Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1.

拜耳公司和作为拜耳公司全资子公司独立运营的基因治疗公司Asklepios BioPharmaceutical，Inc.（AskBio）宣布，美国食品和药物管理局（FDA）已批准AB-1002计划的快速通道。AB-1002是一种研究性一次性基因疗法，用于心脏，旨在帮助促进蛋白质抑制剂1（I-1c）的组成型活性形式的产生，旨在阻断蛋白磷酸酶1的作用。

Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.(1,2) .

抑制这种与充血性心力衰竭（CHF）有关的蛋白质的功能可能会对心脏产生治疗作用。（1,2）。。

'The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of bringing potentially effective treatments to patients with advanced congestive heart failure,' said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.

AskBio首席开发官兼首席医疗官、医学博士姜灿文（Canwen Jiang）说：“FDA对AB-1002的快速通道指定是该项目临床开发的一项重要成就，并突出了我们为晚期充血性心力衰竭患者带来潜在有效治疗的目标。”。

'We look forward to completing our Phase II GenePHIT clinical trial, which is currently enrolling patients with severe heart failure, and are committed to exploring the full potential of AB-1002 for the treatment of this devastating disease.' .

“我们期待着完成我们的II期GenePHIT临床试验，该试验目前正在招募严重心力衰竭患者，并致力于探索AB-1002治疗这种毁灭性疾病的全部潜力。”。。

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.(3) The purpose of the Program is to get important new therapeutics to patients earlier.(3) Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. .

FDA快速通道计划旨在促进旨在治疗严重疾病和满足未满足医疗需求的新疗法的开发和加速审查。（3） 该计划的目的是尽早为患者提供重要的新疗法。（3） 获得该名称的治疗剂有资格与FDA更频繁地会面以讨论开发计划，如果符合相关标准，则有资格获得加速批准和优先审查。。。

'The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,' said Christian Rommel, PhD, Head of Research and Development at Bayer's Pharmaceuticals Division. 'This designation underpins the potential of AB-1002 to address currently high unmet medical need, and we are excited about the opportunity to accelerate its development.' .

拜耳制药部研发负责人克里斯蒂安·隆美尔（ChristianRommel）博士说：“AB-1002的快速通道设计强调需要快速推进新的治疗方式，例如针对充血性心力衰竭患者的基因治疗。”这一名称巩固了AB-1002解决当前高度未满足的医疗需求的潜力，我们对加速其发展的机会感到兴奋。”。。

AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio is currently enrolling patients in the Phase II GenePHIT (Gene Phosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.(4) .

AB-1002是一种尚未获得上市授权的研究性基因疗法，其疗效和安全性尚未确定或充分评估。AskBio目前正在招募患者参加AB-1002（也称为NAN-101）治疗CHF的II期GenePHIT（基因磷酸酶抑制疗法）试验。（四）。。

About GenePHIT

关于GenePHIT

GenePHIT is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA New York Heart Association (NYHA) Class III heart failure symptoms.(4) Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo.

GenePHIT是一项II期适应性，双盲，安慰剂对照，随机，多中心试验，旨在评估年龄>18岁的男性和女性通过冠状动脉内顺行输注一次性服用AB-1002的安全性和有效性，伴有非缺血性心肌病和NYHA纽约心脏协会（NYHA）III级心力衰竭症状。（4） 受试者以1:1:1的方式随机分为三个治疗组之一，分别为低剂量，高剂量或安慰剂。

Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test (6MWT).(4) For more information, please visit clinicaltrials.gov (NCT#05598333) or visit askbio.com. .

主要结局指标包括心血管相关死亡和NYHA分类基线变化，左心室射血分数（LVEF），峰值摄氧量（pVO2）和六分钟步行试验（6MWT）。（4） 有关更多信息，请访问clinicaltrials.gov（NCT#05598333）或访问askbio.com。。。

About Congestive Heart Failure

关于充血性心力衰竭

Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body's needs, including providing sufficient oxygen to the organs.(5) Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up.(6) This causes congestion in the body's tissues.

当心脏不能有效地泵送血液以满足身体的需要，包括为器官提供足够的氧气时，就会发生心力衰竭。（5） 充血性心力衰竭导致流出心脏的血流减慢，导致血液通过静脉回流到心脏。（6） 这会导致身体组织充血。

Symptoms include swelling in the legs and ankles. Sometimes, fluid collects in the lungs and interferes with breathing. Approximately 26 million people worldwide are living with congestive heart failure.(7) .

