An Open-Label, Multicenter, Randomized, Non-Inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Weekly Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Patients With Alpha1-Antitrypsin Deficiency Compared to Corresponding Standard 60 mg/kg/Week and 120 mg/kg/Week Doses of Intravenous Alpha1-Proteinase Inhibitor (5%)

This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein.
Adults with Alpha-1 Antitrypsin Deficiency will take part. Participants will be randomly assigned to one of the treatment groups, and both the study doctors and participants will know which treatment is being given.
The main goals of the study are to understand how the body processes the medicine (pharmacokinetics) and to assess how safe and well tolerated the different weekly doses are.

开始日期2026-05-05

申办/合作机构

Grifols Therapeutics LLC

CTIS2024-517613-33-00

/ Recruiting临床2期IIT

A two-center, randomized, double-blind, placebo-controlled study of intravenous plasma-purified alpha-1 antitrypsin for hospitalized patients with COPD exacerbations (AECOPD study)

开始日期2025-10-16

申办/合作机构

Fondazione IRCCS Policlinico San Matteo

NCT07125664

/ Not yet recruiting临床2期IIT

A Two-center, Randomized, Double-blind, Placebo-controlled Study of Intravenous Plasma-purified Alpha-1 Antitrypsin for Hospitalized Patients With COPD Exacerbations (AECOPD Study)

Intravenous augmentation therapy with purified preparations of AAT (Alpha1-antitrypsin) derived from human plasma is a well consolidated specific therapeutic option to treat the severe deficient state of AAT. Prolastin is used to restore the balance between AAT and elastases in the lung and consequently to prevent a further deterioration in the pulmonary emphysema. Recently, in patients with COVID-19, without genetically lowered AAT levels with moderate to severe ARDS, treatment with AAT was demonstrated to be safe, feasible, and biochemically efficacious as an anti-inflammatory therapeutic therapy.
The aim of the study, based on biological plausibility, is to evaluate the safety and efficacy (from a biological perspective) of the administration of intravenous plasma-purified AAT as an anti-inflammatory treatment for patients admitted to hospital because of a COPD exacerbation leading to an acute or an acute on chronic respiratory failure.
Thirty-six adult patients hospitalized because of a COPD exacerbation leading to an acute or an acute on chronic respiratory failure will be enrolled by the two sites involved in the study, the Pneumology Unit of Fondazione IRCCS Policlinico San Matteo (Pavia, Italy) and the Pneumology Unit of IRCCS Istituto Clinico Humanitas, Rozzano (Milano, Italy).
Participants will be randomized 2:1 in the active treatment group (standard treatment + IV administration of Prolastin) or in the placebo group (standard treatment+ IV administration of 0.9% sodium chloride).
To address the clinical question from a biological perspective, we will investigate the decrease in inflammatory markers in the active treated group in comparison with the placebo group.
The primary objective is to demonstrate a significant reduction in systemic inflammation by IV Prolastin administered once at 120 mg per kilogram of body weight in patients with moderate to severe AECOPD, as assessed by the change in plasma concentration of IL6 at 7 days after randomization, in the active treatment group with respect to placebo group.
Secondary outcomes are:
1. Difference in change in plasma concentration of IL-1b, IL-5, IL-8, IL-10, and soluble TNF receptor 1 (sTNFR1), CRP at 7 days after randomization.
2. Differences in AAT antielastase activity, the amount of active elastase, the AAT levels in serum at baseline and at 7 days after randomization.
3. Difference in treatment failure rate. Treatment failure is a composite endpoint of need for either NIV or CPAP or need of ETI or need of transfer to ICU or in-hospital death after randomization
4. Impact of AECOPD on overall health, daily life, and perceived well-being in patients with obstructive airways disease by the change of St. George's Respiratory Questionnaire (SGRQ) score from Day 7 to follow-up (30 days after hospital discharge).
5. Differences in type and number of AEs and SAEs in the two groups
The expected duration of subject participation is from randomization to 30 days after hospital discharge, with a follow up phone contact.

开始日期2025-10-01

申办/合作机构-

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项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的文献（医药）

2026-12-01·MOLECULAR BIOLOGY REPORTS

The role of nitric oxide and connexin 43 in the cardioprotective effects of apigenin against doxorubicin-induced cardiotoxicity in male rats

Article

作者: Dehpour, Ahmad Reza ; Azizi, Yaser ; Goudarzi, Mina ; Safaei, Atlasi ; Fallah, Masoud ; Sheibani, Mohammad

BACKGROUND:

Doxorubicin (DOX) is one of the most widespread antibiotics used in the treatment of malignancies. Cardiotoxicity is the main side effect of DOX. Although the protective effects of apigenin have been shown, its mechanisms are still unknown. This study aimed to assess the role of nitric oxide (NO) in the protective effects of apigenin, particularly by altering the expression of connexin 43 (CX43) in the heart tissue.

