历史性成就：FDA批准Abeona Therapeutics的ZEVASKYN加速了发展势头，并扩大了EB社区的治疗选择-动脉网

Historic achievement: FDA approval of Abeona Therapeutics' ZEVASKYN accelerates momentum and expands treatment options for the EB community.

CISION 等信源发布 2025-04-30 00:32

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EB Research Partnership ('EBRP') invested in the work that led to ZEVASKYN at

EB Research Partnership（“EBRP”）投资了导致ZEVASKYN的工作

Stanford University

斯坦福大学

under their pioneering Venture Philanthropy Model. This approval marks a historic moment for the EB community as the third FDA approved treatment and the first autologous, cell-based gene therapy for RDEB (Recessive Dystrophic Epidermolysis Bullosa).

在其开创性的风险慈善模型下。这一批准标志着EB社区的历史性时刻，是第三种获得FDA批准的治疗方式，同时也是针对RDEB（隐性营养不良型大疱性表皮松解症）的首个自体细胞基因疗法。

NEW YORK

纽约

，

April 29, 2025

2025年4月29日

/PRNewswire/ -- In a significant advancement for the Recessive Dystrophic Epidermolysis Bullosa (RDEB) community, the U.S. Food and Drug Administration (FDA) has approved a third treatment for this rare condition. The milestone approval of Abeona Therapeutics' ZEVASKYN not only represents a continued commitment to innovation in RDEB care, but reinforces the growing momentum toward improved quality of life and expanded treatment choices for individuals and families affected by RDEB.

/PRNewswire/ -- 在隐性营养不良型大疱性表皮松解症（RDEB）患者群体的重要进展中，美国食品药品监督管理局（FDA）批准了针对这一罕见病的第三种治疗方案。Abeona Therapeutics公司开发的ZEVASKYN获得里程碑式的批准，不仅代表了对RDEB治疗创新的持续承诺，更加强了改善RDEB患者及其家庭生活质量、扩大治疗选择的日益增长的动力。

ZEVASKYN is the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene..

ZEVASKYN是首个自体细胞基因疗法，用于治疗RDEB，也是首个通过稳定整合的COL7A1基因在伤口部位提供VII型胶原蛋白表达的RDEB治疗方法。

继续阅读

“For over 20 years, I’ve watched my son face the relentless challenges of EB with strength no child should ever have to summon,” says Faye Dilgen, EBRP Board Member and mother of John Hudson Dilgen, who lives with RDEB. “This new treatment doesn’t just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won’t have to endure the same struggles.”.

“二十多年来，我目睹我的儿子面对EB带来的无情挑战，展现出本不应由孩子承受的坚强，”EBRP董事会成员、RDEB患者约翰·哈德森·迪尔根的母亲费伊·迪尔根说道。“这种新疗法不仅带来了新的缓解、希望和更多选择，还让我们更接近一个未来，让患有EB的孩子们不再需要经历同样的痛苦。”

EB is a group of devastating and life-threatening skin disorders that affect children from birth. Children with EB are often called “Butterfly Children” because their skin is as fragile as the wings of a butterfly.

EB是一组从出生起就影响儿童的毁灭性和危及生命的皮肤疾病。患有EB的儿童常被称为“蝴蝶儿童”，因为他们的皮肤如同蝴蝶的翅膀一样脆弱。

A BREAKTHROUGH IS NOW A BLUEPRINT

突破现在成为蓝图

EB Research Partnership (EBRP) is the world's largest nonprofit funding life-changing research to accelerate treatments and find a cure for EB, while pioneering a path for rare disease at large. EB is one of over 10,000 rare diseases, 95% of which have no approved treatments or cures. Through investments in cutting-edge approaches like gene therapies, stem cell therapies, antibody treatments, and more, EBRP is driving scientific breakthroughs that have the potential to impact the lives of 400 million people around the world living with a rare disease..

