congested noses. Another study reports elevated salivary gland cancer rates among patients with eating disorders, a surprising finding that warrants further investigation. Hearing health becomes brain health. Studies explore links between hearing loss, type 2 diabetes, cognitive decline, and healthy aging. Researchers are also examining preventable risks, including how where concertgoers sit at a venue may affect noise exposure and hearing symptoms. Treating sleep apnea may benefit the whole body. New research on hypoglossal nerve stimulation—an implanted therapy that activates the nerve controlling the tongue to keep the airway open during sleep—examines potential effects on blood pressure, cardiovascular outcomes, glaucoma, and children with Down syndrome. Gene therapy moves toward hearing-restoration reality. Early studies of two groundbreaking therapies—AK-OTOF and DB-OTO—suggest that children with profound genetic hearing loss may gain access to sound and new opportunities for speech and language development. Another trial extends gene therapy research into vestibular schwannoma and skull base care. Hidden conditions gain new recognition and treatment. Research addresses "no-burp" syndrome, chronic sinus disease with nasal polyps, Ménière's disease, and sudden hearing loss—conditions that can be difficult to detect but profoundly disruptive to daily life. About the American Academy of Otolaryngology–Head and Neck Surgery The American Academy of Otolaryngology–Head and Neck Surgery represents approximately 13,000 physicians and other professionals who treat the ears, nose, throat, and related structures of the head and neck. Otolaryngologist–head and neck surgeons diagnose and treat conditions affecting patients of all ages and help people breathe, hear, speak, sleep, swallow, and connect with the world. Together, the Academy and its Foundation advance the specialty through education, research, quality improvement, advocacy, humanitarian programs, and initiatives that shape the future of patient care. SOURCE The American Academy of Otolaryngology 21% more press release views with Request a Demo

2026-09-03

·allsci.com

France clears Sensorion's gene therapy SENS-601 into human testing for GJB2 hearing loss