METHODS AND RESULTS:

In this study, 60 male Wistar rats were divided into six equal groups: control (normal), DOX (2 mg/kg every 48 h for 12 days i.p.), apigenin (Api, 50 mg/kg oral administration), DOX + Api, DOX + L-NAME (Nitro-L-arginine methyl ester) (30 mg/kg i.p.), and DOX + Api + L-NAME. Our study assessed alterations in the heart weight and body weight, markers of cardiac function via echocardiography Ejection Fraction (%EF), Fractional Shortening (%FS), Left Ventricular Internal Diameter in Systole (LVIDs), and Left Ventricular Internal Diameter in Diastole (LVIDd), cardiac oxidative stress factors Superoxide Dismutase (SOD), Reduced Glutathione (GSH), Malondialdehyde (MDA), Catalase (CAT), cardiac injury markers Lactate Dehydrogenase (LDH), Creatine Kinase-MB (CK-MB), and Cardiac Troponin I (cTn-I), cardiac fibrosis, and CX43 expression (immunohistochemistry). Apigenin improved cardiac tissue damage and functional parameters such as left ventricular EF. Furthermore, treatment with apigenin, but not DOX, caused a significant reduction in the percentage of cardiac fibrosis and oxidative stress activity. Apigenin can also elevate the cardiac CX43 expression in the DOX + Api compared to the DOX group. NOS inhibition by L-NAME attenuates the protective effects of apigenin in DOX-induced cardiotoxicity COCCLUSION: Apigenin could exert its cardioprotective effects through an NO-dependent mechanism and elevation of CX43 expression.

2026-06-01·JACC. Advances

Integrated Hemodynamic Measures of Cardiac Function and Association With Clinical Outcomes

Article

作者: Trad, Omar K ; Kosyakovsky, Leah B ; Garan, Arthur R ; Grandin, E Wilson ; Quintero Pinzon, Pablo A ; Mounsey, Louisa ; Parekh, Juhi K ; Pomerantsev, Eugene V ; Ho, Jennifer E ; Picard, Michael H ; Sabe, Marwa

BACKGROUND:

Aortic pulsatility index (API) and cardiac power output (CPO) are strongly associated with mortality in patients with cardiogenic shock, but their association with outcomes across broader cardiopulmonary disease is unknown.

OBJECTIVES:

The objective of the study was to examine the association of API and CPO with clinical outcomes across a hospital-based cardiovascular cohort.

METHODS:

We used a hospital-based longitudinal, observational cohort of patients who underwent right heart catheterization. We calculated API = aortic pulse pressure/pulmonary capillary wedge pressure and CPO = (cardiac output x mean arterial pressure)/451. We used multivariable Cox models to examine the association of API and CPO quartiles with all-cause mortality, major adverse cardiovascular events (MACE), and heart failure hospitalization (HFH).

RESULTS:

A total of 4,278 patients (age 64 ± 11 years, 36.5% women, 35.3% prior heart failure) were included. Over a mean of 7 ± 3.2 years, there were 1,552 deaths, 1,429 MACE, and 991 HFH. Patients in the lowest API quartile had double the risk of death (HR: 2.06; 95% CI: 1.71-2.47), nearly double the risk of MACE (HR: 1.94; 95% CI: 1.65-2.30), and close to triple the risk of HFH (HR: 2.82; 95% CI: 2.31-3.45) compared to the highest quartile (all P < 0.001). Similarly, those in the lowest vs highest CPO quartile had greater risk of death (HR: 1.25; 95% CI: 1.08-1.46), MACE (HR: 1.51; 95% CI: 1.29-1.78), and HFH (HR: 1.90; 95% CI: 1.57-2.30; all P < 0.005).

CONCLUSIONS:

Although lower API and CPO are known prognostic metrics in patients with cardiogenic shock, these findings support their broader utility across the spectrum of cardiovascular disease.

2026-06-01·INTERNATIONAL JOURNAL OF PHARMACEUTICS

Development of UV-cured captopril-loaded ink for pharmaceutical 3D printing by custom-built SSE extruder

Article

作者: Czapor-Irzabek, Hanna ; Karolewicz, Bożena ; Śnietura, Wiktoria ; Malec, Katarzyna ; Krzaczkowski, Paweł ; Kozakiewicz-Latała, Marta ; Brożyna, Julia ; Brożyna, Malwina

