GUCY2D

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investors Proceeds to support advancement of ATSN-201 through potential approval and launch as well as preclinical programs to treat inherited retinal diseases Norbert Riedel, PhD, will join Atsena’s Board of Directors DURHAM, N.C., April 02, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics (“Atsena” or “the Company”), a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the successful close of an oversubscribed $150 million Series C financing. The financing was led by Bain Capital’s Life Sciences team, with participation from an additional new investor, Wellington Management. All the Company’s existing investors also participated in the round, including Lightstone Ventures, Sofinnova Investments, Abingworth, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners, and the Manning Family Foundation. Proceeds from the financing will be used to advance Atsena’s lead program, ATSN-201, for the treatment of X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. The proceeds will also support Atsena’s preclinical pipeline of first-in-class therapies and expand the use of Atsena’s novel spreading AAV.SPR capsid. “Closing our Series C marks a pivotal moment for Atsena as we advance our transformative ocular gene therapies and fuel our next phase of growth, innovation, and clinical progress,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It follows a productive 12 months of key achievements including securing a partner to advance ATSN-101 to a global pivotal trial for Leber Congenital Amaurosis type 1 (LCA1) and initiating Part B of the ATSN-201 LIGHTHOUSE study for XLRS. We’re grateful for the support of our investors and partners who share our vision for the future of leveraging genetic medicine to reverse or prevent blindness.” To date, Atsena’s clinical portfolio has received multiple designations by the U.S. Food and Drug Administration (FDA). ATSN-101, for the treatment of LCA1, has received Rare Pediatric Disease designation, Orphan Drug Designation, and Regenerative Medicine Advanced Therapy designation. ATSN-201 has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug Designations. Updated data from the ongoing LIGHTHOUSE Phase I/II clinical trial evaluating ATSN-201 is anticipated later this year. “We believe Atsena has a unique opportunity to deliver meaningful impact for patients with inherited retinal diseases on the basis of novel science and impressive clinical data generated to date,” said Amir Zamani, a Partner at Bain Capital. “We look forward