Bioactivity | AT-007 is an orally active central nervous system (CNS) penetrant Aldose Reductase inhibitor for treatment of Galactosemia with an IC50 value of 100 pM. AT-007 reduces toxic galactitol levels and prevents disease complications in GALT deficiency rats[1][2]. | ||||||||||||
Target | IC50: 100 pM (Aldose Reductase) | ||||||||||||
In Vivo | AT-007 显着降低了 GALT 缺失大鼠的脏器组织(肝,脑和血浆)中半乳糖醇水平,但没有增加半乳糖或 Gal1P 的水平[2]。 | ||||||||||||
Name | AT-007 | ||||||||||||
CAS | 2170729-29-8 | ||||||||||||
Formula | C17H10F3N3O3S2 | ||||||||||||
Molar Mass | 425.40 | ||||||||||||
Appearance | Solid | ||||||||||||
Transport | Room temperature in continental US; may vary elsewhere. | ||||||||||||
Storage |
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Reference | [1]. Maeve Conneighton, et al. Applied Therapeutics Announces Initiation of Phase 1/2 Study of AT-007 in Galactosemia. [2]. UNITED STATES SECURITIES AND EXCHANGE COMMISSION. APPLIED THERAPEUTICS, INC. |