It is widely acknowledged that conventional dosage forms are frequently not adequately customized to meet the specific clinical requirements of individual patients, what induces the need to process existing dosage forms to improve comfort and patient compliance. This phenomenon is especially evident in pediatric and geriatric populations, where there is a shortage of adequate doses of medication or where a difficulty is reported in their administration. Personalized soft gel-based dosage forms offer a viable solution to the problems associated with the administration of solid forms, especially in special needs populations. One of the most promising technologies that has the potential to facilitate personalization and the production of soft dosage forms is three-dimensional printing (3DP) using the semi-solid extrusion (SSE) method. This method allows the processing of low melting point materials and colloidal materials, e.g. gels and pastes. In this study, a light-curable gel (ink) was designed using a polyethylene glycol diacrylate (PEGDA) as a base polymer. Riboflavin and L-arginine were used as photoinitiators, demonstrating that it is possible to fabricate UV-cured gels without the need for potentially toxic ingredients such as triethylamine, or TPO/DPPO commonly used in this type of formulations. The model drug incorporated into the ink was captopril, which primary indication is the treatment of hypertension and heart failure. The possibility of using an individualized approach to its dosing could reduce the risk of adverse effects, particularly in elderly or pediatric patients. For the 3D printing process of soft dosage forms SSE extruder designed by the authors has been used. Demonstrative version of extruder can be installed in commercial FDM (Fused Deposition Modeling) printers which enables the execution of a considerable number of experiments using a variety of gels under controlled laboratory conditions, without the need for a significant financial investment. In a final experiment, a series of donut-shaped tablets were printed from the designed ink, which exhibited immediate drug release (over 80% within 30 min). Additionally, in this study an interesting phenomenon observed was improvement in polymerization of API-incorporated ink, what based on NMR spectra analysis may be explained by beneficial influence of captopril structure itself and its thiol group on cross-linking process progress. Summarizing, an ink composition formulated in the presented project that contains non-toxic photoinitiators and co-initiators can be utilized in an innovative self-assemble SSE extruder compatible with FDM commercial 3D printer, with optional UV-curing system.

项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的新闻（医药）

2026-04-07

·allsci.com

Airna’s RNA-editing therapy for PiZ mutation in AATD wins orphan status, starts Phase I

Massachusetts-based genetic therapy biotech Airna has dosed the first patient in the RepAIR1 Phase I trial of AIR-001, a GalNAc-conjugated oligonucleotide designed to correct the PiZ mutation in SERPINA1 mRNA by recruiting endogenous ADAR enzymes. The firm simultaneously announced that the FDA has granted Orphan Drug Designation to AIR-001 for alpha-1 antitrypsin deficiency (AATD). The open-label study, registered as NCT07431112, is enrolling adults with the PiZZ genotype across sites in Australia and the United Kingdom, with planned expansion to approximately 20 sites in 11 countries and a target enrolment of 54 patients. Primary endpoints cover safety, pharmacokinetics, and pharmacodynamic changes across total alpha-1 antitrypsin (AAT), functional AAT, Z-AAT, and M-AAT. AATD is caused by mutations in SERPINA1, with the PiZ variant driving the most common and severe form of the disease. The resulting Z-AAT protein misfolds and accumulates in hepatocytes, causing progressive liver injury, while reduced circulating functional AAT leaves lung tissue vulnerable to protease-mediated destruction. The dual organ involvement is a defining feature of AATD biology and a persistent challenge for therapeutic development. Research context The mechanistic logic behind AIR-001 maps directly onto the molecular basis of PiZZ (homozygous PiZ mutation) disease. The PiZ mutation is a single nucleotide substitution — a G-to-A change at the DNA level that produces a glutamate-to-lysine substitution in the AAT protein, driving its misfolding and polymerisation. ADAR enzymes catalyse adenosine-to-inosine editing of RNA, which ribosomes read as guanosine, making the PiZ site a tractable target for A-to-I correction without altering genomic sequence. AIR-001 exploits this by deploying a GalNAc-conjugated guide oligonucleotide to recruit endogenous ADAR to the relevant position on SERPINA1 mRNA. GalNAc conjugation provides hepatocyte-selective uptake via the asialoglycoprotein receptor, concentrating the drug in the liver where AAT is produced. If editing is efficient enough to restore meaningful M-AAT output, the approach could simultaneously reduce pathological Z-AAT accumulation and raise circulating functional AAT — addressing both disease compartments with a single subcutaneous agent. AIRNA’s platform was founded on research by Thorsten Stafforst at the University of Tübingen and Jin Billy Li at Stanford, whose work established the feasibility of recruiting native ADAR for therapeutic RNA correction. The reversibility of RNA editing is a design feature with practical implications. Because SERPINA1 mRNA is continuously transcribed, editing must be maintained through repeat dosing — but this also means the intervention can be stopped, a consideration relevant to safety monitoring in early-phase trials and to future combination strategies. Standard of care and where current therapies fall short The only approved disease-specific therapy for AATD remains intravenous plasma-derived AAT augmentation, first approved in 1987 and available in the US through products including Prolastin-C (Grifols), Aralast NP and Glassia (Takeda), and Zemaira (CSL Behring). Weekly IV infusions partially compensate for low circulating AAT in the lung but do not address the toxic gain-of-function from Z-AAT accumulation in the liver. No pharmacological therapy for AATD-associated liver disease has been approved. For patients with end-stage organ involvement, transplantation remains the only definitive option. The dependence on human plasma supply, the burden of weekly infusions, and the absence of any liver-directed therapy define the gaps that newer modalities are attempting to fill. AIR-001’s subcutaneous route and its theoretical capacity to act on both lung and liver disease position it differently from augmentation therapy, though whether preclinical signals translate to meaningful clinical benefit in both compartments remains to be established in RepAIR1 and subsequent trials. Competitive landscape The most clinically advanced novel agent in AATD is fazirsiran (ARO-AAT), an RNAi therapeutic developed by Arrowhead Pharmaceuticals in partnership with Takeda. Fazirsiran uses siRNA to suppress SERPINA1 expression in hepatocytes, reducing Z-AAT production and liver accumulation. It completed Phase II evaluation in the SEQUOIA trial, where it demonstrated reductions in hepatic Z-AAT and improvements in liver enzyme levels, and holds FDA Breakthrough Therapy Designation for AATD-associated liver disease. An NDA submission is anticipated, which would make fazirsiran the first approved pharmacological treatment for AATD liver disease. The mechanistic distinction between fazirsiran and AIR-001 is significant: fazirsiran suppresses all SERPINA1 expression indiscriminately, reducing Z-AAT but not restoring M-AAT, whereas AIR-001 aims to convert Z-AAT mRNA into M-AAT mRNA, theoretically producing functional protein rather than simply reducing a toxic one. Whether that distinction produces a clinically meaningful difference in lung outcomes — where circulating AAT levels matter — is one of the central questions AIR-001’s development will need to answer. Leave a Reply Cancel reply Your email address will not be published. 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孤儿药上市批准临床2期临床结果临床1期