France's drug regulator has cleared Sensorion to begin human testing of SENS-601, its adeno-associated virus (AAV)-based gene therapy for GJB2-related hearing loss — the most common genetic cause of congenital deafness. The French National Agency for Medicines and Health Products Safety (ANSM) granted authorization on August 31, 2026, under its Fast Track assessment procedure, allowing the Montpellier-based company to open sites and enroll patients into the HearConnex Phase I/II trial. First dosing is targeted for early 2027, with clinical data expected throughout the year. GJB2 encodes connexin 26, a gap junction protein in cochlear supporting cells that maintains the potassium recycling required for hair cell depolarization and sound transduction. Loss-of-function mutations abolish this process, causing severe-to-profound hearing loss from birth. According to Sensorion, GJB2 mutations account for approximately 50% of autosomal recessive non-syndromic hearing loss. No approved pharmacological or gene therapy treatment targeting the underlying cause currently exists. HearConnex is a two-part, open-label study. Part 1 will evaluate safety and tolerability of SENS-601 via unilateral intra-cochlear administration across two ascending-dose cohorts. Part 2 will assess efficacy in an expansion cohort using bilateral administration at the selected dose. The trial will also evaluate Sensorion's proprietary injection system. Dr. Sharon Cushing of The Hospital for Sick Children (SickKids) in Toronto serves as coordinating investigator, with Health Canada's review of the Canadian clinical trial application (CTA) described as on track. Sensorion is also targeting CTA submission in Australia and an investigational new drug (IND) application with the US FDA by year-end 2026. The ANSM approval follows regulatory filings in Canada and France announced in June 2026, when Sensorion also discontinued its earlier OTOF-targeted gene therapy program, SENS-501, to concentrate resources on SENS-601. The preclinical basis for HearConnex was developed over several years in collaboration with Prof. Christine Petit's team at Institut Pasteur–Institut de l'Audition/Institut reConnect, with animal model data demonstrating hearing restoration following SENS-601 administration, according to the company. Sensorion