Russia has synthesized oligonucleotides for the treatment of SMA

Scientists at the Institute of Chemical Biology and Basic Medicine (ICBPM) of the Siberian Branch of the Russian Academy of Sciences have synthesized DNA fragments for the treatment of spinal muscular atrophy (SMA).

SMA is a rare genetic disease characterized by the development of progressive muscle weakness, which over time leads to skeletal destruction and respiratory failure. In Russia, 55 children were born with SMA in 2023. There are only two drugs available in the world to treat SMA. One - domestic - needs to be injected into the patient's spinal cord every three months. The cost of one dose is more than $100,000. The cost of the other, foreign, drug is $1.3 million.

Novosibirsk scientists managed to reduce the number of doses required. For this purpose, they synthesized 18 therapeutic nucleotides, which have shown their stability. Scientists are already ready to cooperate with large pharmaceutical companies. In the future, a drug based on their method of nucleotide synthesis may be used to treat such complex and rare diseases as Duchenne and Becker muscular dystrophies, age-related muscular degeneration, acute hepatic porphyria, familial chylomicronemia syndrome, hereditary amyloid polyneuropathy, and others.