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相关概念视频

Preclinical Development: Overview01:28

Preclinical Development: Overview

5.7K
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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Clinical Trials: Overview01:11

Clinical Trials: Overview

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Drug Discovery: Overview01:26

Drug Discovery: Overview

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

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Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
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In Vitro Drug Release Testing: Overview, Development and Validation01:10

In Vitro Drug Release Testing: Overview, Development and Validation

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In vitro dissolution and drug release tests assess how quickly and how much of a drug is released from its dosage form into an aqueous medium under standardized laboratory conditions. These tests are essential tools in pharmaceutical development and quality assurance, offering insight into the drug's performance before clinical use.During formulation development, dissolution testing identifies incomplete or inconsistent drug release issues. It also supports decisions on selecting the optimal...
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Drug Regulation01:25

Drug Regulation

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Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
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相关实验视频

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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
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药物开发 药物开发

Elizabeth E Evans1, Terrence L Fisher1, Vikas Mishra1

  • 1Vaccinex, Inc., Rochester, NY, USA.

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概括
此摘要是机器生成的。

塞马福林4D (SEMA4D) 阻断抗体pepinemab在治疗神经退行性疾病,如阿尔茨海默氏症,通过减少神经炎症和改善认知功能,显示出有前景. 临床试验表明,在早期阿尔茨海默病患者中,显著的认知效益和生物标志物改善.

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In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
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Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells
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科学领域:

  • 神经科学是一个神经科学.
  • 免疫学 免疫学 免疫学
  • 药理学 药理学是指药理学的学科.

背景情况:

  • 赛马福林4D (SEMA4D) 和它的受体涉及到反应性滑症和神经退行性疾病,如阿尔茨海默病 (AD) 和亨廷顿病 (HD).
  • 患病神经元中的SEMA4D蛋白上调激活星球细胞,导致代谢载体下调和炎症性细胞因子释放.
  • 之前的研究表明,用pepinemab阻断SEMA4D可降低星病生物标志物,并减缓HD患者的认知衰退.

研究的目的:

  • 在临床前模型中研究pepinemab对神经病理学,行为和血管完整性SEMA4D阻塞的影响.
  • 评估与轻度AD痴呆症患者治疗pepinemab相关的安全性,疗效和生物标志物变化.

主要方法:

  • 在临床前的研究中,使用了HD的小鼠模型和体外脑芯片模型来评估SEMA4D阻塞效应.
  • 第1b/2期SIGNAL-AD试验 (NCT04381468) 涉及50名患有轻度AD痴呆症的个体,他们接受了pepinemab或安慰剂治疗12个月.
  • 关键目标包括安全评估,使用标准尺度进行认知评估,并通过CSF O-link蛋白质组分析进行生物标志物分析.

主要成果:

  • 在临床前模型中,SEMA4D阻断减少了质激活,增加了突触标记物,并改善了认知缺陷.
  • 在SIGNAL-AD试验中,佩皮内马布治疗在轻度认知障碍早期AD小组中显示出耐受性和有意义的认知改善,认知衰退的减速超过70%.
  • 脑脊液分析显示,与pepinemab相关的生物标志物对反应性星球细胞,微质清除和病理的生物标志物减少.

结论:

  • 用pepinemab阻断SEMA4D有效降低神经炎症和相关的血管干扰.
  • 佩皮内马布通过向质细胞激活来证明其作为治疗认知功能障碍和神经退行性疾病的潜力.
  • 这些发现支持用于治疗神经退行性疾病的质调节剂的广泛应用,为抗粉样蛋白疗法提供替代或补充.