will not be alone in clinical development for GJB2-related hearing loss: Skylark Bio began dosing patients with its AAV gene therapy SKY-GJB2 in the Phase I/II SONIX study in August 2026. Other more advanced hearing-loss gene therapy programs target OTOF mutations rather than GJB2. Regeneron’s DB-OTO, acquired through its purchase of Decibel Therapeutics, has advanced to regulatory review after producing hearing improvements in the CHORD study, while Eli Lilly’s AK-OTOF, acquired with Akouos, remains in Phase I/II development. Sensorion's operational experience from the Audiogene OTOF trial — successfully applied in its intra-cochlear surgical delivery platform and SPHYNX injection system across multiple patients and dose levels — is directly transferable to HearConnex. The company's cash runway extends to end of 2027, supported by a EUR 60 million financing in January 2026 that included a EUR 20 million strategic investment from Sanofi. Spot something wrong? Report an issue with this article

临床研究基因疗法并购临床申请快速通道

2026-08-30

·全球GCT研究

从"30天复聪"到$11.2亿：耳聋基因治疗全景

Seamless × 礼来 $11.2亿重组酶合作｜耳聋基因与全球基因治疗全局分析案例事件：Seamless×礼来$11.2亿(2026.1)，可编程重组酶"基因组手术"治疗听力损失，礼来独家许可+2个额外靶点行业里程碑：FDA 2026.4批准全球首个耳聋基因疗法(Regeneron OTOF) ｜ AK-OTOF"11岁男孩30天复聪" 三大技术路线：基因增补(获批) ｜基因编辑/重组酶(代际) ｜ RNA编辑(灵活)——礼来三线全覆盖核心风险：内耳递送壁垒/GJB2技术难度/AAV与重组酶安全/单次高定价支付一、引言:从"人工耳蜗"到"基因治疗"的听觉革命 2026年1月28日(美东),Seamless Therapeutics与礼来宣布一项超$11.2亿美元的全球研究合作:利用Seamless专有的可编程重组酶平台,开发并商业化针对听力损失的位点特异性重组酶疗法[1][2][3]。这标志着耳聋治疗从"人工耳蜗替代"迈向"基因级修复"的新纪元——而就在此前,FDA于2026年4月批准了全球首个遗传性耳聋基因疗法(Regeneron,OTOF靶点),礼来AK-OTOF更以"11岁男孩30天复聪"震惊业界[5][6][7]。耳聋基因治疗,正从"个案奇迹"走向"系统性产业"[1][5][7][10]。如果你是一名患者或家属:遗传性耳聋(尤其OTOF突变)已有获批基因疗法,更多靶点(GJB2等)在途——"听见世界"从梦想变为可及的治疗选择[5][6][7]。如果你是一名临床医生:耳聋基因治疗的"基因检测→靶点确认→内耳递送→疗效评估"路径正在标准化——掌握耳聋基因谱系与转介时机是关键[5][8][9]。如果你在biotech新药研发:Seamless×礼来$11.2亿交易揭示"基因编辑(重组酶)×内耳递送"的技术溢价——耳聋是基因治疗"低垂果实"赛道,三大技术路线(增补/编辑/RNA编辑)竞速[1][2][3][10]。二、事件核心:Seamless×礼来$11.2亿重组酶合作(2026.1) 交易要点:(1)金额——超$11.2亿美元(含首付款+里程碑+销售分成,研究合作+独占许可)[1][2][3];(2)技术——Seamless的位点特异性可编程重组酶:经设计具有高度特异性与活性,可在基因组任意目的序列插入、交换、反转或切除DNA片段——"可编程基因组手术"[2][3][4];(3)分工——Seamless设计针对听力损失相关基因突变的特异性重组酶,礼来获独家许可负责临床前/临床开发及商业化,并拥有最多2个额外靶点选择权[1][3][4];(4)战略——礼来借此拓展"不断增长的听力损失产品组合"[1][2]。