Oligonucleotides: Synthesis and Manufacturing Hurdles
November 18, 2025
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6 min
Oligonucleotides are transforming medical treatment by enabling precision therapies that target disease at the genetic level. Unlike traditional drugs, these therapies can modify genes specifically relevant to individual patient profiles, marking a major advancement in precision medicine. With the first FDA-approved oligonucleotide therapy achieved in 2018, applications have rapidly developed, including treatments for LDL cholesterol reduction and rare genetic disorders. Challenges remain, particularly in manufacturing and cost-efficiency, necessitating collaboration between researchers and industry for future scalability and broader application.
1. Oligonucleotides work at the genetic level to treat diseases. 2. First FDA-approved therapy: Onpattro (2018). 3. Major applications include gene silencing and modification. 4. Challenges include manufacturing complexity and cost. 5. AI and bioinformatics enhance drug design. 6. Milasen exemplifies personalized oligonucleotide therapy. 7. Hydrophilic interaction liquid chromatography is a key analysis method. 8. Collaboration is essential for advancing oligonucleotide therapies.
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