Sentynl Therapeutics与PRG S&T达成协议，授权获得哈钦森-吉尔福德早衰综合征分子许可-动脉网

Sentynl Therapeutics Enters into Agreement with PRG S&T to License Molecule for Hutchinson-Gilford Progeria Syndrome

PR Newswire 等信源发布 2026-03-16 20:01

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Sentynl will acquire full rights to the investigational drug candidate, Progerinin (SLC-D011),

Sentynl将获得在研药物候选物Progerinin（SLC-D011）的全部权利，

adding to its commercial portfolio of rare and ultra-rare disease products

进一步丰富其罕见和超罕见疾病产品的商业组合

AHMEDABAD, India and SOLANA BEACH, Calif.

印度艾哈迈达巴德和美国加利福尼亚州索拉纳海滩

，

March 16, 2026

2026年3月16日

/PRNewswire/ --

Sentynl Therapeutics Inc.

森蒂尼尔治疗公司

Sentynl

森蒂尼尔

'), a U.S.-based biopharmaceutical company and wholly-owned subsidiary of Zydus Lifesciences Limited ('

“），一家总部位于美国的生物制药公司，也是 Zydus Lifesciences Limited 的全资子公司（“

Zydus

齐迪斯

'), announced that it is entering into an agreement with

“），宣布将与某公司达成协议，

PRG S&T

PRG 科技与创新

, a Korean company specializing in the development of medicine for rare genetic diseases, to license its investigational molecule Progerinin (SLC-D011) for Hutchinson-Gilford Progeria Syndrome (HGPS or 'progeria').

，一家专注于罕见遗传病药物开发的韩国公司，将其研究分子Progerinin（SLC-D011）授权用于治疗哈钦森-吉尔福德早衰综合症（HGPS或“早衰症”）。

The agreement will allow Sentynl to begin working with PRG S&T immediately to advance the clinical development of Progerinin (SLC-D011) for HGPS, which has been designated as an orphan drug by the United States Food and Drug Administration (FDA). Under the conditions that certain milestones are met, Sentynl will acquire full rights to the molecule for HGPS upon closing, making Progerinin the company's second therapy intended for the treatment of HGPS.

该协议将允许Sentynl立即与PRG S&T合作，推进Progerinin（SLC-D011）用于治疗HGPS的临床开发，该药物已被美国食品药品监督管理局（FDA）指定为孤儿药。在达到特定里程碑的条件下，Sentynl将在协议完成时获得该分子针对HGPS的全部权利，使Progerinin成为该公司第二种用于治疗HGPS的疗法。

The program is currently finalizing a Phase 2A clinical trial and data are expected before the end of 1H 2026..

该计划目前正在完成 2A 期临床试验，预计在 2026 年上半年结束前获得数据。

'This acquisition marks an important step in growing our portfolio of therapies for Hutchinson-Gilford Progeria Syndrome, which can have severe impacts on patient health if left untreated,' said Dr. Sharvil P. Patel, Managing Director, Zydus Lifesciences Limited. 'Supporting patients in living healthy, fulfilled lives is core to what we do, and the agreement with PRG S&T directly furthers this mission by advancing orphan therapies for patients and families impacted by rare diseases.'.

“此次收购标志着我们在扩展针对哈钦森-吉尔福德早衰综合征的疗法组合方面迈出了重要一步，如果不进行治疗，这种疾病可能对患者的健康造成严重影响，”Zydus Lifesciences Limited董事总经理Sharvil P. Patel博士表示。“支持患者过上健康、充实的生活是我们工作的核心，与PRG S&T的协议通过推进针对罕见病影响的患者和家庭的孤儿药疗法，直接推动了这一使命。”

'Children with Hutchinson-Gilford Progeria Syndrome face an unforgiving disease. However, we are seeing real progress in progeria research, with new science changing what's possible,' said Matt Heck, CEO, Sentynl. 'This agreement, which will add Progerinin to our progeria portfolio, represents our commitment to translating that progress into another real therapy for children and families who need them.'.

“患有哈钦森-吉尔福德早衰症的儿童面临着一种无情的疾病。然而，我们在早衰症研究中看到了真正的进展，新的科学正在改变可能性，”Sentynl首席执行官马特·赫克表示。“这项协议将把Progerinin加入我们的早衰症产品组合，代表了我们将这一进展转化为另一种真正疗法的决心，为需要帮助的儿童和家庭提供支持。”

Progerinin is an investigational, orally active small-molecule drug being developed as a potential treatment for Hutchinson-Gilford Progeria Syndrome, a rare genetic disorder characterized by accelerated aging in children. The disease is caused by the accumulation of progerin, an abnormal form of the lamin A protein produced by mutations in the .

