Onasemnogene abeparvovec

ROCKVILLE, Md., Aug. 25, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the appointment of Greg Ciongoli to its Board of Directors, effective August 25, 2026. With multiple upcoming catalysts across its late-stage pipeline of potential first-and best-in-class gene therapies, Mr. Ciongoli joins the Board as REGENXBIO prepares to become a global commercial organization. Mr. Ciongoli has extensive experience in biotechnology investing, capital allocation and corporate strategy to support the Company's next phase of growth. Concurrent with Mr. Ciongoli's appointment and following years of distinguished service, Jerry Karabelas, Ph.D., who served as an independent director since 2015, and Jean Bennett, M.D., Ph.D., who served as an independent director since 2021, are retiring from the Board. "As REGENXBIO enters its next phase of growth, the Board has continued its strategic succession planning process to ensure the Company benefits from a complementary mix of scientific, clinical, commercial and capital markets expertise," said Curran Simpson, President and Chief Executive Officer of REGENXBIO. "Greg brings a combination of financial and strategic expertise that will strengthen our Board as we advance our late-stage portfolio and continue our work to create life-changing treatments for patients and long-term value for our shareholders." "We are grateful to Jerry and Jean for their exceptional service and meaningful contributions to REGENXBIO. Their leadership, insight, and commitment have helped shape the Company through a pivotal period of growth and innovation. As valued members of our Board, they have provided thoughtful guidance and steadfast support, leaving a lasting impact on REGENXBIO. We thank them for their many contributions and wish them every success in the future," said Ken Mills, Chairman of the Board. "I am pleased to join REGENXBIO's Board during this exciting and transformational time for the Company," said Mr. Ciongoli. "REGENXBIO has established a leadership position in gene therapy with a differentiated platform and compelling pipeline of potential first- and best-in-class medicines. I look forward to working with the Board and management team to help advance the Company's mission and strategic objectives." Mr. Ciongoli is the Founder and Managing Partner of Adiumentum Capital Management, an investment firm that works closely with organizations on capital structure optimization and asset allocation strategy. Prior to founding Adiumentum, Mr. Ciongoli spent over 16 years at The Baupost Group, where he served as Partner, Public Group Investing and led investments in public and private companies across multiple industries and geographies. Over the course of his career, he has focused extensively on healthcare investments, corporate governance, and strategic transformation across both public and private markets. Mr. Ciongoli currently serves as Board Chairman of Atara Biotherapeutics and is also a Board member at Zymeworks Inc. His prior board member and observer experience also includes Idenix Pharmaceuticals, Intarcia Therapeutics, Keryx Pharmaceuticals, Orexigen Therapeutics, Translate Bio, and others. Mr. Ciongoli received his MBA from Harvard Business School and BA from Princeton University's School of Public and International Affairs. ABOUT REGENXBIO Inc. REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie, and RGX-121(clemidsogene lanparvovec) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, FDA's review process, the success of clinical trials conducted by REGENXBIO, the ability to obtain and maintain regulatory approval of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2025, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACTS: Dana Cormack Corporate Communications [email protected] George E. MacDougall Investor Relations [email protected] SOURCE REGENXBIO Inc. 21% more press release views with Request a Demo