to supporting Patrick and his strong team as they look to unlock the next phase of Atsena’s growth and innovation while thoughtfully advancing potentially groundbreaking therapies toward patients in need.” In conjunction with the financing, Norbert Riedel, PhD, a seasoned scientist and biopharmaceutical executive, will join Atsena’s Board of Directors. Wedbush & Co. served as exclusive placement agent to Atsena for the Series C financing, Cooley LLP acted as its legal advisor. About Atsena TherapeuticsAtsena Therapeutics (“Atsena”) 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 that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi-org.libproxy1.nus.edu.sg/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. 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:Gina Mangiaracina6 Degrees(917) 797-7904gmangiaracina@6degreespr.com Business Contact:info@atsenatx.com

▎药明康德内容团队编辑 近期，全球细胞和基因疗法（CGT）领域迎来系列进展。美国FDA批准首个直接注入大脑的基因疗法Kebilidi，用于治疗芳香族L-氨基酸脱羧酶缺乏症（AADCD）。美国FDA批准了首个无需风险评估缓解策略计划（REMS）的嵌合抗原受体（CAR）-T细胞疗法Aucatzyl，用于治疗复发或难治性B细胞急性淋巴细胞白血病（r/r B-ALL）的成年患者。斯坦福大学医学院团队开发的CAR-T细胞疗法使1名患有治疗致命脑瘤的患者已获得了持续30多个月的完全缓解（CR）。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 图片来源：123RF ———✦研发进展✦——— ◇ PTC Therapeutics公司宣布，美国FDA加速批准基因疗法Kebilidi（eladocagene exuparvovec）用于治疗芳香族L-氨基酸脱羧酶缺乏症的儿童和成人患者，无论他们的疾病严重程度为何。AADCD患者由于编码AADC酶的DDC基因出现变异，导致AADC酶功能缺失，因此患者无法合成运动功能所必需的神经递质多巴胺。根据新闻稿，这是FDA批准的首个直接注射到大脑的基因疗法。 Kebilidi是一种基于腺相关病毒2（AAV2）载体的基因疗法，旨在通过一次性治疗，将功能正常的DDC基因直接递送到大脑的壳核中，提高AADC酶的水平并恢复多巴胺的生产。该疗法通过立体定向神经外科手术直接施用于大脑壳核。临床试验结果表明，患者在接受该基因治疗后，多巴胺会从头合成，随后逐渐获得运动发育里程碑。2022年，它首次获得欧盟委员会批准上市（商品名Upstaza），成为直接注入大脑的首款获批基因疗法，也是改变AADCD疾病进程的首款获批疗法。 ◇ Autolus Therapeutics公司宣布，美国FDA已批准Aucatzyl（obecabtagene autoleucel，obe-cel）用于治疗复发或难治性B细胞急性淋巴细胞白血病的成年患者。Aucatzyl是一款靶向CD19抗原的自体CAR-T细胞疗法。它旨在克服当前CAR-T细胞疗法在临床活性和安全性方面的局限性。它的独特之处在于，设计的CAR在与CD19结合后具有较快的解离速度（fast off-rate），从而能够尽量减少T细胞的过度激活，在降低毒副作用的同时，不易发生T细胞耗竭，提高CAR-T疗法的持久性。根据新闻稿，Aucatzyl是获美国FDA批准的首个无需风险评估缓解策略计划的CAR-T疗法。 此次美国FDA的批准主要是基于Aucatzyl在r/r B-ALL成人患者中所进行的FELIX临床试验的结果。65名疗效可评的患者中，总CR率为63%，其中包括51%的患者在任何时间达成CR，12%患者在任何时间达成CR伴不完全的血液学恢复（CRi）。此外，主要疗效终点是在3个月内达成CR，共有42%的患者达成，中位缓解持续时间（DOR）为14.1个月。安全性方面，Aucatzyl表现出低水平的细胞因子释放综合征（CRS），3级事件发生率为3%，无4级或5级事件。7%的患者出现≥3级免疫效应细胞相关神经毒性综合征（ICANS）。 ◇ Adaptimmune Therapeutics公司公布了其关键性2期IGNYTE-ESO试验的主要分析数据，该试验评估其在研细胞疗法lete-cel在之前接受过蒽环类药物治疗的晚期滑膜肉瘤或黏液样/圆细胞脂肪肉瘤（MRCLS）患者中的疗效与安全性。独立审评结果显示，27/64（42%）的患者达成根据RECIST v1.1标准的缓解，其中6例为CR，21例为部分缓解（PR）。患者的中位DOR为12.2个月（95% CI：6.8，19.5），中位无进展生存期（PFS）为5.3个月（95% CI：4.0，8.0）。 Lete-cel是一种靶向NY-ESO-1实体瘤抗原的工程化T细胞受体（TCR）-T细胞疗法。NY-ESO-1在滑膜肉瘤和MRCLS细胞中高度表达，该蛋白通过HLA-A*02呈现。