The Approved Drug: Pegcetacoplan (Empaveli/Syfovre) This is actually a single aptamer molecule with two distinct FDA approvals for different diseases: Paroxysmal Nocturnal Hemoglobinuria (PNH): Approved in 2021 under the brand name Empaveli. It is a complement C3 inhibitor used to treat adults with PNH. Geographic Atrophy (GA): Approved in 2023 under the brand name Syfovre. It is used to treat GA secondary to age-related macular degeneration (AMD). How it works: Pegcetacoplan is a pegylated (attached to a polyethylene glycol chain for longer circulation) aptamer that binds specifically to complement protein C3. By inhibiting C3, it suppresses a central part of the body's complement immune system, which is overactive in both PNH and GA. Important Context and Other Notable Aptamers First-Ever Approval: The first aptamer therapeutic ever approved was Pegaptanib (Macugen) in 2004 for wet AMD. It targets VEGF. While it was a pioneering drug, it has since been largely superseded by more effective antibody-based treatments (like ranibizumab and aflibercept). It is still approved but rarely used. International Approvals: Another significant aptamer is Avacincaptad pegol (Izervay), which was also FDA-approved for Geographic Atrophy in 2023. It works by inhibiting complement C5. Aptamers in Diagnostics: While therapeutic aptamers are rare, aptamers are widely used in research and diagnostic tools. A famous example is the SOMAscan platform, which uses thousands of Slow Off-rate Modified…
Aptamers are short, single-stranded DNA or RNA sequences that fold into 3D shapes capable of binding specific targets—proteins, small molecules, ions, cells, or even complex mixtures—with high affinity and selectivity. Because they are chemically synthesized, readily modified, and often less immunogenic than protein binders, aptamers have matured into a versatile “molecular toolkit” used across diagnostics, biosensing, therapeutics, imaging, and bioprocessing. This article explains APTAMER APPLICATIONS from fundamentals to advanced use-cases, with an emphasis on how teams translate an aptamer sequence into a functioning assay, sensor, drug carrier, or imaging probe. 1) How Aptamers Are Created (Why Selection Method Shapes Applications) Most aptamers are discovered through SELEX (Systematic Evolution of Ligands by EXponential enrichment): iterative rounds of binding, separation, and amplification that enrich sequences best suited to a chosen target and conditions. Modern SELEX variants—such as cell-SELEX, microfluidic SELEX, and capillary electrophoresis SELEX—aim to shorten selection time, improve specificity, and better match real-world sample environments. The practical result is that application performance often depends as much on selection constraints (buffer, temperature, counter-selection targets, matrix effects) as on the final nucleotide sequence. Key takeaway: If the intended application involves serum, saliva, food extracts, or environmental water, designing SELEX conditions to…