上周，美国FDA再次批准了一款AAV药物。 Ultragenyx Pharmaceutical的Genglycos（pariglasgene brecaparvovec-opnr，DTX401）获得FDA加速批准，适用于8岁及以上患有Ia型糖原贮积病（GSDIa）的成人和儿童患者。 Genglycos Ia型糖原贮积病（GSDIa）是一种极其罕见的遗传性代谢疾病，由G6PC基因突变导致葡萄糖-6-磷酸酶（G6Pase）缺乏引起。正常情况下，这种酶负责将肝脏和肾脏中储存的糖原分解为葡萄糖并释放到血液中，以维持空腹或两餐之间的血糖稳定。由于酶缺乏，患者在长时间未进食时极易发生严重的低血糖，甚至危及生命，并可能引发长期的代谢并发症。患者必须依靠极其严格的饮食管理，包括频繁进食、避免单糖食物，以及全天候（包括夜间）定时定量服用生玉米淀粉来预防低血糖。这种治疗方式不仅给患者及家庭带来沉重的日常负担，且血糖控制往往不够平稳，漏服一次就可能导致严重后果。 Genglycos是一种基于AAV载体的基因治疗，旨在利用AAV8载体将功能性G6PC基因输送到肝脏，来恢复肝脏释放储存葡萄糖的能力，帮助患者在空腹状态下维持血糖稳定。 Genglycos的获批是基于一项为期48周的随机、双盲、安慰剂对照的III期临床研究的积极数据，该研究结果显示：与安慰剂组相比，接受Genglycos治疗的患者的每日玉米淀粉摄入量较基线平均减少了31%，具有统计学意义；Genglycos治疗组的患者每日补充玉米淀粉的次数也平均减少了1次，均达到统计学显著差异；Genglycos治疗组的患者在低血糖范围（< 70 mg/dL）的血糖百分比较安慰剂平均增加3%。 安全性方面，临床试验中Genglycos治疗组患者报告的严重不良反应包括过敏性休克、肾上腺功能不全、乳酸水平升高和低血糖。最常见的不良反应包括转氨酶升高（肝酶升高）、恶心、头痛、便秘和高血糖。此外，治疗组出现高甘油三酯血症的比例明显高于安慰剂组（29% vs 8%）。该药物的处方信息中包含了关于过敏性休克、肝毒性、肾上腺功能不全及肿瘤发生风险的警告，并明确指出妊娠期妇女不应使用。 作为“加速批准”，Genglycos在上市后仍需要完成额外的上市后临床试验以确认其长期的临床获益。此外，作为加速批准的条件，Ultragenyx已同意通过现有的疾病监测计划，对50名接受商业化治疗的患者和20名对照患者进行为期两年的安全性和有效性数据收集，并对所有相关患者进行长达10年的长期随访。 不被看好的销售 实际上，GSDIa是一种极其罕见的罕见病。根据报道，GSDIa在美国影响1500至2500名患者，全球商业可及范围内约有6000至8000名患者。 Genglycos美国批发采购成本（WAC）定价270万美元。Ultragenyx预计将在20-60天内在专业治疗中心提供治疗服务。 分析师们并不看好Genglycos的销售，根据William Blair的分析师Sami Corwin预测，Glenglycos的峰值销售额将达到3.62亿美元。 不过随着Genglycos获批，Ultragenyx公司将得到一张“优先审评券”。最近，优先审评券的价格持续走高，Rocket的罕见儿科疾病优先评审凭证售价1.8亿美元、Denali售价1.95亿美元、Jazz售价则达到2亿美元。 Ultragenyx公司计划出售优先审评券，以支持另一款基因治疗UX111的商业化准备，UX111是一款IIIA型黏多糖贮积症基因治疗，FDA已于2026年4月接受了UX111的BLA，PDUFA日期为2026年9月19日。 Ultragenyx公司表示，随着Genglycos的获批，以及UX111的潜在商业化，公司预计将在2027年实现盈利。 叫好不叫座 至今，全球范围内已经有11款AAV基因治疗产品获得监管批准，包括UniQure的Glybera、Roche/Spark的Luxturna、诺华的Zolgensma、CSL Behring/UniQure的Hemgenix、BioMarin的Roctavian、Sarepta的Elevidys、辉瑞的Beqvez、PTC的Kebilidi/Upstaza、Regeneron的Otarmeni、Ultragenyx的Genglycos，以及中国药监局批准的信念医药的信玖凝。 但是除了诺华的Zolgensma之外，仍未有第二款十亿美元“重磅炸弹”AAV产品的诞生。 更多的AAV基因治疗产品在销售惨淡-退市之间挣扎。 全球首款AAV基因治疗Glybera早于2017年退市，原因是Glybera的适应症为脂蛋白脂酶缺乏症（LPLD），该患者群体群体极为罕见，市场需求有限。 Luxturna是首款获得FDA批准的眼科AAV基因治疗，其销售额在近年持续下滑。根据公开资料显示，Luxturna在2018年-2019年的累计净销售额达5500万美元，2023年销售额约为5100万美元，2024年销售额降至约2090万美元。 2025年2月，辉瑞的B型血友病基因治疗Beqvez撤市，原因是“需求疲软”，辉瑞停止了Beqvez的一切商业开发和活动。 2026年6月，A型血友病基因治疗Roctavian全面撤市。撤市原因是，Roctavian经历了三年的商业开发，销售业绩持续低迷，以及未有合适的卖家，BioMarin最终无奈选择主动撤市。 延伸阅读：接连撤市！先辉瑞，后BioMarin CSL的血友病B基因治疗Hemgenix自2022年11月获批上市后，一直未有具体的销售额数据披露。2024年财年内仅报告有12名患者接受治疗。在2026年3月，CSL发布公告表示Hemgenix正在经历全球暂时缺货，这将导致部分已具商业准入国家患者的治疗延误。 延伸阅读：CSL：Hemgenix缺货！ Upstaza是一款针对芳香族L-氨基酸脱羧酶缺乏症（AADC）的基因治疗，Upstaza在2023年的销售额为1300万美元；得益于2024年11月在美国获批上市（商品名为Kebilidi/），Upstaza的年销售显著增长达到5662.6万美元；但由于AADC患者基数小，其市场天花板明显。 Elevidys是一款DMD基因治疗，一度被视为第二款AAV重磅炸弹。但是由于在去年爆出的安全性问题，Elevidys销售暴跌，且在部分国家和地区（如巴西）已撤销/取消注册。 延伸阅读：AAV基因治疗Elevidys：冲刺“重磅炸弹”失败 2026年4月，再生元的耳科基因治疗Otarmeni获得FDA批准上市。但Otarmeni在美国免费供应，未来盈利有限。 延伸阅读：首个耳科基因治疗上市！再生元：免费！ Genglycos刚刚上市但销售潜力不被看好。 而国内的信玖凝则是上市一年未有商业患者，武田更是于近期终止了与信念医药关于信玖凝的商业化合作。 此外，也正是基于多款AAV基因治疗的商业市场的不理想，以及AAV新药研发的高难度、高投入、高风险，近几年已经有不少大型制药公司退出了相关领域。 参考资料： 1.https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-patients-aged-8-years-and-older-glycogen-storage-disease-type-ia 2.https://www.biopharmadive.com/news/ultragenyx-fda-approve-genglycos-GSDIa-gene-therapy/828369/