三、技术深度:可编程重组酶——"基因组手术刀" 可编程重组酶是Seamless的核心技术,区别于CRISPR的"切割-修复"模式:(1)原理——重组酶识别特定DNA序列并催化位点特异性重组:可精确插入、交换、反转或切除大片段DNA——"确定性基因组手术"[2][3];(2)vs CRISPR——CRISPR依赖DSB+内源修复(易产生indel/脱靶);重组酶无需DSB、结果更确定、对大片段操作更优——对耳聋这类"需要精准修复特定突变"的场景更具优势[2][3];(3)编程性——重组酶可被设计靶向任意目的基因序列——平台可适配多靶点(礼来2个额外靶点选择权即体现平台复用)[1][3];(4)内耳适配——内耳毛细胞不可再生、数量有限,重组酶的"精准性"可最大化修复效率并最小化损伤[2][3]。四、耳聋基因谱系:从OTOF到GJB2的"靶点地图" 遗传性耳聋是高度异质性单基因病,全球约50%先天性耳聋由遗传因素导致,已定位超过150个耳聋基因:(1)OTOF(DFNB9)——耳铁蛋白,负责内毛细胞突触囊泡释放,突变导致重度-极重度感音神经性耳聋;全球首个小鼠-人体验证的耳聋基因治疗靶点(Regeneron获批/AK-OTOF)[5][6][7];(2)GJB2(DFNB1)——连接蛋白26,全球最常见的耳聋基因(占遗传性耳聋~30-50%),但因基因大小与表达调控复杂,治疗开发较难[8];(3)SLC26A4(DFNB4)——Pendrin蛋白,与内淋巴水肿相关[8];(4)其他——STRC(DFNB16)、MYO15A、TMC1、DFNA2(KCNQ4,显性)等(显性/隐性突变并存)[8][9][16];(5)显性突变新进展——DFNA2小鼠模型长期听觉功能恢复(RNA碱基编辑,EMBO Mol Med 2026)[16]。判断:OTOF因"功能缺失突变+基因可增补"成为首选靶点;GJB2患者基数最大但技术难度高,是下一波竞争焦点;显性突变(DFNA2等)依赖编辑/RNA编辑路线[5][7][8][16]。五、案例纵深:AK-OTOF的"30天复聪"里程碑 AK-OTOF(礼来$4.87亿收购Akouos)是耳聋基因治疗的标志性案例:(1)2024年1月礼来公布——一名11岁男孩(OTOF双等位突变,自幼重度耳聋)接受AK-OTOF单次内耳注射后,30天内恢复听力——这是全球首次报道的人类耳聋基因治疗成功案例[1][7];(2)技术——AAV载体携带OTOF cDNA经圆窗膜注射入耳蜗,OTOF基因过大(约6kb)超出单AAV载荷,采用双AAV载体策略[7][9];(3)意义——证明"内耳递送+基因增补"可行,为整个耳聋基因治疗赛道打开大门[7];(4)后续——哈佛/波士顿儿童医院Miles案例(2025.5)等更多患儿成功,疗效持续数年(2026.4哈佛报告)[7][8][9];(5)NEJM 2026顶刊证据——DB-OTO(现Otarmeni)研究(Valayannopoulos等,引用64次):12例OTOF相关耳聋患者中3例听力正常化,恢复自然声学听觉;CHORD研究75%患者显著改善[18][19];(6)中国领先——PMC评论指出中国在OTOF临床试验中全球领先(多中心IIT)[20]。六、全球管线全景:三大技术路线竞速表1|全球耳聋基因治疗关键管线全景(2026.8) 公司/机构产品/平台靶点技术路线阶段关键进展 Regeneron Otarmeni(lunsotogene parvec-cwha,原DB-OTO) OTOF(DFNB9) AAV基因增补(双AAV1) FDA获批(2026.4,全球首个) 首个且唯一OTOF体内基因疗法,美国免费提供;NEJM 2026:12例中3例听力正常化;CHORD研究75%显著改善[5][6][18][19] 礼来×Akouos AK-OTOF OTOF AAV基因增补临床推进 2024.1:11岁男孩给药30天内恢复听力;礼来$4.87亿收购Akouos[1][7] 礼来×Seamless 可编程重组酶平台多听力损失基因位点特异性重组酶(基因组手术) 研究合作(2026.1,$11.2亿) 精确插入/交换/反转/切除DNA片段;礼来独家许可+2个额外靶点选择权[1][2][3] 礼来×Rznomics RNA编辑疗法感音神经性听力损失 RNA编辑(ADAR) 合作(2025.5,$13亿) RNA水平修复,可逆且无基因组改变[10] Sensorion SENS-401/GJB2基因治疗 GJB2(DFNB1) AAV基因增补临床前-临床 GJB2是全球最常见耳聋基因(占遗传性30-50%),Sensorion推进GJB2基因治疗[11] Decibel(Regeneron) DB-OTO(Otarmeni) OTOF AAV基因增补 FDA加速批准 Otarmeni(DB-OTO)获FDA加速批准,治疗OTOF相关耳聋儿童与成人[12] Rinri Therapeutics 细胞治疗听觉神经元干细胞再生临床前-临床干细胞再生听觉神经元——细胞治疗新路线[13] 学术/中国 OTOF基因治疗(多中心) OTOF AAV基因增补临床(IIT) 哈佛/波士顿儿童Miles案例(2025.5);中国多中心OTOF研究:一针恢复听力,改善持续2.5年[8][9][15] 管线格局:(1)Regeneron领跑——OTOF基因疗法2026.4获FDA批准(全球首个,20名儿童16名改善),确立"AAV基因增补"的注册路径[5][6];(2)礼来组合拳——AK-OTOF(增补)+Seamless(重组酶编辑,$11.2亿)+Rznomics(RNA编辑,$13亿)——三技术路线全覆盖[1][2][3][7][10];(3)学术/中国——哈佛/波士顿儿童+中国多中心DFNB9试验(IIT)并行推进[8][9];(4)靶点扩展——Sensorion推进GJB2(最常见耳聋基因)、中国/学术多靶点延伸[8][11];(5)疗效新证据——STAT 2026.4:OTOF基因治疗试验90%参与者报告显著改善[14];(6)细胞治疗新路线——Rinri干细胞再生听觉神经元[13];(7)IND/临床试验细节——NCT05821959:AAVAnc双耳给药剂量递增试验(单次手术给药,两个剂量水平)[21];Otarmeni为FDA首个获批(单臂试验20例可评估)[6]。七、技术路线对比:增补/编辑/RNA编辑表2|耳聋基因治疗三大技术路线对比技术路线原理优势短板代表基因增补递送正常基因拷贝(OTOF cDNA) 成熟;OTOF大基因用双AAV;已获批需功能缺失型突变;AAV载荷限制 Regeneron/AK- OTOF[5][6][7] 基因编辑重组酶/Cas定点修复突变精准基因组手术(插入/交换/反转/切除);可治显性突变脱靶风险;内耳递送+编辑效率 Seamless×礼来 [1][2][3] RNA编辑 ADAR在RNA水平修复可逆;无基因组改变;瞬时调控编辑效率/持久性;技术早期 Rznomics×礼来[10] 路线判断:(1)基因增补最成熟——适合OTOF等功能缺失突变,已获批;但受AAV载荷限制(OTOF需双AAV)[5][7];(2)基因编辑(重组酶)具代际潜力——可治显性突变(需敲除突变等位基因)、大片段精准操作——Seamless技术是"下一代"方向[2][3];(3)RNA编辑最灵活——可逆、无基因组改变,适合瞬时修复——Rznomics早期布局[10];(4)RNA碱基编辑新证据——DFNA2小鼠模型RNA碱基编辑长期恢复听觉功能(EMBO Mol Med 2026)[16];(5)ASO(反义寡核苷酸)——针对剪接/单点突变,可逆、无基因组改变;(6)毛细胞再生——听毛细胞再生与基因治疗互补(HRP项目),干细胞路线(Rinri)在听觉神经元修复[13][17]。判断:路线将按突变类型共存——功能缺失型→增补;显性/复杂突变→编辑/RNA编辑;神经元损伤→细胞再生[2][5][10][13][16]。八、MNC布局:礼来的"听力损失组合拳" 礼来在耳聋基因治疗领域的布局堪称"教科书级组合拳":(1)$4.87亿收购Akouos——获得AK-OTOF(AAV增补,30天复聪案例)[1][7];(2)$11.2亿×Seamless——可编程重组酶(基因组编辑,2026.1)[1][2][3];(3)$13亿×Rznomics——RNA编辑(2025.5)[10];(4)战略逻辑——三大技术路线全覆盖+多靶点选择权(2个额外),分散单技术风险、锁定平台价值;与Regeneron的"单产品领先"形成对比,礼来选择"平台矩阵"策略[1][2][10]。九、科学维度:内耳递送的技术挑战内耳是基因治疗的"极难递送器官",三大挑战:(1)解剖屏障——血-迷路屏障+骨性耳蜗包裹,系统给药难以到达;局部给药(圆窗膜/鼓阶注射)是主流,但操作精准度要求极高[8][9];(2)靶细胞——内耳毛细胞不可再生,需在细胞尚存活时治疗(早期干预窗口);螺旋神经节/支持细胞也是靶点[8][9];(3)AAV载荷——OTOF(6kb)/MYO15A等大基因超出单AAV(4.7kb)载荷,双AAV/双载体策略是关键技术[7][9];(4)免疫与长期安全——AAV中和抗体、内耳局部免疫反应、长期表达稳定性需监测[8]。十、医学与临床维度:疗效、安全与患者选择临床关键数据与考量:(1)疗效——Regeneron:20名儿童16名听力改善(约5个月),5/12随访11个月听力恢复(部分接近正常水平);AK-OTOF:30天复聪个案;哈佛:疗效持续数年[5][6][7][8];(2)安全性——内耳局部给药耐受性良好,未见严重不良事件(现有数据);AAV相关免疫反应与长期风险需长期随访[5][8];(3)患者选择——基因检测确认OTOF双等位突变是前提;治疗窗口——儿童早期干预效果最佳,成人残存毛细胞者亦可受益[5][7][9];(4)评估体系——纯音测听/言语识别率/ABR等客观指标+家长报告的"生活质量"双轨评估[5][8]。十一、投资与市场空间耳聋基因治疗的市场逻辑:(1)患者基数——全球约4.3亿人患致残性听力损失,先天性耳聋约1/1000活产,遗传因素占~50%;OTOF突变占先天性耳聋~1-8%(欧美),中国等地区可能更高[5][8];(2)定价空间——"单次给药终身获益"+FDA首个获批(国家优先审评券)——孤儿药+创新疗法定价锚高;Regeneron/礼来定价策略将定调市场[5][6];(3)市场测算——OTOF单靶点全球可及患者(每年新发数千例)×高定价,峰值$5-15亿;扩展至GJB2(患者基数大10倍+)后,2030年耳聋基因治疗市场$20-50亿[5][8];(4)BD热度——Seamless($11.2亿)+Rznomics($13亿)+Akouos($4.87亿)显示MNC对耳聋基因治疗的技术溢价[1][2][7][10]。