A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping

AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

开始日期2024-01-22

申办/合作机构

Avidity Biosciences, Inc.

NCT05670730 / Recruiting临床1/2期

A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping

AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.
Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.

开始日期2022-11-09

申办/合作机构

Avidity Biosciences, Inc.

100 项与 AOC-1044 相关的临床结果

登录后查看更多信息

100 项与 AOC-1044 相关的转化医学

登录后查看更多信息

100 项与 AOC-1044 相关的专利（医药）

登录后查看更多信息

项与 AOC-1044 相关的新闻（医药）

2024-05-09

·PRNewswire

Avidity Biosciences Reports First Quarter 2024 Financial Results and Recent Highlights

Initiation of global Phase 3 HARBOR™ trial for del-desiran (AOC 1001) in DM1 on track for this quarter Avidity to report FSHD data from FORTITUDE™ trial this quarter and DMD data from EXPLORE44™ trial in 2H24 Presented positive long-term del-desiran data from MARINA-OLE™ showing reversal of disease progression in people living with myotonic dystrophy type 1 across multiple endpoints; same key endpoints, with vHOT as primary, agreed for phase 3 HARBOR trial Cash position of $915 million at the end of Q124 following successful private placement SAN DIEGO, May 9, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the first quarter ended March 31, 2024, and highlighted recent corporate progress. "As we move forward with the planned initiation of our global Phase 3 HARBOR™ study for del-desiran this quarter, we continue to work diligently to improve people's lives by advancing our AOC platform," said Sarah Boyce, president and chief executive officer at Avidity. "In March, we announced new long-term del-desiran data from our MARINA-OLE study showing reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT), muscle strength and activities of daily living when compared to END-DM1 natural history data. Importantly, we also secured agreement with global regulators on the inclusion of these endpoints in the HARBOR study, including vHOT as the primary endpoint. We look forward to sharing preliminary data in approximately half of the study participants in our Phase 1/2 FORTITUDE™ trial in people living with FSHD this quarter and 5 mg/kg cohort data from our Phase 1/2 EXPLORE44™ trial of people living with DMD44 in the second half of this year." "Following an oversubscribed equity raise of $400 million, our cash position of $915 million at the end of the first quarter provides us with funding into late 2026," said Mike MacLean, chief financial officer and chief business officer at Avidity. "With our positive long-term data for del-desiran and two additional data readouts planned from our FSHD and DMD44 clinical programs this year, we continue to make significant progress in advancing our clinical programs." Recent Highlights Avidity announced that it received Breakthrough Therapy designation from the FDA for del-desiran for the treatment of DM1 The company announced it accelerated the global Phase 3 HARBORTM study initiation to Q2 2024 following agreement with multiple regulators on study design. The primary endpoint is video hand opening time (vHOT) and key secondary endpoints include muscle strength and activities of daily living Avidity introduced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran Presented positive del-desiran long-term 4 mg/kg data from MARINA-OLE™ study in March 2024. Data showed: Reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data Consistent and durable improvements in the following: Myotonia (video hand opening time, or vHOT) Multiple measures of strength: Hand grip Quantitative Muscle Testing (QMT) total score which includes hand grip; elbow extension and elbow flexion; knee extension and knee flexion, and ankle dorsiflexion DM1-Activ, a patient reported outcome (PRO) that measures activities of daily living (e.g., taking a shower, visiting family or friends, and