2024-09-12

·Healthcare Digital

Grifols & Orange Business Launch Egypt Plasma Project

Grifols' Cairo project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines. Grifols & Orange Business Complete landmark phase of Data Centre for Plasma Derivatives Facility, designed to boost healthcare in Egypt Global healthcare company Grifols is working with French telecoms giant Orange to create a sustainable supply of essential therapeutics for life-threatening diseases in Egypt. Orange Business – the enterprise division of telecoms company, Orange Group – has completed the initial stage of a new data centre and communications infrastructure in Cairo, for Grifols Egypt for Plasma Derivatives (GEPD). GEPD is a joint venture between Grifols and Egypt's National Service Projects Organisation (NSPO), which manufactures military and civilian products and provides contracting services. The project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines -- vital treatments extracted from blood plasma for various serious medical conditions. Partnership to boost Egyptian healthcare Grifols, a specialist in plasma-derived medicines, is seeking to interconnect critical healthcare facilities, including a plasma laboratory, co-location data centre, plasma warehouse, 20 plasma donation centres, and a state-of-the-art manufacturing facility. Orange Business is providing co-location services, which allows GEPD to house its servers in Orange's data centre. The company is also supplying communication services and support with equipment procurement. The networked healthcare infrastructure is designed to ensure efficient operations and “the timely delivery of plasma-derived medicines to patients in need”, said Dr Ahmed Serag, Chief Strategy and Projects Officer at GEPD. Kristof Symons, CEO International at Orange Business, said: "Our technical skills, experienced resources, and global knowledge were pivotal in enabling Grifols to replicate its global business model in Egypt. This project shows how Orange Business can support its customers in creating a positive impact in today's society." About Grifols Multinational healthcare company Grifols was founded in 1940 in Barcelona, Spain employs 24,000 people worldwide, and specialises in the production of plasma-derived medicines and the development of diagnostic solutions. Its core activities include plasma collection, the manufacture of protein therapies derived from plasma, and fractionation, a process that separates a mixture into two or more fractions with different chemical and physical properties. Grifols is conducting clinical trials for its Alzheimer's treatment , a plasma-based therapy aimed at slowing cognitive decline in patients with mild to moderate Alzheimer's disease. The company is also researching treatments for liver diseases and immunodeficiencies. Among Grifols' products are Gamunex, an immune globulin therapy used to treat primary immunodeficiency, and Prolastin, used in the treatment of alpha-1 antitrypsin deficiency. The firm also produces albumin solutions for fluid replacement and coagulation factors for haemophilia treatment. In the diagnostic sector, Grifols offers blood typing systems and infectious disease screening tools. The company operates a network of plasma donation centres, primarily in the US, which form a crucial part of its supply chain for plasma-derived therapies.