基于所公布的积极数据，Adaptimmune计划在2025年底前启动lete-cel的生物制品许可申请（BLA）滚动提交，用于治疗晚期或转移性滑膜肉瘤和MRCLS。 ◇ 近日，《柳叶刀》（The Lancet）杂志上发表了使用经改良优化的基因疗法betibeglogene autotemcel（beti-cel）治疗严重输血依赖型β地中海贫血患者的疗效和安全性结果。Beti-cel是一款已上市的基因疗法，于2022年8月在美国获批上市。在两项1/2期beti-cel的研究中，研究人员发现非β0/β0基因型患者中约79%可“摆脱”输血依赖，而大多数β0/β0基因型患者（63%）仍存在输血依赖，不同基因型β地中海贫血患者结局之间的差异，提示需改良细胞生产技术，增加载体拷贝数，改善转导的造血干细胞质量和产量。 研究人员将beti-cel进行了改良优化，使用BB305慢病毒载体转导以提高转导效率。在一项3期临床试验中，改良的beti-cel使89%（16/18）不同基因型的严重输血依赖型β地中海贫血患者摆脱输血依赖，效果维持至少3年多，有望使患者血红蛋白水平接近正常，且无异基因自体造血干祖细胞（HSPC）移植风险和局限性。该研究结果表明，改良的beti-cel有望成为β地中海贫血患者的潜在治愈方式。 ◇ Neurogene公司公布了其用于治疗Rett综合征的基因疗法NGN-401在1/2期临床试验中的积极中期数据。截至2024年10月17日的数据，所有受试者在临床医生评定的临床总体印象改善量表（CGI-I）上与基线水平相比提高了2分。所有受试者在护理者填写的Rett综合征行为问卷（RSBQ）上有所改善，与基线水平相比改善了28%-52%不等。所有在基线时有睡眠障碍、便秘和吞咽困难的受试者都表现出客观的改善。技能和发育里程碑方面的进步是一致的、持久的，并且随着时间的推移而加深，显示出超出自然病程预期的改善。此外，低剂量NGN-401具有良好的耐受性和有利的安全性。 NGN-401是一种在研AAV基因疗法，递送全长人甲基胞嘧啶结合蛋白2（MECP2）的转基因。NGN-401利用Neurogene专有的基因调控技术，能够实现目标水平的MECP2转基因表达，而不会引起与传统基因治疗相关的过表达相关毒性。 ◇ Fate Therapeutics公司公布了其靶向人表皮生长因子受体2（HER2）的多重工程化CAR-T细胞疗法FT825/ONO-8250的初步临床数据。在一项正在进行的1期研究中，3名既往接受过大量治疗的晚期实体瘤患者接受了低剂量的FT825/ONO-8250治疗，没有观察到剂量限制性毒性（DLT）和任何级别的CRS、ICANS或移植物抗宿主病（GvHD）。在治疗后第8天观察到CAR-T细胞扩增峰值，并且患者外周血的FT825/ONO-8250表型表明CAR-T细胞处于激活状态，没有耗竭的证据。 ◇ 近日，斯坦福大学医学院的研究团队在《自然》杂志上发表了一项研究，公布了使用GD2（一种二唾液酸神经节苷脂）靶向CAR-T细胞疗法治疗致命脑和脊髓肿瘤儿童患者的1期临床研究的积极结果。11名患有H3K27M突变型弥漫性内源性脑桥胶质瘤（DIPG）或脊髓弥漫性中线胶质瘤（sDMG）的患者接受了GD2-CART治疗，9例患者因疾病引起的神经功能残疾获得了改善，4名患者表现出显著的肿瘤体积缩小（52%、54%、91%和100%），3名患者的肿瘤体积有较小的缩小。其中一名患者自入组后已获得了持续30多个月的CR。 ◇ Next Generation Gene Therapeutics（NGGT）公司宣布其用于治疗苯丙酮尿症（PKU）的在研基因疗法NGGT002在中国进行的研究者发起的试验（IIT）中的积极数据。接受高剂量NGGT002的6名患者中有5名在给药3周后血浆苯丙氨酸水平恢复至正常范围内。首位接受NGGT002给药的患者在给药后40周，血浆苯丙氨酸水平持续保持在正常范围内。目前，该疗法已在中国和美国启动1/2期临床试验，以评估其治疗成人PKU患者的安全性和有效性。 ◇ Atamyo Therapeutics公司宣布，美国FDA已批准其一次性AAV基因疗法ATA-200的IND申请，可针对γ-肌聚糖蛋白相关肢带型肌营养不良2C/R5型（LGMD2C/R5）患者开展1b/2b期临床试验。γ-肌聚糖蛋白是一种跨膜蛋白，参与肌肉纤维与其周围组织的连接。LGMD2C/R5是一种罕见的遗传病，由γ-肌聚糖蛋白基因的突变引起。ATA-200能提供一个正常的基因拷贝，以产生γ-肌聚糖蛋白。在临床前小鼠模型中，ATA-200证明了其耐受性和纠正病症及病理生物标志物的能力。该公司计划在2024年第四季度开始招募患者。 ◇ 精准生物自主研发的CD19靶向CAR-T细胞疗法pCAR-19B（普基仑赛注射液）的2期临床研究数据入选美国血液学会（ASH）年会口头报告。数据显示，普基仑赛注射液针对中国3~21岁儿童和青年CD19阳性r/r B-ALL患者，最佳客观缓解率（ORR）达90.63%。根据ASH官网的公示，该研究代表了亚洲人群中首个儿童B-ALL的关键临床试验，有望为中国儿童和青少年r/r B-ALL患者提供了一种新的治疗选择。 目前，普基仑赛注射液的新药上市申请已经获中国国家药品监督管理局（NMPA）受理，并被纳入优先审评，用于治疗3～21岁儿童和青少年CD19阳性的r/r B-ALL患者。精准生物此前新闻稿介绍，该产品有望成为中国首款针对儿童白血病的CAR-T产品。 ◇ 中国国家药监局药品审评中心（CDE）官网公示显示，来恩生物自主研发的GZL-016注射液获临床试验默示许可，适应症为乙肝病毒相关肝细胞癌。GZL-016注射液是一种自体T细胞疗法，通过mRNA编码表达靶向乙肝病毒（HBV）表面抗原的TCR，赋予T细胞特异性识别并杀伤乙肝病毒相关肝细胞癌的能力。GZL-016注射液通过有效利用mRNA瞬时表达的特性，结合多次输注的方式，可很好地控制安全性并维持疗效。此外，患者在治疗前无需清淋，进一步提高了患者用药的安全性和可及性。