Progerinin是一种在研的、口服活性小分子药物，正在被开发作为治疗哈钦森-吉尔福德早衰综合征的潜在药物，这是一种罕见的遗传疾病，其特征是儿童出现加速衰老。该疾病由早老素（progerin）的积累引起，早老素是由基因突变产生的异常形式的核纤层蛋白A。

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gene, which disrupts nuclear structure and leads to premature cellular aging. Progerinin is designed to inhibit the interaction and harmful effects of progerin within cells, thereby improving nuclear integrity and reducing cellular damage. It is not currently approved by FDA or any other health authority..

基因，它破坏核结构并导致细胞过早衰老。Progerinin旨在抑制progerin在细胞内的相互作用及其有害影响，从而改善核完整性并减少细胞损伤。目前，它尚未获得FDA或任何其他卫生机构的批准。

'The Progeria Research Foundation (PRF) is proud to have funded the foundational research that led to the development of Progerinin, and we are happy to see this potential path moving forward,' said Leslie Gordon, MD, PhD, Medical Director at Progeria Research Foundation. 'PRF's mission is to find treatments and the cure for Progeria, and we are grateful for the efforts of PRG S&T and Sentynl to improve the lives of the children and young adults in our Progeria patient community.'.

“普罗杰里亚研究基金会（PRF）为资助了导致Progerinin开发的基础研究感到自豪，我们很高兴看到这一潜在路径正在向前推进，”普罗杰里亚研究基金会医学主任莱斯利·戈登医学博士、哲学博士表示。“PRF的使命是寻找普罗杰里亚的治疗方法和治愈手段，我们对PRG S&T和Sentynl为改善我们普罗杰里亚患者社区中的儿童和年轻人的生活所做出的努力深表感谢。”

Early research and clinical trials aim to determine whether Progerinin can slow disease progression and improve survival in HGPS patients, potentially offering another therapeutic option. Currently, Zokinvy

早期研究和临床试验旨在确定Progerinin是否能够减缓HGPS患者的疾病进展并提高生存率，可能提供另一种治疗选择。目前，Zokinvy

(lonafarnib) is the only approved treatment for HGPS and certain processing-deficient Progeroid Laminopathies in the U.S., European Union, Great Britan, Israel, and Japan.

(lonafarnib) 是美国、欧盟、英国、以色列和日本唯一批准用于治疗 HGPS 和某些加工缺陷型早衰症核纤层蛋白病的药物。

About Progerinin

关于Progerinin

Progerinin is a small-molecule drug candidate being developed to treat Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic disorder that causes rapid aging in children. It works by targeting progerin, an abnormal protein produced from a mutation in the

普罗杰林是一种小分子药物候选物，正在开发用于治疗哈钦森-吉尔福德早衰综合征 (HGPS)，这是一种导致儿童快速衰老的罕见遗传疾病。它通过靶向早衰素（一种由基因突变产生的异常蛋白质）起作用。

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gene. In HGPS, this mutation causes cells to produce progerin instead of normal lamin A, which leads to defects in the cell nucleus and accelerates aging symptoms. In mouse models of HGPS, progerinin demonstrated encouraging outcomes. In mice with a severe form of the disease, treatment increased lifespan by 8–10 weeks and improved body weight.

基因。在HGPS中，这种突变导致细胞产生早衰素而不是正常的核纤层蛋白A，这会引起细胞核缺陷并加速衰老症状。在HGPS的小鼠模型中，早衰素显示出令人鼓舞的结果。在患有严重形式疾病的小鼠中，治疗使寿命延长了8到10周，并改善了体重。

The untreated control group had a shorter lifespan (average = 16.8 weeks; maximum = 18 weeks), whereas the treated group exhibited a significantly extended lifespan (average = 25.2 weeks; maximum = 26 weeks; p < 0.001)..

