Description

Emixustat Hydrochloride is a potent and selective small molecule inhibitor of retinal pigment epithelium-specific 65 kDa protein (RPE65), a key enzyme in the visual cycle. This compound is of significant interest for its potential therapeutic application in the treatment of retinal degenerative diseases, such as age-related macular degeneration (AMD) and Stargardt disease. It is primarily utilized by pharmaceutical research organizations, biotechnology companies, and academic institutions engaged in ophthalmic drug discovery and development.

Application

• Ophthalmic Drug Development: A key investigational agent for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
• Retinal Disease Research: Used as a pharmacological tool to study the visual cycle and the role of RPE65 in preclinical models of retinal disorders.
• Biochemical Assays: Serves as a reference standard in high-throughput screening (HTS) campaigns to identify novel RPE65 modulators.
• Mechanism of Action Studies: Employed to investigate the pathophysiology of inherited retinal dystrophies and validate therapeutic targets.
• Active Pharmaceutical Ingredient (API): Used in the formulation development of novel ophthalmic therapeutics under current Good Manufacturing Practice (cGMP) conditions.

Basic Information

Product Name	Emixustat Hydrochloride
CAS No.	1141934-97-5
Molecular Formula	C24H21F3N2O3 • HCl
Molecular Weight	485.89 g/mol (free base: 450.43 g/mol)
Synonyms	ACU-4429 Hydrochloride; Emixustat HCl; (2R)-2-[(1E)-1-(3,5,5,8,8-Pentamethyl-5,6,7,8-tetrahydronaphthalen-2-yl)-3-(trifluoromethyl)prop-1-en-1-yl]piperidine-1-carboxylic acid hydrochloride; RPE65 Inhibitor ACU-4429; ALK-001 (Hydrochloride salt); Zimura precursor (related compound).
EINECS	Contact for details

Quality Control

Our Emixustat Hydrochloride is manufactured and tested under a strict quality management system. Each batch is subjected to comprehensive analytical testing, including identification, purity assay, and impurity profiling, to ensure it meets the stringent requirements for pharmaceutical research. A Certificate of Analysis (COA) detailing the results for identity, purity (by HPLC), residual solvents, and related substances is provided with every shipment to guarantee traceability and batch-to-batch consistency.

Storage

Preserve in a tightly closed container, protected from light. Store in a cool, dry place at a controlled room temperature (15-25°C). This material is hygroscopic (moisture-sensitive) and should be handled under dry conditions to maintain stability and purity.

Specification

Item	Specification
Appearance	White to off-white solid
Identification (IR)	Conforms to structure
Identification (HPLC)	Retention time matches reference standard
Purity (HPLC)	≥98.0%
Water Content (KF)	≤1.0%
Residual Solvents (GC)	Meets ICH Q3C guidelines
Heavy Metals	<20 ppm
Assay (Titration)	97.0% - 103.0% (on anhydrous basis)

Note: Specifications can be tailored. Please contact us for the detailed technical data sheet of a specific grade.