新闻稿指出，GZL-016注射液是全球首个针对HBV的mRNA编码TCR-T细胞疗法，也是来恩生物继取得美国FDA和新加坡卫生科学局（HSA）默示许可后的又一重要成果。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ Nippon Shinyaku公司和Atsena Therapeutics公司就Atsena公司的潜在“first-in-class”基因疗法ATSN-101在美国的商业化以及在日本的开发和商业化签订了独家许可协议。ATSN-101是一种以AAV5为载体的基因治疗药物，通过视网膜下注射将功能性的人类GUCY2D基因引入光感受器，用于治疗因GUCY2D双等位基因突变引起的Leber先天性黑蒙。ATSN-101已获得美国FDA授予的罕见儿科疾病认定、再生医学先进疗法认定和孤儿药资格，用于治疗GUCY2D相关LCA1。 根据许可协议的条款，Nippon Shinyaku公司将获得ATSN-101在美国和日本的独家商业权利，而Atsena公司将保留世界其他地区的商业权利。Atsena Therapeutics公司将获得预付款以及额外的里程碑付款。 ◇ Vittoria Biotherapeutics公司宣布完成了2500万美元的私募融资，本轮融资是在2023年11月获得的1500万美元融资基础上追加的1000万美元。新闻稿指出，这笔资金将主要用于支持其主要肿瘤候选药物VIPER-101的1期临床试验。 VIPER-101是一种自体、删除CD5的基因编辑细胞疗法，用于治疗T细胞淋巴瘤患者。85%以上的T细胞淋巴瘤患者的癌细胞都存在CD5。VIPER-101利用公司专有的Senza5平台技术，通过基因编辑消除CD5介导的免疫抑制，从而增强T细胞的效应功能。在临床前研究中，VIPER-101已显示出优于传统CD5靶向CAR-T细胞的疗效。 ◇ 中盛溯源完成1.5亿元B轮融资，将加速推进诱导多能干细胞（iPSC）治疗领域多款临床管线的临床开发，以及后续产品商业化。围绕iPSC核心技术，该公司在抗炎修复、肿瘤免疫和再生医学三大医学方向上，将多条细胞药物管线推进至注册临床开发阶段。截止目前，已有1条iPSC来源的间充质干细胞（iMSC）和2条iPSC来源的自然杀伤细胞（iNK）管线处于1/2期临床试验，另有帕金森病和糖尿病等多条产品管线将陆续进入注册临床试验。 ◇ 康霖生物宣布完成1.5亿元A轮融资。该公司成立于2015年，专注于基因治疗创新药的研发和商业化，针对的领域多为严重影响人类健康的疾病，如地中海贫血症、艾滋病、血友病和帕金森病等。KL003细胞注射液是康霖生物的核心产品，为一款基因修饰自体造血干细胞产品，拟开发用于治疗成人或儿童输血依赖型β-地中海贫血症。KL003于2024年1月获得NMPA的批准开展临床试验，目前已经完成1期临床全部患者的入组工作。 ◇ 莱芒生物宣布完成超亿元A轮融资首关，所得资金将主要用于极低剂量代谢增强型CAR-T细胞药物IND申报、自动化生产工艺研发，以及加速推进代谢增强型实体瘤细胞治疗药物的临床转化。该公司新闻稿指出，其进展最快的在研管线为代谢增强型CD19 CAR-T疗法（Meta10-19注射液）。在一项IIT中，该疗法以极低的给药剂量（低至传统CAR-T疗法剂量的1%）实现了肿瘤细胞的完全清除，成功帮助超过20名复发难治性白血病或淋巴瘤患者达到CR状态并顺利出院。在最新的IIT临床研究中，临床专家将代谢增强型CD19 CAR-T疗法的注射剂量进一步降低至常规剂量的千分之一后仍然能够在患者体内高效扩增，并成功助力数位患者获得CR。莱芒生物还将启动针对实体瘤的代谢增强型细胞治疗临床研究，以期彻底改变细胞治疗对实体瘤疗效欠佳的现状。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy. Retrieved November 13, 2024 from https://www.prnewswire.com/news-releases/ptc-therapeutics-announces-fda-approval-of-aadc-deficiency-gene-therapy-302304880.html [2] Monje, M., Mahdi, J., Majzner, R. et al. Intravenous and intracranial GD2-CAR T cells for H3K27M diffuse midline gliomas. Nature (2024). https://doi-org.libproxy1.nus.edu.sg/10.1038/s41586-024-08171-9+ [3] Autolus Therapeutics Announces FDA Approval of AUCATZYL® (obecabtagene autoleucel – obe-cel) for adults with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). Retrieved November 8, 2024 from https://www.globenewswire.com/news-release/2024/11/08/2977884/0/en/Autolus-Therapeutics-Announces-FDA-Approval-of-AUCATZYL-obecabtagene-autoleucel-obe-cel-for-adults-with-relapsed-refractory-B-cell-acute-lymphoblastic-leukemia-r-r-B-ALL.html [4] Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024 Nov 8:S0140-6736(24)01884-1. doi: 10.1016/S0140-6736(24)01884-1. Epub ahead of print. PMID: 