GJB2-GT gene therapy(Sensorion SAS)

2026年4月，再生元的耳聋AAV基因治疗Otarmeni获得FDA批准上市。与此同时，再生元与美国政府达成协议，将向美国患者免费提供Otarmeni。 而现在，再生元正凭着这一“免费供应”策略开始杀死比赛。 首位竞品公司已经宣布退赛，后续可能将继续影响其他OTOF基因治疗开发项目。 延伸阅读：首个耳科基因治疗上市！再生元：免费！ Sensorion退赛 Sensorion是一家法国生物技术公司，专注于开发恢复、治疗和预防听力损失障碍的创新疗法。其最领先的管线就是靶向OTOF基因的AAV基因治疗SENS-501（OTOF-GT）。 SENS-501（OTOF-GT）是一款双载体AAV基因治疗，由Sensorion于2019年与巴斯德研究所合作而成的。SENS-501旨在通过利用AAV将功能性OTOF基因直接导入毛细胞，恢复由OTOF介导的听力损失。已经获得欧盟委员会孤儿药资格认定，及美国FDA罕见儿科疾病资格认定、孤儿药资格认定。 SENS-501于2024年6月在clinicaltrials.gov上注册了首个临床试验。2026年3月，Sensorion报告了SENS-501的临床试验六个月随访数据，数据显示：早期疗效信号持续，具有良好的安全性和耐受性。 但是随着首个OTOF基因治疗药物的获批上市——免费的Otarmeni横空出世，彻底改变了OTOF基因治疗的开发环境和市场环境。 Sensorion经过审查后，决定停止SENS-501的临床开发活动，转而将资源集中在GJB2基因治疗SENS-601上，该项目最近在加拿大和法国提交了临床试验申请，并获得法国机构（ANSM）批准的快速通道程序。 是否会影响更多？ OTOF是听神经病常见的遗传学原因之一。据统计，全球范围内约有20万名因OTOF介导的听力损失。这是一个具有广阔市场的疾病，也促使了多家企业进行OTOF AAV基因治疗的开发。 包括再生元，其OTOF AAV基因治疗Otarmeni已经成功在美国获批上市。其他竞争对手包括礼来（礼来于2022年10月以4.87亿美元收购Akouos，获得了OTOF基因治疗AK-OTOF），以及已经宣布退赛的Sensorion；国内也有几家公司正在开发OTOF AAV，包括鼎新基因、星奥拓维等。 延伸阅读：中国基因治疗令五名耳聋患儿恢复听觉 然而随着再生元的Otarmeni成功上市并在美国免费供应，美国OTOF听力损失市场将彻底被再生元统治。 在失去了美国这一全球最大的创新药市场后，Sensorion果断放弃，其他项目会怎么样发展呢？ 根据礼来于2026年4月30日发布的第一季度财报显示，AK-OTOF仍在持续推进中，目前正在进行1/2期临床，已经招募了22名患者，预计将于2028年10月完成研究。 而海外其他的OTOF AAV项目信息不多，但预计Sensorion不会是第一个放弃的。 但，国内OTOF AAV项目受到的影响可能不会太高。随着中国创新药市场规模的不断增长，医生对细胞与基因治疗等创新药物临床应用意识的不断提升，未来中国的听力基因治疗市场规模仍然可观。并且，中国创新药企还能将药物拓展至周边亚太地区国家。 参考资料： 1.https://www.businesswire.com/news/home/20260609361868/en/Sensorion-Achieves-Key-Regulatory-Milestones-for-SENS-601-for-GJB2-related-Hearing-Loss-and-Selects-Program-as-Lead-Gene-Therapy-Candidate 2.https://sensorion2021tf.q4web.com/English/news/news-details/2026/Sensorion-Reports-Six-Month-Update-from-the-Audiogene-Phase-12-Trial-of-SENS-501-and-Advances-GJB2-GT-SENS-601-Toward-First-in-Human-Clinical-Development/default.aspx 3.https://investor.lilly.com/static-files/587c9bd3-6551-45c3-989d-ab60c49036bb 4.https://www.biopharmadive.com/news/sensorion-gene-therapy-hearing-loss-otof-gjb2/822609/

Dive Brief:France-based biotechnology company Sensorion is discontinuing development of a gene therapy for a rare form of deafness after Regeneron Pharmaceuticals won approval for a rival product and agreed to offer it for free in the U.S.Researchers have been testing Sensorions treatment, called SENS-501, in a Phase 1/2 study of patients with mutations in a gene known as OTOF. Yet, the approval of Regenerons Otarmeni in April notably changed the development environment, Sensorion said Wednesday. Regeneron will offer Otarmeni at no cost as part of a larger drug pricing deal with the U.S. government.Following a strategic review, Sensorion has chosen to instead focus on starting human trials of SENS-601, a different experimental medicine designed to treat a more common form of deafness linked to mutations in the GJB2 gene. Concentrating Sensorions resources on SENS-601, where the unmet need is an order of magnitude larger and no approved therapy is currently available, is the right strategic decision, the company said.Dive Insight:While the move sets back the timeline for Sensorion to bring a product to market, it makes sense in the long run, according to analysts. Without the expenses of continuing the Audiogene trial of SENS-501, the biotechs cash reserves should last until the end of 2027.For a company of Sensorions size, careful resource allocation is critical, Stifel analyst Clemence Thiers wrote in a note to clients. Regenerons introduction of Otarmeni and the decision to offer it at no cost would likely have made the U.S. market near impossible to access for a follow-on product.The work done to advance SENS-501 wont go to waste. Lili Nsongo, an analyst at Leerink Partners, argues that Sensorion gained valuable experience with trial development and regulatory issues that will likely result in greater clinical development efficiency for SENS-601.The pivot to SENS-601 is a sensible move, Nsongo wrote in her own client note.Sensorion has been working with a French research team led by the renowned geneticist Christine Petit to develop SENS-601, a gene therapy that employs adeno-associated viruses. The treatment has prompted significant hearing restoration in animal models, Petit said in the companys statement.The scientific and clinical advances of Audiogene are directly guiding and increasing our confidence in SENS-601, she added.Sensorion shares, which trade on the Euronext Paris stock exchange, fell roughly 25% between Wednesday and Thursday. '