Amaurosis Congenita of Leber, Type 1

DURHAM, N.C., Oct. 10, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting taking place October 18-21, 2024, in Chicago. ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. Presentation details are as follows: Session: First-time Results of Clinical TrialsDate and Time: Friday, October 18, 2024, 4:54 - 5:33 p.m. CDTPresentation Title: Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS)Presentation Time: 5:08 p.m. CDTPresenter: Christine Nichols Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics The safety and tolerability of ATSN-201 is being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. About X-linked Retinoschisis (XLRS)XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. About AAV.SPRAAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection bleb margins to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to those original margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. About Atsena TherapeuticsAtsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood and leads to blindness later in life. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/. Media Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com Business Contact:info@atsenatx.com

DURHAM, N.C., Sept. 24, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it will present at Chardan’s 8th Annual Genetic Medicines Conference taking place September 30 - October 1, 2024, in New York City. Chief Executive Officer Patrick Ritschel will deliver a company presentation on Tuesday, October 1, 2024, at 11:30 a.m. ET. Atsena Chief Medical Officer, Kenji Fujita, MD, and Mr. Ritschel will also participate in one-on-one meetings with potential investors during the conference. About Atsena TherapeuticsAtsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/. Media Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com Business Contact:info@atsenatx.com

行业动态速览 会议推荐 01 相约沈阳，派真生物诚邀您参加国际基因组学大会第七届眼科大会(ICGEYE) 国际基因组学大会第七届眼科大会(ICGEYE)”将于 2024年9月20日-9月21日在沈阳召开。派真生物产品总监钟德清有幸受邀在基因治疗与基因大数据论坛下就《AAV基因治疗产品CMC开发关键考量-载体与处方设计》主题进行演讲，分享心得与经验，同时派真生物在展位HO4特设展台，安排多名专业技术人员在现场与您面对面沟通，答疑解惑；此外，我们还准备了精美礼品诚邀您亲临展位现场指导交流。 推荐阅读：会议邀请 | 相约沈阳，派真生物诚邀您参加国际基因组学大会第七届眼科大会(ICGEYE) 02 派真生物诚邀您共赴苏州-中国神经科学学会第十七届全国学术会议 中国神经科学学会第十七届全国学术会议将于2024年9月26日－29日在苏州国际博览中心A馆召开，派真生物在B34特设展台，参展期间我们将为您带来多样的互动环节和精美的礼品，参与即可领取礼品，更有我们的技术专家与您面对面交流洽谈，答疑解惑！期待与您相约，不见不散。 推荐阅读：会议邀请 | 派真生物诚邀您共赴苏州-中国神经科学学会第十七届全国学术会议 热点聚焦 01 体内编辑新赛道：mRNA介导的体内编辑CAR-巨噬细胞用于治疗肝癌的免疫治疗的研发结果发布 在2024年9月6号于北京召开的第八届免疫基因及细胞治疗大会上及2024年7月于德国汉堡举办的国际干细胞研究协会（ISSCR） 年会上，赛元生物创始人团队的研究人员们做了mRNA介导的体内编辑的CAR-巨噬细胞用于肝癌免疫治疗的报告。 