2024-08-04

·药明康德

使90%阿尔茨海默病患者认知功能稳定或改善的NK细胞疗法；旨在功能性治愈乙肝的创新疗法进入临床…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 治疗阿尔茨海默病（AD）的自然杀伤（NK）细胞疗法SNK01在一项1期临床试验中使90%患者的认知功能稳定或有改善。 2. FDA批准了能在体内制造嵌合抗原受体（CAR）-T细胞的创新疗法UB-VV111的IND申请，预计将于今年年底前启动1期临床试验。 3. 有望功能性治愈乙型肝炎病毒（HBV）感染的重组多克隆抗体疗法GIGA-2339获许可进入临床。药明康德内容团队整理 SNK01：公布1期临床试验的新数据 NKGen Biotech公布了其在研NK细胞疗法SNK01治疗阿尔茨海默病患者的临床试验数据。SNK01是一款自体、非基因工程改造的NK细胞产品，它具有增强的细胞毒性，活化受体表达率超过90%，可从任何供体中稳定生产。该候选疗法能够有效降低AD患者脑脊液（CSF）中的α-突触核蛋白（α-syn）水平，该蛋白的升高被认为与认知能力下降有关。目前，已批准的AD疗法均未将α-syn作为靶点。此前公布的1期试验结果显示，SNK01除了先前披露的对淀粉样蛋白β（Aβ）和神经炎症生物标志物的积极影响外，对晚期AD患者的认知功能也具有临床疗效。在10名可评估的AD患者中，90%患者的阿尔茨海默病综合评分（ADCOMS，一种反映患者认知功能的评分）较基线时有改善或保持稳定。此次公布的结果显示，尽管70%的受试者接受的是相对低剂量的SNK01治疗，仍有60%的受试者（6/10）的CSF α-syn水平较基线时有所下降。第11周时，6名患者中有5名受试者的α-syn下降与ADCOMS的稳定/下降相对应。安全性方面，没有观察到与治疗相关的不良事件。AD受试者对SNK01的耐受性良好，在所有测试剂量下均未观察到剂量限制性毒性。该公司表示，这些数据值得在更大剂量、更长疗程、更大规模的2期试验中进一步研究。 UB-VV111：IND申请获得FDA许可 Umoja Biopharma宣布，美国FDA已批准其在研CD19靶向原位生成（in-situ generated）CAR-T细胞疗法UB-VV111的IND申请，用以治疗血液恶性肿瘤。Umoja预计将在2024年底前启动1期试验并进行首位患者给药。根据新闻稿，UB-VV111可能是血液学人体试验中首个原位生成CD19靶向CAR-T细胞疗法。 UB-VV111是一款通过VivoVec平台开发的体内CAR-T细胞疗法。UB-VV111包含一个表面经过工程化的病毒包膜，以及编码CD19靶向CAR和雷帕霉素激活细胞因子受体（RACRTM）的转基因，旨在于患者体内扩增UB-VV111工程化CAR-T细胞。该疗法是VivoVec基因递送平台进入临床的首个管线。 GIGA-2339：IND申请获得FDA许可 GigaGen公司宣布，美国FDA已批准其IND申请，将于2024年第四季度启动一项1期试验，以评估该公司首款用于治疗HBV感染的重组多克隆抗体疗法GIGA-2339。GIGA-2339采用GigaGen的下一代平台开发，含有1000多种全人重组抗HBV抗体，旨在复制人体的自然免疫反应。GIGA-2339的效力是血浆衍生HBV药物的2000多倍，涵盖了广泛的循环HBV变异。在小鼠模型中，GIGA-2339中和并清除了HBV的病毒DNA及其抗原。通过这种独特的作用机制，GIGA-2339有可能清除病毒颗粒并激活免疫反应，从而提供HBV感染患者的功能性治愈。 Sabirnetug（ACU193）：公布1期临床试验的新数据 Acumen Pharmaceuticals公司公布了sabirnetug（ACU193）用于治疗AD的1期临床试验的新结果。Sabirnetug是一种选择性靶向毒性可溶性β淀粉样蛋白寡聚体（AβOs）的人源化单克隆抗体。Sabirnetug能够结合AβOs，由于其独特的结合特性，sabirnetug有可能在产生淀粉样蛋白相关成像异常（ARIA，一种抗体疗法产生的常见主要副作用）风险较低的情况下提供治疗益处，因为sabirnetug可以在不直接靶向淀粉样斑块的情况下阻断AβOs的毒性作用。此次公布的结果显示，接受sabirnetug给药3次可显著降低患者脑脊液中突触前和突触后蛋白水平，这与sabirnetug抑制AβOs突触结合的作用机制一致。VAMP2是一种与突触损伤相关的生物标志物，在所有多剂量递增组中均显著降低，似乎是该研究中发现的对sabirnetug最敏感的生物标志物。Acumen公司计划在正在进行的为期18个月的2期临床试验中评估生物标志物的长期变化及其与临床结果的关系，以进一步证实sabirnetug的作用机制。此外，Acumen公司还宣布了sabirnetug皮下制剂治疗早期AD的1期临床试验已完成首例受试者给药。 Pepinemab：公布1b/2a期临床试验数据 Vaccinex公司公布了pepinemab治疗AD的1b/2期试验数据的良好初步结果。Pepinemab是一种人源化IgG4单克隆抗体，旨在阻断SEMA4D与plexin-B1受体结合，从而避免引发细胞内肌动蛋白细胞骨架的崩溃，以及进一步导致的大脑中星形胶质细胞和其他胶质细胞以及免疫组织中树突状细胞的稳态功能丧失。