EB研究合作组织（EBRP）是全球最大的非营利组织，致力于资助改变生命的EB研究，以加速治疗方法的研发并寻找治愈EB的方法，同时为罕见病领域开辟道路。EB是超过一万种罕见疾病之一，其中95%的疾病尚无获批的治疗方法或治愈手段。通过投资于基因疗法、干细胞疗法、抗体治疗等前沿方法，EBRP正在推动科学突破，这些突破有望影响全球四亿名罕见病患者的生活。

'For over 20 years, I've watched my son face the relentless challenges of EB with strength no child should ever have to summon,' says

“二十多年来，我目睹我的儿子以一种不该由孩子承担的力量面对EB带来的无情挑战，”

Faye Dilgen

费伊·迪尔根

, EBRP Board Member and mother of

，EBRP董事会成员，也是

John Hudson Dilgen

约翰·哈德森·迪尔根

, who lives with RDEB. 'This new treatment doesn't just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won't have to endure the same struggles. We are incredibly grateful to the scientists, advocates, and especially to Abeona and EB Research Partnership for their tireless dedication in making this day possible for families like ours and future generations.'.

，他与RDEB一起生活。“这种新疗法不仅带来了新的缓解、希望和更多选择，还让我们离一个未来更近一步，在那个未来里，患有EB的儿童将不必再承受同样的痛苦。我们对科学家、倡导者，特别是Abeona和EB研究合作组织表达深深的感激，感谢他们不懈的努力，为像我们这样的家庭和后代创造了今天这一可能。”

The game-changing approval of ZEVASKYN underscores how progress in EB can serve as a model for advancements across the rare disease space. 'Abeona's development and advancement of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate the therapeutic landscape for not only EB, but many other rare diseases and conditions,' says .

ZEVASKYN 的获批是一项变革性的进展，突显了EB领域的进步如何为罕见病领域的整体发展提供借鉴。Abeona公司对ZEVASKYN的开发和推进为全球EB群体带来了里程碑式的时刻，其在基因治疗领域的领导地位不仅为EB，还为许多其他罕见病和病症的治疗前景带来了巨大的创新希望，相关人员表示。

Michael Hund

迈克尔·亨德

, CEO of EBRP.

，EBRP首席执行官。

A MODEL TO SCALE SOLUTIONS

一个可扩展解决方案的模型

With this FDA approval, EBRP gains continued momentum in its mission to discover treatments and a cure for EB. This milestone reinforces the validity of their scalable, sustainable Venture Philanthropy model, and, most importantly, provides treatment options and ignites hope for families around the world affected by EB..

随着FDA的批准，EBRP在其发现治疗和治愈EB的使命中获得了持续的动力。这一里程碑强化了其可扩展、可持续的风险慈善模型的有效性，而且最重要的是，为受EB影响的全球家庭提供了治疗选择并点燃了希望。

EBRP invested in the ground-breaking work that led to ZEVASKYN at

EBRP 投资了导致 ZEVASKYN 的开创性工作。

Stanford University

斯坦福大学

under their Venture Philanthropy Model. The funding followed the highly competitive application and screening process overseen by EBRP's Scientific Advisory Board (SAB), which is composed of leading scientists and physicians. EBRP was able to realize a significant return on its investment and reinvest that capital back into additional promising EB research projects..

在其风险慈善模型下。这笔资金的提供经历了由EBRP科学顾问委员会（SAB）监督的高度竞争性申请与筛选过程，该委员会由领先的科学家和医生组成。EBRP能够在其投资上获得显著回报，并将这些资本重新投入到更多有前景的EB研究项目中。

EBRP's Venture Philanthropy business model has been highlighted for its leadership by

东湾社区基金会的风险慈善商业模式因其领导力而受到关注。

Harvard Business School

哈佛商学院

, Stanford Social Innovation Review,

，斯坦福社会创新评论，

Yale University

耶鲁大学

, the Milken Institute, and

，米尔肯研究所，和

MIT

麻省理工学院

。

'EB Research Partnership is proud to be an early investor via our Venture Philanthropy Model in the science leading to ZEVASKYN at

EB研究合作组织通过我们的风险慈善模型，为ZEVASKYN的科学研究成为早期投资者而感到自豪。

Stanford University

斯坦福大学

. The collaboration between EBRP and

EBRP与

Stanford

斯坦福大学

exemplifies the mission of EBRP to advance commercially sustainable research aimed at treating and ultimately curing epidermolysis bullosa. We are honored to partner with the entire Abeona team and commend their leadership, determination, and passion to deliver much needed innovative solutions for individuals and their families living with EB.