推荐阅读：体内编辑新赛道：mRNA介导的体内编辑CAR-巨噬细胞用于治疗肝癌的免疫治疗的研发结果发布 02 使用双AAV9载体，首款DMD碱基编辑药物完成首例患者给药，开启DMD基因编辑治疗新篇章 近日，新芽基因宣布其碱基编辑药物GEN6050X注射液在一项研究者发起的临床试验（IIT）中，已完成首例DMD患者给药。 推荐阅读：使用双AAV9载体，首款DMD碱基编辑药物完成首例患者给药，开启DMD基因编辑治疗新篇章 03  瑞博生物自主研发治疗肾病的核酸药物RBD7007首个临床试验申请获得欧盟批准 近日，苏州瑞博生物技术股份有限公司（简称“瑞博生物”）自主研发的、靶向C5的siRNA药物RBD7007获欧盟临床试验许可，将在瑞典启动首次人体临床试验。  推荐阅读：瑞博生物自主研发治疗肾病的核酸药物RBD7007首个临床试验申请获得欧盟批准 04 中检院最新 | 改良mRNA疫苗质控dsRNA检测方法 2024年8月21日，中检院团队在Protein & Cell(IF=13.6)上发表题为“An improved method for the detection of double-stranded RNA suitable for quality control of mRNA vaccines”的Research文章，展示了一种关于dsRNA检测的改进方法。 推荐阅读：中检院最新 | 改良mRNA疫苗质控dsRNA检测方法 创新突破 01 柳叶刀：AAV基因治疗，将遗传性失明患者视力提升超百倍 一项最新临床研究表明，AAV基因治疗对GUCY2D基因突变引起的Leber先天性黑蒙症1型（LCA1）展示了良好的治疗前景。LCA1是一种罕见的遗传性视网膜疾病，常导致儿童早期失明。该临床试验结果显示，接受基于AAV5的基因疗法（ATSN-101）治疗后，一些患者的视力提高了100倍，而一些接受最大剂量治疗的患者的视力甚至提高了10000倍。 Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study 为题，于2024年9月7日发表在了国际顶尖医学期刊《柳叶刀》（The Lancet）上。 推荐阅读：柳叶刀：AAV基因治疗，将遗传性失明患者视力提升超百倍 02 Nature子刊：曾春雨团队证实α-酮戊二酸可促进心肌梗死后心脏再生 近日，陆军军医大学陆军特色医学中心（大坪医院）曾春雨团队在 Nature 子刊 Nature Cardiovascular Research 上发表了题为：α-Ketoglutarate promotes cardiomyocyte proliferation and heart regeneration after myocardial infarction 的研究论文。 该研究表明，α-酮戊二酸（α-KG）促进心肌梗死（MI）后心肌细胞（CM）的增殖和心脏再生。 推荐阅读：Nature子刊：曾春雨团队证实α-酮戊二酸可促进心肌梗死后心脏再生 03 Moderna公司最新论文：mRNA疗法治疗T细胞介导的自身免疫病 2024年9月6日，mRNA疫苗巨头Moderna公司在 Cell 子刊 Cell Reports Medicine 上发表了题为：mRNA-delivery of IDO1 suppresses T cell-mediated autoimmunity 的研究论文。 该研究表明，使用脂质纳米颗粒（LNP）递送mRNA以表达IDO1，能够抑制多种由T细胞介导的自身免疫疾病。 推荐阅读：Moderna公司最新论文：mRNA疗法治疗T细胞介导的自身免疫病 04 sgRNA设计方法及靶基因细胞敲除的应用 基于CRISPR经典的基因敲除系统包含sgRNA序列和Cas蛋白，sgRNA序列长度一般为20个核苷酸。常用于DNA双链切割活性的Cas蛋白包括Cas9和Cas12。由于不同Cas蛋白识别的PAM序列，切割效率及特异性不同，本文将以Cas9蛋白为例详细介绍sgRNA的设计，质粒构建及细胞敲除过程。 推荐阅读： sgRNA设计方法及靶基因细胞敲除的应用 资本速递 01 新锐！2600万美元种子轮融资，开发AAV基因疗法治疗肿瘤 9月12日，加利福尼亚州圣地亚哥，Vironexis Biotherapeutics专注于通过开创AAV递送的T细胞免疫疗法来改变癌症治疗的未来，宣布完成2600万美元种子轮融资，推出了其TransJoin™AAV基因治疗平台和一系列用于血液瘤、实体瘤转移预防和癌症疫苗的十多种候选产品。 推荐阅读：新锐！2600万美元种子轮融资，开发AAV基因疗法治疗肿瘤 02 贝斯生物完成A2轮融资，推进基于基因编辑技术的细胞与基因疗法 近日，贝斯生物宣布完成A2轮融资，融资金额达数千万人民币。 推荐阅读：贝斯生物完成A2轮融资，推进基于基因编辑技术的细胞与基因疗法 参考资料： https://mp.weixin.qq.com/s/2Kannds8cujPY1ytxT4FFg https://mp.weixin.qq.com/s/cYG5GRQ7YKjx0LJ8w65rKA https://mp.weixin.qq.com/s/cI2I6PqXUtoEugZdpQr40w https://mp.weixin.qq.com/s/mjLkPe3e-s06mEPUUanYfw https://mp.weixin.qq.com/s/RpTxD5sD7I-J_6govS7DQA https://mp.weixin.qq.com/s/cgH6tdZK98rBtPg-tHUOzQ https://mp.weixin.qq.com/s/KJjvi-b4_qr_LFsimYW50A https://mp.weixin.qq.com/s/yTTTwJ8dPtQXSaGEfz5MWQ https://mp.weixin.qq.com/s/0WxWBOtYmt9GmvB8-CATrA https://mp.weixin.qq.com/s/N18ZJlvKn7syHDlYgA9rqw https://mp.weixin.qq.com/s/maM-ap696FK6MeA6iDDW9Q https://mp.weixin.qq.com/s/cWRxc0btbhY8_z7TECSaJg 关于派真生物