RECTAS-2.0

• Description:

  RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, used for the research of Fabry disease[1].

• UNSPSC:

  12352005

• Target:

  DNA/RNA Synthesis

• Related Pathways:

  Cell Cycle/DNA Damage

• Field of Research:

  Metabolic Disease

• Smiles:

  ClC1=NC(C(OC[C@H]2CN(C)C(O2)=O)=NC=C3)=C3C(NCC4=CC=CO4)=C1

• Molecular Formula:

  C18H17ClN4O4

• Molecular Weight:

  388.80

• References & Citations:

  [1]Tomonari Awaya, et al. Invention of an oral medication for cardiac Fabry disease caused by RNA mis-splicing. Sci Adv. 2025 Apr 11;11 (15) :eadt9695.

• Shipping Conditions:

  Room temperature

• Scientific Category:

  Reference compound1

• Clinical Information:

  No Development Reported

• CAS Number:

  [3038234-95-3]