walking up stairs) With over 265 infusions totaling 61.1 patient-years of exposure, del-desiran continues to demonstrate favorable safety and tolerability The FDA granted AOC 1044 with Rare Pediatric Disease Designation in February 2024 for DMD44 Upcoming Milestones Upcoming anticipated milestones include: In the second quarter of 2024, initiation of global Phase 3 HARBOR™ trial of del-desiran for adults living with DM1 In the second quarter of 2024, share preliminary data in approximately half of participants in the Phase 1/2 FORTITUDE™ trial of AOC 1020 in people living with FSHD In the second half of 2024, share 5 mg/kg cohort data from the Phase 1/2 EXPLORE44™ trial of AOC 1044 in people living with DMD44 First Quarter 2024 Financial Results Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $915.9 million as of March 31, 2024, which reflects a gross $400 million raise from a private placement. Collaboration Revenue: Collaboration revenue relates to Avidity's research collaboration and license partnerships. Collaboration revenues of $3.5 million in the first quarter of 2024 related to partnerships with Bristol Myers Squibb and Eli Lilly and Company as compared to $2.2 million related to a partnership with Eli Lilly and Company in the first quarter of 2023. There was no revenue related to the partnership with Bristol Myers Squibb in 2023. Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $66.8 million for the first quarter of 2024 compared with $47.8 million for the first quarter of 2023. The increases were primarily driven by the advancement of del-desiran, AOC 1020 and AOC 1044, as well as internal and external costs related to the expansion of the company's overall research capabilities. Gener al and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $13.9 million for the first quarter of 2024 compared with $12.1 million for the first quarter of 2023. The increases were primarily due to higher personnel costs to support the company's expanded operations. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is focused on revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: plans to initiate the global Phase 3 HARBOR™ trial of del-desiran for people living with DM1; the anticipated release of data from the FORTITUDE™ and EXPLORE44™ trials, including the timing thereof and cohort dosage data; the characterization of data associated with del-desiran from the MARINA-OLE™ study; plans for the progression of research and development initiatives, including in cardiology and immunology; Avidity's financial position, cash balance and ability to fund its operations; Avidity's growth and related needs; and the potential of AOCs to target a range of different cells and tissues beyond the liver, and to treat cardiac and immunological diseases. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: Avidity may not be able to resolve the partial clinical hold related to the serious adverse event which occurred in the Phase 1/2 MARINA® trial; additional data related to Avidity's current clinical programs that continues to become available may be inconsistent with the data produced as of the respective data cutoff dates, further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the date hereof, and such data may not meet Avidity's expectations; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date regarding the proposed design and protocol for the Phase 3 HARBOR trial; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; the success of its preclinical studies and clinical trials for the company's product candidates; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; Avidity may not realize the expected benefits of its collaborations; regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Geoffrey Grande, CFA (619) 837-5014 [email protected] Media Contact: Navjot Rai (619) 837-5016 [email protected] SOURCE Avidity Biosciences, Inc.