未处理的对照组寿命较短（平均 = 16.8 周；最长 = 18 周），而处理组的寿命显著延长（平均 = 25.2 周；最长 = 26 周；p < 0.001）。

About Progeria

关于早衰症

Collectively known as progeria, Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. HGPS is caused by an

统称为早衰症，哈钦森-吉尔福德早衰症综合征和早衰样核纤层蛋白病是超罕见、致命的遗传性早衰疾病，会加速年轻患者的死亡。HGPS 是由

LMNA

point mutation that produces the farnesylated lamin A variant, progerin, via aberrant splicing. Progeroid laminopathies are genetic disorders of accelerated aging caused by mutations in

通过异常剪接产生法尼基化早衰蛋白A变异体（早衰素）的点突变。早衰样层蛋白病是由加速衰老的基因突变引起的遗传疾病。

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and/or

和/或

ZMPSTE24

that impair lamin A processing, resulting in the accumulation of farnesylated prelamin A proteins including progerin.

这些因素会损害lamin A的加工过程，导致包括progerin在内的法尼基化prelamin A蛋白的积累。

2,3

Children with HGPS commonly die of atherosclerosis, the same heart disease that affects millions of normally aging adults by an average age of 14.5 years. Disease manifestations include severe failure to thrive, scleroderma-like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes..

患有HGPS的儿童通常在平均14.5岁时死于动脉粥样硬化，这种心脏病同样影响着数百万正常衰老的成年人。疾病表现包括严重的生长不良、类似硬皮病的皮肤、全身性脂肪营养不良、脱发、关节挛缩、骨骼发育不良、全身加速性动脉粥样硬化伴心血管衰退以及致残性中风。

About Zydus Lifesciences Limited

关于Zydus Lifesciences有限公司

Zydus Lifesciences Limited is an innovation-led life-sciences company with leadership positions across pharmaceuticals and consumer wellness, supported by an emerging MedTech franchise and a global footprint across the United States, India and other international markets. As of September 30, 2025, the group employs 29,000 people worldwide including 1,500 scientists engaged in R&D and is driven by its mission to unlock new possibilities in life sciences through quality healthcare solutions that impact lives.

Zydus Lifesciences Limited 是一家创新驱动的生命科学公司，在制药和消费者健康领域占据领先地位，拥有新兴的医疗技术业务，并在美国、印度及其他国际市场具备全球影响力。截至 2025 年 9 月 30 日，该集团在全球拥有 29,000 名员工，其中包括 1,500 名从事研发的科学家，其使命是通过影响生命的优质医疗保健解决方案，开拓生命科学的新可能。

The group aspires to transform lives through path-breaking discoveries. For more details visit .

该集团希望通过开创性的发现来改变生活。欲了解更多信息，请访问。

https://www.zyduslife.com

。

About Sentynl Therapeutics Inc

关于Sentynl Therapeutics Inc

Sentynl Therapeutics Inc. ('

Sentynl Therapeutics Inc.（

Sentynl

森蒂尼尔

') is a commercial stage U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. Recognized for its commitment to the rare disease community, Sentynl leverages its global operations as well as its parent organization, Zydus Group, to advance the development, manufacturing, and delivery of treatments to patients who need them in numerous countries worldwide.

')是一家处于商业化阶段的美国生物制药公司，专注于为罕见病患者带来创新疗法。Sentynl因其对罕见病社区的承诺而受到认可，公司利用其全球业务以及母公司Zydus集团的资源，推动治疗药物的研发、生产和交付，为全球众多国家的患者提供所需治疗。

Sentynl is dedicated to improving patient outcomes and access while upholding ethical standards and operating in compliance with applicable laws, regulations, and industry guidelines. For more information, visit .

Sentynl致力于在遵守道德标准并符合适用法律法规和行业指南的前提下，改善患者的治疗效果和获取途径。欲了解更多信息，请访问。

https://sentynl.com

。

About PRG S&T

关于PRG S&T

PRG S&T is a research & development company specializing in the treatment of rare genetic diseases. PRG S&T has been developing therapeutics for rare genetic diseases (laminopathies, neurodegenerative diseases, neuro-oncology etc.) with small molecules, which are derived from target sites on aberrant Protein-Protein Interactions (PPI).

PRG S&T 是一家专注于治疗罕见遗传疾病的研发公司。PRG S&T 一直致力于利用小分子开发针对罕见遗传疾病（如层蛋白病、神经退行性疾病、神经肿瘤学等）的治疗方法，这些小分子源自异常蛋白质-蛋白质相互作用 (PPI) 的靶点。

For more information, please visit .

有关更多信息，请访问。

www.eng.prgst.com

。

Resources

资源

1. Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford Progeria Syndrome. 2003 Dec 12

1. 戈登 LB，布朗 WT，柯林斯 FS。哈钦森-吉尔福德早衰症综合征。2003年12月12日

[Updated

[已更新

2019 Jan 17]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet].