Shanghai Yuli Biotechnology CO.,LTD.
Eteplirsen

CAS: 1173755-55-9

Purpose: Treats exon 51 deletion in dystrophin, for DMD

Formula: C364H569N177O122P30

Eteplirsen
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Product description
Avacincaptad Pegol is a nucleic acid aptamer active pharmaceutical ingredient (API) that selectively targets complement component C5. It is a next-generation ophthalmic therapeutic featuring long-acting efficacy, high target specificity, and low immunogenicity. Unlike conventional anti-inflammatory therapies that rely on broad-spectrum suppression of inflammatory pathways, Avacincaptad Pegol precisely binds to the active site of complement C5, blocking complement cascade activation at its source and preventing the generation of the inflammatory mediators C5a and C5b. This targeted mechanism effectively suppresses chronic retinal inflammation and slows progressive retinal atrophy while offering excellent ocular biocompatibility, minimal off-target effects, and sustained therapeutic activity.
The molecule is optimized through PEGylation to overcome the rapid degradation and short plasma half-life commonly associated with nucleic acid therapeutics, making it well suited for long-acting local ocular administration. Manufactured using a proprietary site-specific modification and precision synthesis process, the product achieves a purity of ≥98%, with trace synthetic by-products and residual impurities strictly controlled in accordance with pharmacopeial and ICH quality standards. It provides outstanding batch-to-batch consistency, stable molecular conformation, and highly reproducible target-binding activity, fully meeting the quality requirements for the development and large-scale manufacture of advanced long-acting ophthalmic biologics.
Manufactured on standardized GMP-compliant production lines, the product can be supplied with a complete Certificate of Analysis (COA), comprehensive impurity profiles, and DMF documentation to support research, process development, regulatory submission, and commercial manufacturing of complement-targeted ophthalmic therapeutics, including intravitreal injection formulations and treatments for degenerative retinal diseases.
By selectively inhibiting complement C5, the product effectively suppresses excessive complement-mediated inflammation in the retina, protects retinal photoreceptor cells, delays macular tissue degeneration and atrophy, and helps maintain retinal physiological homeostasis. It combines precise complement pathway inhibition, ocular anti-inflammatory activity, retinal cell protection, and long-term intervention for degenerative retinal disorders, making it a high-quality pharmaceutical intermediate for the development of innovative therapies targeting geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and other complement-mediated retinal diseases.
This product is intended solely as a pharmaceutical and biopharmaceutical intermediate and is not intended for direct human use.
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