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Biocad plans to investigate the first drug in the Russian Federation for the treatment of spinal muscular atrophy

Biocad plans to investigate the first drug in the Russian Federation for the treatment of spinal muscular atrophy

The Russian biotech company Biocad has sent documents to the Ministry of Health of the Russian Federation for permission to conduct a clinical trial of a drug for the treatment of spinal muscular atrophy (SMA). This was reported to the editorial board of "Made in Russia" by the Biocad press service.

It is planned to include children with SMA under the age of 8 months and the onset of the disease under the age of 6 months in clinical trials.

Today, a drug with a similar effect of Zolgensma is the most expensive in the world and costs $ 2,125 million.

"Based on the completed preclinical studies of the drug and experimental data obtained on animals, an effective and safe dose for the first administration to patients was determined," the press service said.

This therapy is pathogenetic. It is expected that one injection of the drug will be enough. The introduction of the drug will ensure the delivery of the functional SMN1 gene and the production of the SMN protein, the lack of which underlies the development of motor skills development disorders in children with spinal muscular atrophy. Zolgensma has a similar effect: this drug is by far the most expensive in the world, one injection costs about 2,125 million dollars.

As part of phase 1-2 clinical trials, data on the safety and efficacy of the drug in children with SMA will be obtained. It is planned to connect research centers in Moscow, St. Petersburg, Yekaterinburg and other cities.

Biocad has invested more than 4 billion rubles in the development and preclinical studies of the first Russian gene therapy drug for the treatment of SMA.

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Author: Ekaterina Ivanova
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