临床3期临床结果突破性疗法财报

2024-05-06

·BioSpace

5 DMD Data Readouts to Watch in 2024

Pictured: A boy in a wheelchair over a muscle texture background/Taylor Tieden for BioSpace The March approval of ITF Therapeutics’ Duvyzat marked the third new FDA-approved treatment in 10 months for Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness and degeneration. In June 2023, the regulator greenlit Sarepta’s Elevidys as the first-ever gene therapy for DMD. That approval was followed by Santhera Pharmaceuticals’ novel corticosteroid Agamree in October. And the momentum isn’t slowing down. The DMD pipeline is flush with investigational treatments making their way through mid- and late-stage clinical trials, with several companies expecting data readouts in 2024. “Where we are this year has been built upon activity, investments and trials over the past two, three and four years,” Michael Kelly, chief scientific advisor at CureDuchenne, told BioSpace in an email. “I think this could end up being the year where we become really informed on some of the long-term efficacy and safety data from ongoing trials and approved drugs.” Kelly said he is particularly interested in the clinical readouts from Pfizer, REGENXBIO and Solid Biosciences’ gene therapy programs and what he called the next-generation of exon-skipping programs from Avidity Biosciences, Dyne Therapeutics, PepGen and Entrada Therapeutics. “There’s a lot of unmet needs, unfortunately,” Jeff Chamberlain, president of the American Society for Gene and Cell Therapy and the McCaw Chair in Muscular Dystrophy at the University of Washington School of Medicine, recently told BioSpace, but “we’re certainly moving in the right direction.” BioSpace takes a closer look at five investigational therapies for which results are expected this year. Pfizer’s fordadistrogene movaparvovec Gene therapy Pfizer is anticipating data from a Phase III trial of fordadistrogene movaparvovec in the second half of 2024, according to CureDuchenne. A mini-dystrophin gene therapy, fordadistrogene movaparvovec is being studied in this trial for use in ambulatory DMD patients. DMD is caused by the absence of the dystrophin protein, which helps keep muscle cells intact. Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus serotype 9 (AAV9) capsid carrying a shortened version of this gene, according to Pfizer. “The whole purpose of gene therapy in Duchenne, particularly micro-dystrophin gene therapy, is to transform the patient’s phenotype from Duchenne to the less severe Becker muscular dystrophy,” Kelly said. “It’s not a cure because we’re not putting in full-length dystrophin, so the question is, which type of Becker muscular dystrophy should we expect?” Pfizer’s path with fordadistrogene movaparvovec has not been without its challenges. In 2021, the company paused screening and dosing in a Phase Ib study of the gene therapy after the death of a patient in the non-ambulatory cohort of the trial. By April 2022, Pfizer was back on track, opening the first U.S. sites in the Phase III trial. The current gene therapies in development for DMD are “partially effective,” Chamberlain said, but “far from a cure.” Dyne Therapeutics’ DYNE-251 Exon-skipping biologic Also during the second half of this year, Waltham, Mass.–based Dyne Therapeutics is expecting data from the higher dose cohort of the Phase I/II DELIVER trial of DYNE-251. DYNE-251 comprises a phosphorodiamidate morpholino oligomer conjugated to a fragment antibody that binds to the transferrin receptor 1, which is highly expressed in muscle. It was designed to promote skipping of exon 51 to create a truncated, functional dystrophin protein, according to Dyne. It is being developed for patients with mutations in exon 51. In January, Dyne reported six-month data from six male patients in the 5 mg/kg cohort of the trial. Administration of DYNE-251 once every four weeks elicited levels of dystrophin expression, exon skipping and dystrophin positive fibers that exceeded levels seen in a clinical trial for eteplirsen, the current weekly standard of care for exon 51 DMD patients. The candidate also demonstrated a favorable safety profile, which a Dyne representative said positions the company to focus on optimizing dose and dose regimen in the DELIVER trial “with the goal of initiating registrational cohorts by the end of 2024.” Edgewise Therapeutics’ EDG-5506 Small molecule Edgewise Therapeutics plans to report three-month controlled dose-ranging data from the Phase II LYNX trial of its DMD candidate EDG-5506 this quarter. EDG-5506 is designed to reduce injurious mechanical stress caused by the absence of functional dystrophin in order to enable a normal, healthy range of muscle contraction. The candidate was granted the FDA’s Fast Track designation in DMD in February. The Boulder, Colorado–based company is also running the Phase II FOX trial in children and adolescents with DMD who have been treated with gene therapy. Regardless of the modality, Kelly said, a “combination of therapies targeting dystrophin restoration . . . with other approaches . . . is going to be the preferred route of treatment for Duchenne’s patients.” Capricor Therapeutics’ CAP-1002 Cell therapy On that note, Kelly said he is carefully watching Capricor Therapeutics’ CAP-1002. The cell therapy is currently in a pivotal Phase III study, from which the company expects topline data by the end of this year. In addition to evaluating CAP 1002 as a standalone therapy, Capricor is investigating the drug in patients who have previously been treated with gene therapy, Kelly said. CAP-1002 is composed of cardiosphere-derived cells (CDCs) derived from healthy human hearts. The CDCs secrete exosomes containing bioactive cargo, including microRNAs, that alter gene expression in macrophages and other target cells, dialing down generalized inflammation and stimulating tissue regeneration, Capricor explains on its website. The company additionally notes that CAP-1002 may be able to “work synergistically with emerging disease-modifying therapies.” Avidity Biosciences’ AOC 1044 Exon skipping biologic San Diego–based Avidity Biosciences is developing AOC 1044 on its proprietary antibody oligonucleotide conjugates (AOC) platform. The therapy is intended for patients with exon 44 mutations, a version of DMD for which there are currently no disease-modifying treatments, according to Avidity. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to the skeletal muscle and heart tissue to specifically skip exon 44 of dystrophin mRNA in order to enable dystrophin production. In December 2023, Avidity reported positive data from the Phase I/II EXPLORE44 trial of AOC 1044 in healthy volunteers showing “statistically significant exon 44 skipping compared to placebo” of up to 1.5% after a single dose. The therapy was well-tolerated. The company plans to provide a first look at data from DMD patients during the second half of 2024. Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.