症状包括腿部和脚踝肿胀。有时，液体会积聚在肺部并干扰呼吸。全世界约有2600万人患有充血性心力衰竭。（七）。。

About AskBio

关于AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.

Asklepios BioPharmaceutical，Inc.（AskBio）是拜耳公司的全资和独立运营子公司，是一家全面整合的基因治疗公司，致力于开发挽救生命的药物和改变生活。该公司拥有一系列神经肌肉、中枢神经系统、心血管和代谢疾病适应症的临床项目组合，临床阶段包括充血性心力衰竭、亨廷顿舞蹈病、肢带型肌营养不良症、多系统萎缩、帕金森氏病和庞贝氏病的治疗。

AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing.

AskBio的基因治疗平台包括Pro10™，这是一种业界领先的专有细胞系制造工艺，以及广泛的衣壳和启动子文库。该公司的全球总部位于北卡罗来纳州三角研究公园，欧洲总部位于苏格兰爱丁堡，已经产生了数百种专有衣壳和启动子，其中一些已经进入临床前和临床测试。

An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric capsids..

作为基因治疗领域的早期创新者，该公司在五个国家拥有900多名员工，在AAV生产和嵌合衣壳等领域拥有800多项专利和专利申请。。

About Bayer

拜耳简介

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 'Health for all, Hunger for none,' the company's products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.

拜耳是一家全球性企业，在医疗保健和营养等生命科学领域拥有核心竞争力。该公司的产品和服务旨在帮助人们和地球繁荣，支持人们努力应对全球人口不断增长和老龄化带来的重大挑战，这与“人人健康，无人饥饿”的使命相一致。

Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros.

拜耳致力于推动可持续发展，并对其业务产生积极影响。与此同时，该集团旨在通过创新和增长来提高其盈利能力和创造价值。拜耳品牌代表着全世界的信任、可靠性和质量。2023财年，该集团雇佣了约10万人，销售额476亿欧元。

R&D expenses before special items amounted to 5.8 billion euros..

特殊项目前的研发费用为58亿欧元。。

1. Fish KM, Ladage D, Kawase Y, et al. AAV9.I-1c delivered via direct coronary infusion in a porcine model of heart failure improves contractility and mitigates adverse remodeling. Circ Heart Fail. 2013;6(2):310-317.

1.Fish KM，Ladage D，Kawase Y等。在猪心力衰竭模型中通过直接冠状动脉输注递送的AAV9.I-1c可改善收缩力并减轻不良重塑。Circ心脏衰竭。2013年；6（2）：310-317。

2. Pathak A, del Monte F, Zhao W, et al. Enhancement of cardiac function and suppression of heart failure progression by inhibition of protein phosphatase 1. Circ Res. 2005;96(7):756-766.

2.Pathak A，del Monte F，Zhao W等。通过抑制蛋白磷酸酶1增强心脏功能和抑制心力衰竭进展。中国保监会第2005号决议；96（7）：756-766。

3. US FDA - Fast Track. Available at: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track. Accessed April 2024.

美国FDA-快速通道。网址：https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track.2024年4月访问。

4. Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (GenePHIT). Available at: https://classic.clinicaltrials.gov/ct2/show/NCT05598333. Accessed April 2024.

冠状动脉内基因治疗对非缺血性NYHA III级心力衰竭（GenePHIT）患者的磷酸酶抑制作用。网址：https://classic.clinicaltrials.gov/ct2/show/NCT05598333.2024年4月访问。

5. CDC. Heart failure. Centers for Disease Control and Prevention. Published 2022. Available at: https://www.cdc.gov/heartdisease/heart_failure.htm. Accessed April 2024.

5、疾病预防控制中心。心力衰竭。疾病控制和预防中心。2022年出版。网址：https://www.cdc.gov/heartdisease/heart_failure.htm.2024年4月访问。

6. American Heart Association: Types of Heart Failure. Available at: https://www.heart.org/en/health-topics/heart-failure/what-is-heart-failure/types-of-heart-failure. April 2024

美国心脏协会：心力衰竭的类型。网址：https://www.heart.org/en/health-topics/heart-failure/what-is-heart-failure/types-of-heart-failure.2024年4月

7. Savarese G, Lund LH. Global Public Health Burden of Heart Failure. Card Fail Rev. 2017;3(1):7-11. doi:10.15420/cfr.2016:25:2.

萨瓦雷斯G，隆德LH。心力衰竭的全球公共卫生负担。卡片故障版本2017；3（1）：7-11。内政部：10.15420/cfr。2016:25:2。