针对DMD疾病根源的新治疗模式
偶联技术提高寡核苷酸疗法递送效率和表达水平
参考资料:
[1] Roberts et al, (2023). Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery. https://doi.org/10.1038/s41573-023-00775-6
[2] MDA Kicks Off Muscular Dystrophy Awareness Month in September with ‘30 Days of Strength’ Campaign with Hundreds of Events to Advance Research and Care for the Neuromuscular Disease Community. Retrieved September 19, 2024, from https://www.mda.org/press-releases/2024/mda-kicks-off-muscular-dystrophy-awareness-month-in-september-with-30-days-of-strength-campaign-with-hundreds-of-events-to-advance-research-and-care-for-the-neuromuscular-disease-community
[3] RNA Platform Leveraging RNA technology to treat rare disease. Retrieved September 19, 2024, from https://www.sarepta.com/science/rna-platform
[4] Sarepta Therapeutics Corporate Presentation 2024. Retrieved September 19, 2024, from https://investorrelations.sarepta.com/static-files/54793bd6-09b6-4c68-8281-39e586071e88
[5] Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020 Oct;19(10):673-694. doi: 10.1038/s41573-020-0075-7. Epub 2020 Aug 11. PMID: 32782413; PMCID: PMC7419031.
[6] Avidity Biosciences Announces Positive AOC 1044 Data Demonstrated Significant Increase of 25% in Dystrophin Production and Reduction of Creatine Kinase Levels to Near Normal in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping in the Phase 1/2 EXPLORE44™ Trial. Retrieved September 22, 2024 from https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-aoc-1044-data-demonstrated-significant-increase-of-25-in-dystrophin-production-and-reduction-of-creatine-kinase-levels-to-near-normal-in-people-living-with-duchenne-muscular-dystrophy-amenab-302218647.html
[7] Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51. Retrieved September 22, 2024 from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-positive-data-part-b-momentum
[8] Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1. Retrieved September 22, 2024 from https://investors.pepgen.com/static-files/ff601ea2-0a29-435f-82b1-c885924cfb94
[9] Watanabe N, Tone Y, Nagata T, Masuda S, Saito T, Motohashi N, Takagaki K, Aoki Y, Takeda S. Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide. Mol Ther Nucleic Acids. 2023 Sep 20;34:102034. doi: 10.1016/j.omtn.2023.102034. PMID: 37854955; PMCID: PMC10579524.
[10] Myotonic Dystrophy Type 1 Program. Retrieved September 22, 2024 from https://www.entradatx.com/dm1
[11] Tsoumpra MK, Fukumoto S, Matsumoto T, Takeda S, Wood MJA, Aoki Y. Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases. EBioMedicine. 2019 Jul;45:630-645. doi: 10.1016/j.ebiom.2019.06.036. Epub 2019 Jun 27. PMID: 31257147; PMCID: PMC6642283.
免责声明:药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的,文中观点不代表药明康德立场,亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导,请前往正规医院就诊。
版权说明:本文来自药明康德内容团队,欢迎个人转发至朋友圈,谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”,获取转载须知。
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针对DMD疾病根源的新治疗模式
偶联技术提高寡核苷酸疗法递送效率和表达水平
参考资料:
[1] Roberts et al, (2023). Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery. https://doi.org/10.1038/s41573-023-00775-6
[2] MDA Kicks Off Muscular Dystrophy Awareness Month in September with ‘30 Days of Strength’ Campaign with Hundreds of Events to Advance Research and Care for the Neuromuscular Disease Community. Retrieved September 19, 2024, from https://www.mda.org/press-releases/2024/mda-kicks-off-muscular-dystrophy-awareness-month-in-september-with-30-days-of-strength-campaign-with-hundreds-of-events-to-advance-research-and-care-for-the-neuromuscular-disease-community
[3] RNA Platform Leveraging RNA technology to treat rare disease. Retrieved September 19, 2024, from https://www.sarepta.com/science/rna-platform
[4] Sarepta Therapeutics Corporate Presentation 2024. Retrieved September 19, 2024, from https://investorrelations.sarepta.com/static-files/54793bd6-09b6-4c68-8281-39e586071e88
[5] Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020 Oct;19(10):673-694. doi: 10.1038/s41573-020-0075-7. Epub 2020 Aug 11. PMID: 32782413; PMCID: PMC7419031.
[6] Avidity Biosciences Announces Positive AOC 1044 Data Demonstrated Significant Increase of 25% in Dystrophin Production and Reduction of Creatine Kinase Levels to Near Normal in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping in the Phase 1/2 EXPLORE44™ Trial. Retrieved September 22, 2024 from https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-aoc-1044-data-demonstrated-significant-increase-of-25-in-dystrophin-production-and-reduction-of-creatine-kinase-levels-to-near-normal-in-people-living-with-duchenne-muscular-dystrophy-amenab-302218647.html
[7] Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51. Retrieved September 22, 2024 from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-positive-data-part-b-momentum
[8] Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1. Retrieved September 22, 2024 from https://investors.pepgen.com/static-files/ff601ea2-0a29-435f-82b1-c885924cfb94
[9] Watanabe N, Tone Y, Nagata T, Masuda S, Saito T, Motohashi N, Takagaki K, Aoki Y, Takeda S. Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide. Mol Ther Nucleic Acids. 2023 Sep 20;34:102034. doi: 10.1016/j.omtn.2023.102034. PMID: 37854955; PMCID: PMC10579524.
[10] Myotonic Dystrophy Type 1 Program. Retrieved September 22, 2024 from https://www.entradatx.com/dm1
[11] Tsoumpra MK, Fukumoto S, Matsumoto T, Takeda S, Wood MJA, Aoki Y. Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases. EBioMedicine. 2019 Jul;45:630-645. doi: 10.1016/j.ebiom.2019.06.036. Epub 2019 Jun 27. PMID: 31257147; PMCID: PMC6642283.
免责声明:药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的,文中观点不代表药明康德立场,亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导,请前往正规医院就诊。
版权说明:本文来自药明康德内容团队,欢迎个人转发至朋友圈,谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”,获取转载须知。
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