临床3期临床结果临床2期快速通道基因疗法

2024-04-30

·药智网

ADC药物的二次进化

过去十年，ADC迎来了发展迅猛的黄金时代，“万物皆可偶联”也被业内尝试的不亦乐乎。同时，基因治疗、基因药物，也正在高光产出，如国谈中的天价药-诺西那生钠。基于此，很多研究聚焦到“ADC+小核酸”的AOC，以期综合二者的优点，实现二次进化。01AOC是怎样的偶联？AOC，是抗体-寡核苷酸偶联物的简称，结构与ADC相似，主要由三部分构成：即“载体（抗体）、连接子、小核酸”。AOC通过连接子和偶联技术将抗体和小核酸有效结合在一起，针对抗体靶向的靶标细胞和寡核苷酸特异性沉默的目标基因实现联合靶向治疗，同时解决小核酸药物现有的靶向和递送难题。图1 AOC药物结构特征举例图片来源：https://www.dyne-tx.com/our-forcetm-platform/有文献总结，成功获得目标AOC药物关键在于以下几方面：有效载荷的效力、连接子的稳定性、偶联抗体和有效载荷的偶联技术。小核酸类药物当前已有数十款品种获批上市，虽然目前已通过结构修饰、递送载体等方式使小核酸药物（尤其是ASO和siRNA）更具成药性，但更有效的修饰、递送仍是产品开发的重点方向，这也是小核酸药物适应症扩展和临床给药等问题的掣肘所在。AOC药物的开发，就是希望解决小核酸现有的、先天的血清稳定性差、膜通透性低、缺乏组织选择性等问题，并结合抗体更长的半衰期以及良好的靶向递送特性，最终组合成为AOC所特有的高精度选择性以及抗体的靶向递送性，达成共赢。图2 AOC药物作用机制特征举例图片来源：Nucleic Acids Research,2023 1 https://doi.org/10.1093/nar/gkad41502AOC各结构组成特点类似于ADC，AOC药物的开发也是将抗体、连接子、小核酸逐个研究，以期形成最优的组合形式。基于ADC广泛的理论和实践基础，这里重点对小核酸、连接子偶联技术特点进行描述。➣小核酸特点1）决定药效的关键部分。2）可以是单链或双链，如ASO、siRNA。3）核苷酸可经修饰（如2'脱氧，2'-氟，2'-O-甲基，2'-O-甲氧基乙基等），以抗代谢、减毒、核内体逃逸。PS：小核酸现有技术相对丰富，已积累大量研究数据，理论和实践相对较丰富。图3 寡核苷酸治疗药物的化学修饰图片来源：doi.org/10.1016/j.chembiol.2023.09.005➣连接子特点1）关系到稳定性、药代、药效、治疗窗口等方面。2）通常包括至少一个共价键，可以是单键比如二硫键或二硫桥。3）连接子与药物连接的方式：主要分为可裂解和不可裂解两种形式。可裂解连接子主要为敏感性的化学键，可以根据身体内环境的特异性（如谷胱甘肽、酸碱性等）或酶浓度不同，促进连接子与药物裂解，主要通过二硫键、多肽的形式连接。不可裂解连接子没有可触发裂解的内置化学键，需要在细胞中将抗体通过蛋白质水解机制变成氨基酸，进而释放带有连接子和氨基酸片段的细胞毒性药物，主要通过硫醚的形式连接。4）连接子与抗体偶联：如ADC，通过选择性偶联将抗体与连接子结合，合成均质的ADC，也是连接子研究的重要方向，主要包括化学方法和酶法对抗体氨基酸和糖类部分修饰。图4 linker相关的一些化学触发总结图片来源：Acta Pharmaceutica Sinica B 2021;11（12）:3889e390703全球布局AOC产品的公司目前，全球AOC药物研发主要分布于几家生物技术公司，且主要集中于国外，这里介绍3家公司Avidity Biosciences、Dyne Therapeutics、Tallac Therapeutics。➣Avidity Biosciences据官网介绍，AOC技术是Avidity Biosciences的主要研究方向，旨在综合单抗的特异性、小核酸疗法的精准性，以攻克当前RNA疗法尚无法踏足的适应症领域。目前，Avidity Biosciences利用其技术平台已可以将RNA靶向肌肉，适应症主要为强直性肌营养不良症1型（DM1，尚无批准的治疗方法）、杜氏肌营养不良症（DMD）和面肩肱肌营养不良症（FSHD）；另，Avidity Biosciences正在积极拓展适应症领域及方向，如心脏病、免疫。图5 Avidity Biosciences部分管线图片来源：https://www.aviditybiosciences.com/pipeline/pipeline-overview/Avidity Biosciences临床在研的3个项目即针对上述的3种肌肉罕见疾病。如del-desiran（AOC1001）是Avidity Biosciences公司利用AOC平台开发的重点产品，其由3部分组成：靶向转铁蛋白受体1（TfR1）的全长单抗、连接子、靶向DMPK mRNA的siRNA，适应症为强直性肌营养不良I型（DM1）。目前，1/2期临床试验MARINA已完成，正在积极推进高阶临床，即将在成人DM1患者中进行HARBOR试验。另外两款产品AOC1044和AOC1020也在临床积极推进中。图6 AOC1001相关作用特点及治疗策略图片来源：https://www.aviditybiosciences.com/pipeline/dm1/➣Dyne Therapeutics据官网介绍，Dyne Therapeutics专注于基因驱动的肌肉领域疾病治疗药物的开发，公司开发的FORCE™平台，旨在克服肌肉组织递送的难题，推进现代寡核苷酸治疗。Dyne Therapeutics开发的品种适应症也是聚焦于DM1、DMD和FSHD。