Givosiran

• 常用名: Givosiran
• 英文名: Givosiran
• CAS号: 1639325-43-1
• 分子量: 1710.994
• 密度: 1.3±0.1 g/cm3
• 沸点: 1780.6±65.0 °C at 760 mmHg
• 分子式: C₇₈H₁₃₉N₁₁O₃₀
• 熔点: N/A
• 闪点: 1030.6±34.3 °C

Givosiran用途

Givosiran(ALN-AS1)是一种针对肝脏氨基乙酰丙酸合成酶1(ALAS1)信使RNA的小干扰RNA。Givosiran下调ALAS1 mRNA并防止神经毒性δ-氨基乙酰丙酸和疏孔素原水平的积累。Givosiran可用于急性间歇性卟啉症的研究[1][2]。

Givosiran名称

• 英文名: 21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide

Givosiran生物活性

• 描述: Givosiran(ALN-AS1)是一种针对肝脏氨基乙酰丙酸合成酶1(ALAS1)信使RNA的小干扰RNA。Givosiran下调ALAS1 mRNA并防止神经毒性δ-氨基乙酰丙酸和疏孔素原水平的积累。Givosiran可用于急性间歇性卟啉症的研究[1][2]。
• 相关类别:
  信号通路 >> 其他 >> 其他
  研究领域 >> 神经疾病
• 体外研究: Givosiran与三价N-乙酰半乳糖胺配体结合,该配体与去唾液酸糖蛋白受体特异性结合,从而实现向肝细胞的靶向递送[2]。
• 参考文献:

  [1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339.

  [2]. Balwani M, et, al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301.

Givosiran物理化学性质

• 密度: 1.3±0.1 g/cm3
• 沸点: 1780.6±65.0 °C at 760 mmHg
• 分子式: C₇₈H₁₃₉N₁₁O₃₀
• 分子量: 1710.994
• 闪点: 1030.6±34.3 °C
• 精确质量: 1709.968872
• LogP: -4.24
• 蒸汽压: 0.0±0.6 mmHg at 25°C
• 折射率: 1.579

Givosiran英文别名

• 21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy ]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide
• 1-Pyrrolidinedodecanamide, N-[2-[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]-1,1-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-gala ctopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]ethyl]-4-hydroxy-2-methyl-λ-oxo-, (2R,4R)-