39527960. [5] Next Generation Gene Therapeutics (NGGT) Announces Positive New Data on NGGT002 for the Treatment of Phenylketonuria (PKU). Retrieved November 15, 2024, from https://www.prnewswire.com/news-releases/next-generation-gene-therapeutics-nggt-announces-positive-new-data-on-nggt002-for-the-treatment-of-phenylketonuria-pku-302300911.html [6] Fate Therapeutics Highlights Cancer-selective, HER2-Targeting Profile of FT825 / ONO-8250 CAR T-cell Product Candidate for Treatment of Advanced Solid Tumors at 2024 SITC Annual Meeting. Retrieved November 15, 2024, from https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-highlights-cancer-selective-her2-targeting [7] Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome. Retrieved November 15, 2024, from https://ir.neurogene.com/news-releases/news-release-details/neurogene-reports-positive-interim-efficacy-data-first-four-low [8] IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA. Retrieved November 15, 2024, from https://www.businesswire.com/news/home/20241112690214/en/IND-for-ATA-200-a-Gene-Therapy-for-the-Treatment-of-Limb-Girdle-Muscular-Dystrophy-Type-2CR5-LGMD2CR5-cleared-to-proceed-by-FDA [9] 4205 A Phase 2 Clinical Trial of Anti-CD19 CAR-T (pCAR-19B) in Chinese Pediatric and Young Adult with Relapsed/Refractory (R/R) CD19+ B-ALL: The First Pivotal Study in an Asian Population. Retrieved November 15, 2024, from https://ash.confex.com/ash/2024/webprogram/Paper206408.html [10] Vittoria Biotherapeutics Completes $25 Million in Private Financing to Propel Groundbreaking Cell Therapy Candidates. Retrieved November 15, 2024, from https://www.globenewswire.com/news-release/2024/11/12/2978833/0/en/Vittoria-Biotherapeutics-Completes-25-Million-in-Private-Financing-to-Propel-Groundbreaking-Cell-Therapy-Candidates.html [11] 莱芒生物完成超亿元A轮融资首关，加速推进代谢增强型免疫疗法临床转化. Retrieved November 15, 2024, from https://mp.weixin.qq.com/s/cT-tzg1d0nURECS8xohd3A [12] Adaptimmune's Lete-cel Achieves Primary Endpoint in Pivotal Trial. Retrieved November 13, 2024 from https://www.adaptimmune.com/investors-and-media/news-center/press-releases/detail/277/adaptimmunes-lete-cel-achieves-primary-endpoint-in-pivotal [13] 全球首个！来恩生物mRNA编码TCR-T获批临床，精准攻克肝细胞癌. Retrieved November 15, 2024 from https://mp.weixin.qq.com/s/0BvwbitxvT2hc4isp0qryg [14] Nippon Shinyaku and Atsena Therapeutics enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan. Retrieved November 15, 2024 from https://www.globenewswire.com/news-release/2024/11/13/2979922/0/en/Nippon-Shinyaku-and-Atsena-Therapeutics-enter-into-an-Exclusive-Strategic-Collaboration-for-ATSN-101-in-the-U-S-and-Japan.html 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。 分享，点赞，在看，聚焦全球生物医药健康创新