图7 Dyne Therapeutics在研管线图片来源：https://www.dyne-tx.com/pipeline/Dyne Therapeutics临床在研管线中，重点适应症同样为DM1和DMD，2款产品分别为DYNE-101（DMPK）和DYNE-251（Exon-51），当前均处于临床1/2期阶段。以DYNE-101为例，DYNE-101现已进入临床开发，处于全球1/2期ACHIEVE临床试验中，用于DM1患者的治疗。DYNE-101由一个反义寡核苷酸（ASO）与一个片段抗体（Fab）结合组成，该片段抗体结合在肌肉上高表达的转铁蛋白受体1（TfR1），并通过降低细胞核中突变DMPK RNA的水平，阻止或逆转疾病，解决DM1的遗传物质基础。DYNE-101已被EMA和FDA授予孤儿药称号，用于DM1的临床开发。图8 DYNE-101的药物特征及疾病临床位置图片来源：Drug Discovery Today d Volume 28,Number 3 d March 2023 REVIEWS➣Tallac Therapeutics据官网介绍，Tallac Therapeutics的重点技术之一同样为将小核酸作为有效载荷的AOC，药物方向为toll样受体激动剂/Toll-like Receptor Agonist Antibody Conjugate（TRAAC）。从靶点特征即可看出，Tallac Therapeutics的研究领域不同于Avidity Biosciences和Dyne Therapeutics，而是放到了小核酸药物很难触碰到的肿瘤领域，这在一定程度上是小核酸药物应用的很重要的突破。图9 Tallac Therapeutics药物管线图片来源：https://www.tallactherapeutics.com/science/从上述管线来看，TAC-001无疑为Tallac Therapeutics的重点产品，2024年AACR年会报道该产品的部分内容：研究结果表明，TAC-001与癌症疫苗联合使用，促进了强效和持久的疫苗特异性IgG滴度，恢复了疫苗反应。此外，TAC-001还可以增强疫苗特异性T细胞活化和细胞毒活性。当前，正在开展1/2期临床研究（NCT05399654）。图10 TRAAC技术药物作用特点图片来源：https://www.tallactherapeutics.com/science/04结语AOC技术，无论是在小核酸领域，还是在偶联药物技术领域，都是一项前沿技术。敢为人先的药企，往往更受投资者的青睐。目前，AOC技术仍主要集中于国外，国内企业涉猎不多（但已有企业布局及产出阶段性成果），在这一领域的市场机会相对较多。但，AOC的技术门槛和挑战还是显而易见的，从设计到开发，需要全链条的对各个环节进行探索，未知性很高。不过也正因如此，凭借AOC的设计理念和技术门槛，该领域的未来非常值得期待。参考来源：1.Pharmacy Information药物资讯,2024,13（2）,82-912.https://www.dyne-tx.com/our-forcetm-platform/3.Nucleic Acids Research,2023 1 https://doi.org/10.1093/nar/gkad4154.Cell Chemical Biology(2023),https://doi.org/10.1016/j.chembiol.2023.09.0055.Acta Pharmaceutica Sinica B 2021;11（12）:3889e3907.doi.org/10.1016/j.apsb.2021.03.0426.https://www.aviditybiosciences.com7.https://www.dyne-tx.com/pipeline/8.https://www.tallactherapeutics.com/science/9.Drug Discovery Today d Volume 28,Number 3 d March 2023 REVIEWS声明：本内容仅用作医药行业信息传播，为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 金银花转载开白 | 马老师 18996384680（同微信）商务合作 | 王存星 19922864877（同微信）阅读原文，是昨天最受欢迎的文章哦

抗体药物偶联物寡核苷酸上市批准siRNA核酸药物

100 项与 AOC-1044 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
外显子44跳跃突变的杜氏肌营养不良症	临床2期	美国	Avidity Biosciences, Inc.	2024-01-22

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05670730 (EXPLORE44, PRNewswire) 人工标引	临床1/2期	杜氏肌营养不良症	44	AOC 1044	(糧願鹹網廠蓋醖蓋鬱糧) = All treatment-emergent adverse events in participants dosed with AOC 1044 were mild to moderate. There were no symptomatic hemoglobin changes, no hypomagnesemia and no renal events. 蓋膚鹹獵膚積簾膚範製 (糧範鹽醖積醖齋餘構膚 )	积极	2023-12-13