此次公布的结果证实了以激活星形胶质细胞为目标的新型作用机制有望延缓AD从早期轻度认知障碍发展为老年痴呆症的进程。该研究结果与此前pepinemab治疗亨廷顿病的2期临床试验结果类似，因为AD与亨廷顿病在神经免疫病理学方面具有相似性。 RGX-202：公布1/2期临床试验的新数据 REGENXBIO公司宣布，其在研杜氏肌营养不良症（DMD）基因疗法RGX-202在治疗1至11岁DMD患者的1/2期临床试验中获得了积极的中期安全性和疗效数据。在接受关键性临床试验推荐剂量（剂量水平2）的RGX-202治疗的5.8岁和8.5岁患者中，接受治疗后三个月时微抗肌营养不良蛋白表达水平分别为健康对照组的77.2%和46.5%。该试验中按剂量和年龄分类的微抗肌营养不良蛋白表达结果如下表所示： ▲AFFINITY DUCHENNE试验中接受不同剂量RGX-202治疗患者的微抗肌营养不良蛋白表达水平（图片来源：参考资料[1]） RGX-202是一种一次性基于腺相关病毒载体（AAV）的基因疗法，它编码包含天然抗肌营养不良蛋白C端（CT）结构域的微抗肌营养不良蛋白，以更好地模拟天然蛋白的功能。临床前研究中，CT结构域已被证明可以保护肌肉免受收缩引起的应力，并提高其自我修复的能力。 XPro：公布1期临床试验数据 INmune Bio公司公布了其下一代肿瘤F坏死因子（TNF）抑制剂XPro用于治疗AD的1b期临床试验结果。XPro的作用与现有的TNF抑制剂不同，它能中和可溶性TNF（sTNF），而不影响跨膜TNF（tmTNF）或TNF受体。XPro可在不抑制免疫的情况下减轻神经炎症，从而对神经系统疾病患者产生潜在的实质性益处。此次公布的结果显示，接受XPro治疗12周后，患者的突触蛋白发生了显著变化，而突触蛋白对神经元之间的交流至关重要。 NTLA-3001：获批在英国启动1/2期临床试验 Intellia Therapeutics公司宣布其临床试验申请（CTA）获得英国药品与保健品管理局（MHRA）的授权，可启动一项1/2期研究以评估NTLA-3001治疗α1抗胰蛋白酶缺乏症（AATD）相关的肺部疾病的效果。AATD是一种罕见的遗传性疾病，最常见的表现是由于α1抗胰蛋白酶（AAT）蛋白水平不足导致的肺功能障碍。NTLA-3001是一种全身给药的体内一次性基因编辑疗法，它旨在通过CRISPR/Cas9技术精确插入编码AAT蛋白的SERPINA1基因的健康拷贝，单次给药就有可能使功能性AAT蛋白永久表达并恢复到治疗水平。这种方法旨在改善患者的治疗效果，包括消除每周静脉注射AAT的疗法或严重病例对肺移植的需求。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202. Retrieved August 1, 2024, from https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-new-positive-data-affinity-duchenner-trial [2] Acumen Pharmaceuticals Presents Patient Experience and Biomarker Data from Phase 1 INTERCEPT-AD Study at the Alzheimer’s Association International Conference (AAIC®) 2024. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/28/2919969/0/en/Acumen-Pharmaceuticals-Presents-Patient-Experience-and-Biomarker-Data-from-Phase-1-INTERCEPT-AD-Study-at-the-Alzheimer-s-Association-International-Conference-AAIC-2024.html [3] Acumen Pharmaceuticals Announces First Subject Dosed in Phase 1 Study of Subcutaneous Sabirnetug (ACU193) for Early Alzheimer’s Disease. Retrieved August 2, 2024, from https://investors.acumenpharm.com/news-releases/news-release-details/acumen-pharmaceuticals-announces-first-subject-dosed-phase-1 [4] NKGen Biotech Presents New Positive SNK01 Biomarker Data at the 2024 Alzheimer’s Association International Conference. Retrieved August 2, 2024, from https://nkgenbiotech.com/nkgen-biotech-presents-new-positive-snk01-biomarker-data-at-the-2024-alzheimers-association-international-conference/ [5] Umoja Biopharma Announces FDA Clearance of IND Application for UB-VV111, a CD19 Directed in situ CAR T for Hematologic Malignancies. Retrieved July 31, 2024 from https://www.umoja-biopharma.com/news/umoja-biopharma-announces-fda-clearance-of-ind-application-for-ub-vv111-a-cd19-directed-in-situ-car-t-for-hematologic-malignancies/ [6] Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2920893/0/en/Intellia-Therapeutics-Receives-Authorization-to-Initiate-Phase-1-2-Clinical-Trial-of-NTLA-3001-for-the-Treatment-of-Alpha-1-Antitrypsin-Deficiency.html [7] Vaccinex Reports Positive Data for SIGNAL-AD Phase 1b/2 trial of Pepinemab in Alzheimer’s Disease. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921918/34858/en/Vaccinex-Reports-Positive-Data-for-SIGNAL-AD-Phase-1b-2-trial-of-Pepinemab-in-Alzheimer-s-Disease.html [8] Citryll Completes Patient Dosing In The Repeat Dose Stage Of The Phase 1 Trial. Retrieved August 2, 2024, from https://citryll.com/newsitems/citryll-completes-patient-dosing-in-the-repeat-dose-stage/ [9] GigaGen Receives FDA Clearance of IND Application for Phase 1 Trial of Recombinant Polyclonal for HBV Treatment, GIGA-2339. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921826/0/en/GigaGen-Receives-FDA-Clearance-of-IND-Application-for-Phase-1-Trial-of-Recombinant-Polyclonal-for-HBV-Treatment-GIGA-2339.html [10] Compugen Announces FDA Clearance of IND for COM503 for the Treatment of Solid Tumors. Retrieved August 2, 2024, from https://www.prnewswire.com/news-releases/compugen-announces-fda-clearance-of-ind-for-com503-for-the-treatment-of-solid-tumors-302208442.html [11] Georgiamune Announces First Patient Dosed in A First-In-Class Phase 1 Cancer Treatment Trial of GIM-531. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2921055/0/en/Georgiamune-Announces-First-Patient-Dosed-in-A-First-In-Class-Phase-1-Cancer-Treatment-Trial-of-GIM-531.html [12] INmune Bio Inc. Announces New Phase 1 Study Data Demonstrating Dose-Dependent Effect of XPro™ on Proteins that Regulation Synapses in Alzheimer’s Patients. Retrieved August 2, 2024, from https://www.inmunebio.com/index.php/newsroom/2024-news/muneioncnnouncesewhase1tudyataemonstrati20240729060507 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床申请临床1期免疫疗法临床2期