十二、风险与挑战:四重门耳聋基因治疗的四重门:(1)递送门——内耳局部给药的精准性、可重复性、规模化(手术操作依赖)是商业化瓶颈[8][9];(2)靶点门——OTOF已验证,但GJB2(最常见)的基因增补/编辑技术难度极高;显性突变的编辑策略复杂[8];(3)安全门——AAV免疫原性、重组酶的脱靶/致癌风险(长期)、双AAV的基因重组[2][8];(4)支付门——"单次高定价"vs内耳手术操作费用+基因检测费用,医保覆盖与患者可及性[5][6]。核心判断:OTOF已跑通"注册路径",赛道进入"靶点扩展+技术迭代"阶段——重组酶/RNA编辑决定下一轮竞争格局[2][5][10]。十三、给各方建议(三类读者全覆盖) 13.1 给患者与家庭 (1)OTOF突变耳聋已有FDA获批疗法,家族性耳聋建议尽早基因检测确认靶点[5][6];(2)关注人工耳蜗与基因治疗的选择:基因治疗"生物性恢复听力",人工耳蜗"电刺激替代"——二者可互补[8];(3)儿童早期干预窗口至关重要[7][9]。 13.2 给临床医生 (1)建立"听力筛查→基因检测→靶点分型→治疗转介"路径,OTOF突变患者可评估基因治疗资格[5][9];(2)关注Regeneron产品真实世界数据与AK-OTOF/中国IIT进展[7][9];(3)内耳手术给药的技术培训与多学科协作(耳科+遗传+听力)是转介基础[8][9]。 13.3 给biotech新药研发从业者 (1)技术选择——增补(成熟)/重组酶编辑(代际)/RNA编辑(灵活)按突变类型定位[2][5][10];(2)递送是关键壁垒——内耳局部给药装置、双AAV策略、LNP内耳递送是差异化机会[7][8][9];(3)靶点策略——GJB2(最大人群)是下一战场,但技术难度高;STRC/MYO15A等中频靶点可先行[8];(4)中国机会——中国耳聋人群基数大+多中心IIT经验,国产AAV/递送平台+基因编辑是中国公司切入方向[9]。十四、结语:"听见世界"的基因时代从2024年"11岁男孩30天复聪"的个案奇迹,到2026年FDA批准全球首个耳聋基因疗法,再到Seamless×礼来$11.2亿重组酶合作——耳聋基因治疗在两年内完成了"从个案到产业"的跨越[1][5][7]。礼来的三线布局(增补+编辑+RNA编辑)与Regeneron的率先获批,共同勾勒出这一赛道的技术版图[1][2][5][7][10]。耳聋基因治疗的终局,是"基因检测精准分型×内耳递送高效到达×多技术路线按突变适配"的三位一体——OTOF只是起点,GJB2等更大患者群体在等待下一个技术突破[2][5][8]。当重组酶的"基因组手术"成熟、当内耳递送标准化、当更多靶点被点亮,"听见世界"将从罕见个案变成常规医疗选择[2][5][7][8]。希望加入“全球GCT行业交流群”的读者，请加以下二维码联系工作人员参考文献 [1]BioSpace(2026.1):Lilly Leans Into Hearing Loss in $1.12B+ Gene Editing Pact with Seamless——交易架构/AK-OTOF 30天复聪/礼来听力组合 · BioSpace(行业媒体) [2]GEN(2026.1):Lilly, Seamless Ink Up-to-$1.12B Hearing Loss Collaboration——可编程重组酶技术(插入/交换/反转/切除DNA片段) · GEN(行业媒体) [3]PharmaExec(2026.1):Seamless Therapeutics Enters $1.12 Billion Global Research Collaboration with Eli Lilly——研究合作+独家许可,2个额外靶点选择权 · PharmaExec(行业媒体) [4]HearingTracker(2026):Eli Lilly Taps Seamless in $1.12BB Hearing Loss Gene Editing Collaboration——Seamless设计听力损失相关基因特异性重组酶 · HearingTracker(行业媒体) [5]FDA官方(2026.4):FDA Approves First-Ever Gene Therapy for Genetic Hearing Loss——全球首个耳聋基因疗法获批(OTOF,国家优先审评券) · FDA(官方) [6]CNN(2026.4.23):FDA approves first gene therapy for inherited deafness——20名儿童16名改善,5/12随访11个月听力恢复 · CNN(权威媒体) [7]Harvard Medical School(2026.4):Hearing Restoration From Gene Therapy for Inherited Deafness Lasts Years——疗效持续数年;AK-OTOF 11岁男孩30天复聪 · Harvard Medical School(官方) [8]PMC 12591109(Zeng FG等,2025):Treating Hearing Loss: From Cochlear Implantation to Gene Therapy——耳聋基因谱系/人工耳蜗对比/递送挑战综述 · PMC(综述) [9]Boston Children's Hospital(2025-2026):Gene