体现了EBRP推动以治疗最终治愈表皮松解症为目标的商业化可持续研究的使命。我们很荣幸与Abeona团队合作，并赞扬他们的领导力、决心和热情，为表皮松解症患者及其家庭提供亟需的创新解决方案。

They continue to share our values and commitment to accelerate treatments to the EB community as quickly as possible,' says .

他们继续分享我们对尽快加速为EB社区提供治疗的承诺和价值观，' 说。

Michael Hund

迈克尔·洪德

, CEO of EBRP.

，EBRP首席执行官。

A COURAGEOUS COMMUNITY DRIVING CHANGE

一个推动变革的勇敢社区

EB is a group of devastating and life-threatening skin disorders that affect children from birth. Children with EB are often called 'Butterfly Children' because their skin is as fragile as the wings of a butterfly. With skin this fragile, those with EB face severe pain, open external and internal wounds, and grueling bandaging processes.

EB是一组从出生起就影响儿童的严重且危及生命的皮肤疾病。患有EB的儿童常被称为“蝴蝶儿童”，因为他们的皮肤像蝴蝶翅膀一样脆弱。由于皮肤如此脆弱，EB患者要承受剧烈疼痛、外部和内部伤口溃烂以及痛苦的包扎过程。

For the 500,000 people around the world living with EB, everyday activities like eating, sleeping, walking and playing can become monumental tasks that often require modification. .

对于世界各地生活在EB（大疱性表皮松解症）中的50万人来说，日常活动如吃饭、睡觉、走路和玩耍可能会变成艰巨的任务，常常需要进行调整。

Over the last decade, EBRP has made undeniable progress in its mission to find a cure for this community, including:

在过去十年中，EBRP在为这个群体寻找治愈方法的使命上取得了不可否认的进展，包括：

Raising over

筹集超过

$70M

7000万美元

Funding 160+ EB projects

资助160多个EB项目

Contributing to a 25x growth in the EB clinical trial landscape

为EB临床试验领域贡献了25倍的增长

Directly funding two FDA-approved EB treatments

直接资助两种FDA批准的EB治疗方法

Today's FDA approval of the first autologous, cell-based gene therapy for RDEB is an exciting proof point of this progress. EBRP will continue to build off this momentum and is dedicated to delivering a cure.

今天，FDA批准了首个针对RDEB的自体细胞基因疗法，这是这一进展令人兴奋的证明。EBRP将继续以此为基础，致力于实现治愈的目标。

A PERIOD OF UNPRECEDENTED AWARENESS

前所未有的意识觉醒时期

ZEVASKYN's approval arrives during an unprecedented wave of awareness for EB, fueled by recent high-impact initiatives led by EBRP and their Co-Founders Eddie and

ZEVASKYN的批准是在EBRP及其联合创始人Eddie和近期领导的高影响力举措引发的对EB前所未有的关注浪潮中到来的。

Jill Vedder

吉尔·维德дер

。

Launched in 2025, the Vedder Cup is an annual series between the San Diego Padres and Seattle Mariners that carries a meaningful purpose beyond the game. Through their partnership with EBRP, both teams are actively driving increased awareness and visibility for Epidermolysis Bullosa. This collaboration is not only shining a national spotlight on EB but also advancing efforts to accelerate the development of treatments and a cure..

