YC-001 is an inverse agonist and antagonist of rod opsin. YC-001 reversibly binds to rod opsin and stabilizes the structure of rod opsin. YC-001 protects mice from bright light-induced retinal degeneration. YC-001 may be used for studying retinal degeneration.

Physicochemical Properties

Molecular Formula	C12H7CLO2S2
Molecular Weight	282.765779733658
Exact Mass	281.957
CAS #	748778-73-6
PubChem CID	2377702
Appearance	White to yellow solid powder
Density	1.5±0.1 g/cm3
Boiling Point	494.2±45.0 °C at 760 mmHg
Flash Point	252.7±28.7 °C
Vapour Pressure	0.0±1.3 mmHg at 25°C
Index of Refraction	1.686
LogP	3.35
Hydrogen Bond Donor Count	0
Hydrogen Bond Acceptor Count	4
Rotatable Bond Count	2
Heavy Atom Count	17
Complexity	370
Defined Atom Stereocenter Count	0
InChi Key	RCLLWMPOHNZNAO-UHFFFAOYSA-N
InChi Code	InChI=1S/C12H7ClO2S2/c13-10-4-3-8(17-10)7-6-15-12(14)11(7)9-2-1-5-16-9/h1-5H,6H2
Chemical Name	3-(5-chlorothiophen-2-yl)-4-thiophen-2-yl-2H-furan-5-one
HS Tariff Code	2934.99.9001
Storage	Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month
Shipping Condition	Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

Biological Activity

ln Vitro	YC-001 (0-100 µM) exhibits an efficacy of 150–310% and a potency activity of 8.7 µM[1]. P23H opsin mutants exhibit improved glycosylation properties when treated with YC-001 (0.5, 1, 5, 10, 20, 40 µM) [1]. Rod photoreceptor proteins are reversibly bound by YC-001 (0,-1.5 µM), with an EC50 of 0.98 µM[1]. In NIH3T3 cells, YC-001 (0.313, 0.625, 1.25, 2.5, 5, 10, 20, 80 µM) raises cAMP levels in a dose-dependent way [1].
ln Vivo	Bright light-induced retinal degeneration in Abca4−/−Rdh8−/− mice is prevented by intraperitoneal injection of YC-001 (50, 200 mg/kg) [1]. After systemic administration, mice receive an intraperitoneal injection of YC-001 (200 mg/kg), which enters their eyes but does not stay there for very long [1]. YC-001 (intraperitoneal injection, 100 mg/kg, 200 mg/kg; once daily for 24 days) exhibited almost no acute toxicity [
Cell Assay	Western Blot Analysis[1] Cell Types: NIH3T3 Cell Tested Concentrations: 0.5, 1, 5, 10, 20, 40 µM Incubation Duration: Experimental Results: Improved glycosylation profile of P23H opsin.
Animal Protocol	Animal/Disease Models: 6weeks old Abca4 −/−Rdh8−/− mice [1] Doses: 50, 200mg/kg Route of Administration: IP Experimental Results: Protection of Abca4−/−Rdh8−/− mice from strong light-induced Retinal degeneration. Animal/Disease Models: C57BL/6 mice [1] Doses: 200 mg/kg Route of Administration: IP Experimental Results: After systemic administration, it enters the eyes of mice but does not remain for a long time. Animal/Disease Models: C57BL/6 mice[1] Doses: 100 mg/kg, 200 mg/kg Route of Administration: intraperitoneal (ip) injection; one time/day for 24 days Experimental Results: No acute toxicity was shown.
References	[1]. A novel small molecule chaperone of rod opsin and its potential therapy for retinal degeneration. Nat Commun. 2018 May 17;9(1):1976.

Solubility Data

Solubility (In Vitro)

DMSO : ~100 mg/mL (~353.64 mM)

Solubility (In Vivo)

Solubility in Formulation 1: ≥ 2.5 mg/mL (8.84 mM) (saturation unknown) in 10% DMSO + 40% PEG300 + 5% Tween80 + 45% Saline (add these co-solvents sequentially from left to right, and one by one), clear solution. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 25.0 mg/mL clear DMSO stock solution to 400 μL PEG300 and mix evenly; then add 50 μL Tween-80 to the above solution and mix evenly; then add 450 μL normal saline to adjust the volume to 1 mL. Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH₂ O to obtain a clear solution. Solubility in Formulation 2: ≥ 2.5 mg/mL (8.84 mM) (saturation unknown) in 10% DMSO + 90% Corn Oil (add these co-solvents sequentially from left to right, and one by one), clear solution. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 25.0 mg/mL clear DMSO stock solution to 900 μL of corn oil and mix evenly.  (Please use freshly prepared in vivo formulations for optimal results.)

Preparing Stock Solutions		1 mg	5 mg	10 mg
	1 mM	3.5364 mL	17.6822 mL	35.3644 mL
	5 mM	0.7073 mL	3.5364 mL	7.0729 mL
	10 mM	0.3536 mL	1.7682 mL	3.5364 mL

*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.