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mRNA Therapy

mRNA Therapy

How does RNA therapy function?

RNA therapy operates by targeting and correcting mutations within the RNA molecules of individuals affected by a particular disease. These mutations can disrupt the production of essential proteins needed for normal cellular functions. By addressing these mutations at the RNA level, the therapy aims to enable the cell to produce the required proteins, thereby addressing the underlying cause of the disease.

Why focus on RNA therapies?

RNA therapies offer a significant advantage in their ability to address a wide range of diseases and disorders, particularly those resulting from genetic mutations or deficiencies in protein production. Unlike traditional treatments that may only alleviate symptoms, RNA therapies have the potential to correct the fundamental molecular abnormalities driving the disease process. This broader applicability makes RNA therapies an attractive avenue for medical research and treatment development, with promising implications for improving patient outcomes across various conditions.

What exactly is RNA?

RNA, or ribonucleic acid, serves as a critical intermediary in the process of protein synthesis within cells. Unlike its counterpart, DNA, which stores genetic information, RNA carries out instructions encoded in the DNA to produce specific proteins essential for various cellular functions. Through a process known as transcription, RNA molecules are synthesized based on the genetic code contained within DNA. These RNA molecules then undergo translation, where they guide the assembly of amino acids into functional proteins according to the encoded instructions.

Antisense oligonucleotides

Sepulbio’s RNA therapies belong to a class of compounds known as antisense oligonucleotides (AONs) or ASO treatments. These therapies utilize short, synthetically designed RNA sequences that are chemically modified to enhance their stability and cellular uptake. By precisely targeting specific RNA sequences associated with disease-causing genes, AONs can modulate gene expression and correct aberrant protein production. This approach has demonstrated efficacy in treating various genetic disorders, offering a promising avenue for personalized medicine and targeted therapeutics. Several RNA therapies utilizing antisense oligonucleotides have already received regulatory approval and are being used to treat patients, underscoring the growing significance of this innovative therapeutic approach in modern medicine.