100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Alpha1-proteinase inhibitor(Grifols SA)	B02AB02	DB00058

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
α1-抗胰蛋白酶缺乏症	日本	OrphanPacific, Inc.	2021-01-22
肺气肿	美国	Grifols Therapeutics, Inc.	1987-12-02

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
HIV感染	临床3期	美国	Grifols Biologicals, Inc.	2012-04-01
新型冠状病毒感染	临床2期	西班牙	Instituto Grifols SA	2020-07-29
1型糖尿病	临床2期	美国	Grifols Therapeutics, Inc.	2014-03-01
囊性纤维化	临床2期	-	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	丹麦	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	瑞典	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	英国	Grifols Therapeutics, Inc.	2003-12-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02947087 (CTgov)	临床1/2期	慢性胰腺炎	48	Prolastin-C (Prolastin-C)	鹽簾簾獵製遞蓋齋鏇繭(遞襯構獵顧憲衊淵製鹽) = 顧窪蓋鑰艱選齋夢窪蓋顧膚餘積壓壓鏇衊窪餘 (衊窪艱艱鏇夢鹹網網積, 42) 更多	-	2023-05-09
				Placebo (Placebo)	鹽簾簾獵製遞蓋齋鏇繭(遞襯構獵顧憲衊淵製鹽) = 襯繭衊製遞鑰壓廠築夢顧膚餘積壓壓鏇衊窪餘 (衊窪艱艱鏇夢鹹網網積, 78) 更多
NCT04547140 (CTgov)	临床2期	新型冠状病毒感染	57	Standard Medical Treatment	鏇鹹醖簾遞願憲窪壓鑰 = 鑰艱糧夢壓艱廠鏇糧獵遞鏇範鏇醖遞襯觸餘鑰 (艱願醖膚鑰遞淵憲膚繭, 鬱觸願願網糧獵夢壓繭 ~ 齋鹽夢醖築繭鹽願窪鹽) 更多	-	2023-03-22
JPRN-JapicCTI-163194 \| NCT02870348 (GTI1401-OLE, Pubmed \| Respir Investig) 人工标引	临床1/2期	α1-抗胰蛋白酶缺乏症	4	Alpha1-proteinase inhibitor	顧構顧遞願獵憲顧築簾(襯鑰蓋簾餘獵壓築艱憲) = 選艱廠衊遞齋壓鑰膚簾繭築鹹選壓簾餘鬱鑰範 (襯艱鏇鹽範襯糧鏇壓構 ) 更多	积极	2022-08-12
NCT02870348 (GTI1401-OLE, CTgov)	临床1/2期	α1-抗胰蛋白酶缺乏症	4	Alpha-1 MP	網鹹鏇製齋鏇糧鑰衊製 = 選醖製繭蓋淵鏇製積夢艱鏇憲糧艱夢遞鹹齋鑰 (糧範淵餘蓋選鏇膚鹹遞, 淵餘膚蓋憲壓獵壓餘淵 ~ 餘構艱廠構膚淵淵製簾) 更多	-	2022-03-21
NCT01731691 (CTgov)	临床2/3期	HIV感染	12	α1 Proteinase Inhibitor (α1 Proteinase Inhibitor in HIV Disease)	夢廠遞鹹繭鬱夢鹽壓憲(網餘顧廠構窪鑰鏇衊窪) = 鏇憲願遞製積鏇構壓憲築積壓齋網糧淵築範繭 (構壓製築範壓鑰積壓築, 103) 更多	-	2020-08-31
				Placebos (Placebo in HIV Disease)	夢廠遞鹹繭鬱夢鹽壓憲(網餘顧廠構窪鑰鏇衊窪) = 顧廠醖憲築網餘觸鑰築築積壓齋網糧淵築範繭 (構壓製築範壓鑰積壓築, 180) 更多
NCT02093221 (CTgov)	临床2期	1型糖尿病	76	180 mg/kg Alpha1-PI (Alpha1-PI 180 mg/kg/wk, 26 Weeks)	構顧願淵顧憲廠製鬱蓋(齋鹽醖鹽顧蓋鑰鹹鏇鬱) = 艱鏇艱製鑰齋簾鬱選鹽觸醖鏇鏇齋壓鑰選鹽願 (簾餘簾網壓憲壓鏇願築, 27.7184) 更多	-	2018-04-19
				180 mg/kg Alpha1-PI (180 mg/kg/wk Alpha1-PI, 13 Weeks)	構顧願淵顧憲廠製鬱蓋(齋鹽醖鹽顧蓋鑰鹹鏇鬱) = 築鑰鬱憲餘鬱鬱鬱餘積觸醖鏇鏇齋壓鑰選鹽願 (簾餘簾網壓憲壓鏇願築, 28.9450) 更多
NCT02282527 (CTgov)	临床2/3期	α1-抗胰蛋白酶缺乏症	32	Liquid Alpha₁-PI+Alpha1-PI (Liquid Alpha₁-PI)	網衊齋鹹願艱齋糧範糧(蓋襯衊範鹹糧醖齋鬱衊) = 願積鬱觸餘繭糧繭簾艱製遞構觸鏇構齋膚衊鹽 (遞窪築願衊築網製廠選, 23.041) 更多	-	2017-03-13
				Prolastin-C (Prolastin-C)	網衊齋鹹願艱齋糧範糧(蓋襯衊範鹹糧醖齋鬱衊) = 觸艱醖範醖築積鬱觸餘製遞構觸鏇構齋膚衊鹽 (遞窪築願衊築網製廠選, 25.230) 更多
NCT01936896 (VCU-α1RT \| VCU-Alpha1RT, CTgov)	临床1/2期	ST段抬高心肌梗死 \| 急性心肌梗塞	10	Alpha 1-Antitrypsin	憲鹽網簾膚鏇憲簾鏇壓(鬱鹹襯糧憲壓蓋鹽淵簾) = 齋範顧鬱齋選簾築憲鑰醖範膚網製壓襯糧夢積 (餘獵繭膚醖艱衊積憲範, 積製糧糧鏇壓顧襯簾淵 ~ 鏇壓糧簾壓壓築鹽壓築) 更多	-	2016-02-12
NCT01213043 (SPARK, CTgov)	临床2期	α1-抗胰蛋白酶缺乏症 \| 肺气肿	30	Prolastin-C (60 mg/kg Prolastin-C)	簾鑰鏇膚糧簾餘獵積構 = 淵鏇夢餘鑰繭構糧積遞餘鑰齋遞廠餘壓鹽構築 (遞積淵築淵餘鹹顧顧選, 餘襯觸顧醖顧齋積淵觸 ~ 衊繭鑰積憲夢夢鹹製壓) 更多	-	2013-05-20
				Prolastin-C (120 mg/kg Prolastin-C)	簾鑰鏇膚糧簾餘獵積構 = 淵艱製鬱簾製鑰鏇網餘餘鑰齋遞廠餘壓鹽構築 (遞積淵築淵餘鹹顧顧選, 廠鬱網選繭獵蓋簾憲築 ~ 艱壓願齋觸鏇憲網鏇鑰) 更多