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相关概念视频

Pharmacovigilance01:19

Pharmacovigilance

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Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
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Preclinical Development: Overview01:28

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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相关实验视频

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A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
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贝雷玛格尼 (Beremagene Geperpavec):第一次获得批准

Sohita Dhillon1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|July 11, 2023
PubMed
概括

贝雷玛格尼 (Beremagene geperpavec-svdt) (VYJUVEKTM) 是一种用于突变性表皮溶解的基因疗法. 它提供功能性COL7A1基因以恢复原蛋白,获得美国伤口治疗批准.

科学领域:

  • 生物技术是生物技术.
  • 基因治疗 基因治疗
  • 皮肤病学 皮肤病学

背景情况:

  • 形表皮溶解 (DEB) 是一种罕见的遗传疾病,其特征是严重的皮肤水泡形成.
  • COL7A1基因的突变损害了对皮肤完整性至关重要的七型原蛋白的产生.
  • 目前对DEB的治疗方法在很大程度上是辅助的,缺乏疾病修饰的选择.

研究的目的:

  • 总结一下贝雷马基因geperpavec-svdt (VYJUVEKTM) 的发展里程碑.
  • 为了突出基因治疗的旅程,导致其第一个监管批准.
  • 为了概述其在治疗形表皮溶解中的机制. bullosa.

主要方法:

  • 一种复制缺陷的单纯疹病毒-1 (HSV-1) 载体的发展.
  • 基因治疗的局部应用,以提供功能的人类 COL7A1 基因.
  • 切莱诺细胞和纤维细胞的转导以恢复 COL7 蛋白质的表达.

主要成果:

  • 贝雷马基因geperpavec-svdt成功地传导了质细胞和纤维细胞.
  • 已被证明可以恢复功能性的 COL7 蛋白质.
  • 在2023年5月获得美国FDA批准,用于治疗6个月及以上的DEB患者的伤口.

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结论:

  • 贝雷马根 (Beremagene geperpavec-svdt) 代表了DEB治疗的重大进展.
  • 基因疗法为主导性和衰退性DEB提供了潜在的疾病修饰方法.
  • 预计将在欧洲提交监管文件,扩大治疗可访问性.