Nov. 13, 2024 -- Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO): Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first-in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S. Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial. ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV. “ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.” “This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities. We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” said Patrick Ritschel, CEO of Atsena Therapeutics. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.” LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1 is one of the most common forms of LCA and there are no approved treatments for it. ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for LCA1. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Results from this trial were recently published in The Lancet. The Rare Pediatric Disease Designation is granted by the FDA for promoting the development of new drugs for rare diseases that occur in the U.S. before the age of 18 and affect fewer than 200,000 people. With this designation, the product will be eligible to receive a Priority Review Voucher upon approval that could be used to advance another program. The Regenerative Medicine Advanced Therapy (RMAT) Designation is an FDA-designated system for advanced regenerative medicine therapies targeted at developing serious diseases that have shown certain effect in clinical trials based on the 21st Century Cures Act in the U.S. Companies with RMAT designations are given the opportunity for priority review and accelerated approval for the product. The Orphan Drug Designation is provided for orphan drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. With orphan designation, the FDA grants a seven-year market exclusivity for the product and provides certain incentives such as tax credits towards the cost of development, upon approval. Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. In an ongoing Phase I/II clinical trial, the company is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. In another ongoing clinical trial, the company is evaluating ATSN-201 for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood. 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/. Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. The content above comes from the network. if any infringement, please contact us to modify.