Pharmaceutical Companies Unite to Advance Nucleotide-Based Medicines

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Scientists work together to create more effective medicines

Advances continue to progress personalised medicine, a field in which drugs are tailored to patients’ needs based on their genetic information. Historically, simply screening patients’ genomes enabled this trend but more recently, synthetic nucleotide-based medicines have taken this a step further. Nucleotide-based medicines use artificial DNA and RNA (nucleotides) to target cellular genetic information directly, shutting off genetic diseases at their source. Mipomersen, for example, an RNA based drug, binds to cellular messenger RNA, preventing RNA from allowing the synthesis of malfunctioning proteins in sufferers of a genetic disorder that causes high cholesterol.

Recognising the potential of nucleotide-based medicines, the rapidly growing pharmaceutical giant Biogen has recently acquired a large proportion of Ionis. Ionis is a small specialist company that developed Vitravene, the first RNA drug, in 1998. Ionis researchers have often utilised their knowledge to provide drugs for diseases where little success has been found before. They developed the drug ‘Spinraza’—the first available treatment for spinal muscular atrophy. As of the 5th of June, Biogen and Ionis finalised a deal to collaborate and investigate treatments for further neurological diseases. These include dementia and other neuromuscular diseases for which notoriously few drugs are currently available. Concurrent with this deal, are academic improvements of nucleotide synthesis methods and the approval of Alnylam’s Onpattro, the first RNAi drug (a branch of nucleotide-based medicines).  Working together, scientists and investors may make this field of personalised medicine fruitful in the near future.

Matthew Harris is a student at the University of Cambridge. Image: pixabay