2025年推出的维德杯（Vedder Cup）是圣地亚哥教士队和西雅图水手队之间的一项年度系列赛，其意义超越了比赛本身。通过与EBRP的合作，两支球队正在积极推动对表皮松解症（Epidermolysis Bullosa）的认知和关注。这一合作不仅让EB在全国范围内受到更多关注，还推动了加速治疗和治愈方法的研发工作。

The upcoming premiere of 'Matter of Time', a compelling documentary capturing the electrifying energy of

即将上映的《时间问题》是一部引人入胜的纪录片，捕捉了令人振奋的能量

Eddie Vedder's

艾迪· Vedder的

October 2023

2023年10月

solo concerts in

个人演唱会

Seattle

西雅图

, is bringing new levels of EB visibility on a global scale. Premiering in

，正在全球范围内带来新的EB可见度水平。首次亮相于

June 2025

2025年6月

at the Tribeca Festival, 'Matter of Time' weaves together powerful music and the moving stories of individuals impacted by EB - including families, researchers, and thought leaders - and highlights how innovation and determination are driving progress toward a cure. One such researcher chronicled throughout the film is Dr.

在翠贝卡电影节上，《时间问题》将感人的音乐和受到EB影响的个人故事交织在一起，其中包括家庭、研究人员和意见领袖，并突显了创新和决心如何推动治愈进展。影片中记录的一位研究人员是博士。

。

Jean Tang

唐珍

的

Stanford University

斯坦福大学

, who was lead principal investigator on the clinical of this third FDA approved treatment. The documentary is a testament to the power of music, community, and unwavering determination to drive change and inspire hope.

，他是这第三种FDA批准的治疗药物临床试验的首席研究员。这部纪录片证明了音乐、社区和坚定不移的决心能够推动变革并激发希望的力量。

Public awareness of EB has never been stronger—and this most recent FDA approval underscores the momentum building around research, advocacy, and support.

公众对EB的认识从未如此强烈——最近这次FDA的批准强调了在研究、倡导和支持方面不断增强的势头。

About EB Research Partnership

关于EB研究合作组织

Founded in 2010 by a dedicated group of parents along with

成立于2010年，由一群专心致志的家长与

Jill and Eddie Vedder

吉尔和艾迪· Vedder

of Pearl Jam, EB Research Partnership (EBRP) is the largest global nonprofit dedicated to funding research to accelerate treatments and find a cure for Epidermolysis Bullosa (EB), a group of devastating and life-threatening skin disorders that affect children from birth.

珍珠果酱乐队、EB 研究合作组织 (EBRP) 是全球最大的非营利组织，致力于为加速治疗和寻找治愈表皮松解症 (EB) 的研究提供资金支持。表皮松解症是一组从出生起就影响儿童的毁灭性且危及生命的皮肤疾病。

When making a grant to a research project, EBRP utilizes a Venture Philanthropy model. They retain the added upside of generating a recurring revenue stream if the therapy or product is commercially successful, then use the return on investment to fund additional EB research until a cure is found. While EB is a rare disease, there are 10,000 rare diseases that affect 1 in 10 people in the world, 95% of which have no treatments, that can benefit from the research they fund and this innovative model..

在为研究项目提供资助时，EBRP采用了风险慈善模式。如果疗法或产品在市场上取得成功，他们将保留产生经常性收入流的额外好处，然后利用投资回报来资助更多的EB研究，直到找到治愈方法。尽管EB是一种罕见病，但世界上有10,000种罕见病影响着十分之一的人口，其中95%没有治疗方法，这些研究和这一创新模式可以使他们受益。

To learn more about EBRP, visit

要了解有关 EBRP 的更多信息，请访问

www.ebresearch.org

For more information on ZEVASKYN, visit

有关 ZEVASKYN 的更多信息，请访问

www.zevaskyn.com

For more information on Matter of Time, visit

有关《时间问题》的更多信息，请访问

www.ebresearch.org/matter-of-time-film

www.ebresearch.org/时间问题电影

SOURCE EB Research Partnership

来源 EB研究合作组织