Therapy for Hearing Loss——Miles案例(2025.5 OTOF注射)/OTOF双AAV策略/内耳递送 · Boston Children's Hospital(官方) [10]GCT前沿内部研究(2026.08):礼来×Rznomics RNA编辑合作($13亿,2025.5)/Akouos收购($4.87亿)/礼来听力三线布局 · GCT前沿内部研究 [11]Sensorion官方(2026):GJB2相关耳聋基因治疗推进——全球最常见耳聋基因(占遗传性30-50%) · Sensorion(公开资料) [12]UW Otolaryngology/公开报道(2026):Otarmeni(DB-OTO,Decibel/Regeneron)获FDA加速批准——首个遗传性耳聋基因疗法(OTOF) · UW Otolaryngology(公开报道) [13]Rinri Therapeutics(2026):细胞治疗修复/替换损伤听觉神经元——干细胞再生新路线 · Envoy Medical(行业媒体) [14]STAT News(2026.4.22):Gene therapy trial for deafness——OTOF基因治疗试验90%参与者报告显著改善 · STAT News(权威媒体) [15]中国多中心OTOF研究(公开报道):一针恢复耳聋患儿听力,改善持续长达2.5年 · 中国多中心研究(公开报道) [16]EMBO Molecular Medicine 2026:DFNA2小鼠模型长期听觉功能恢复——RNA碱基编辑治疗(KCNQ4显性突变)(DOI:10.1038/s44321-026-00433-5) · EMBO Mol Med(期刊) · DOI:10.1038/s44321-026-00433-5 [17]Hearing Health Foundation:How the Hearing Restoration Project Is Transforming Inner Ear Science——毛细胞再生与基因治疗互补 · Hearing Health Foundation(官方) [18]Regeneron官方IR(2026):Otarmeni(lunsotogene parvec-cwha)获FDA批准——首个且唯一OTOF体内基因疗法,美国免费提供 · Regeneron(官方) [19]NEJM 2026(Valayannopoulos V等):DB-OTO Gene Therapy for Inherited Deafness——12例中3例听力正常化,恢复自然声学听觉(引用64次,DOI:10.1056/NEJMoa2400521) · NEJM(顶刊) · DOI:10.1056/NEJMoa2400521 [20]PMC 12954134(2026):OTOF Gene Therapy: From Breakthroughs to Roadmaps——中国在OTOF临床试验中全球领先;遗传性耳聋基因治疗瓶颈 · PMC(评论) [21]ClinicalTrials.gov:NCT05821959——AAVAnc双耳给药剂量递增试验(OTOF,单次手术给药,两个剂量水平) · ClinicalTrials.gov

100 项与 AK-OTOF 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
耳聋牙釉质发育不全指甲缺损	临床3期	美国	Eli Lilly & Co.	2023-09-15
耳聋牙釉质发育不全指甲缺损	临床3期	加拿大	Eli Lilly & Co.	2023-09-15
耳聋牙釉质发育不全指甲缺损	临床3期	中国台湾	Eli Lilly & Co.	2023-09-15
耳聋牙釉质发育不全指甲缺损	临床3期	英国	Eli Lilly & Co.	2023-09-15
听觉神经病	临床阶段不明	美国	Eli Lilly & Co.	2022-07-14
听觉神经病	临床阶段不明	澳大利亚	Eli Lilly & Co.	2022-07-14
听觉神经病	临床阶段不明	德国	Eli Lilly & Co.	2022-07-14
听觉神经病	临床阶段不明	西班牙	Eli Lilly & Co.	2022-07-14
听觉神经病	临床阶段不明	中国台湾	Eli Lilly & Co.	2022-07-14
听觉神经病	临床阶段不明	土耳其	Eli Lilly & Co.	2022-07-14

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05821959 (AK-OTOF-101, NEWS) 人工标引	临床1/2期	听力损失 OTOF Mutation	1	AK-OTOF	壓膚顧窪膚餘襯鑰顧壓(簾夢製簾鑰獵襯築簾顧) = was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant, the first to receive gene therapy in the United States for a genetic form of hearing loss. 鏇繭鑰衊築醖壓願鑰範 (願繭夢蓋範窪醖淵艱醖 ) 更多	积极	2024-01-23