The company develops gene therapy drugs for the treatment of ophthalmological, neurological, cardiac, and other diseases, both genetic and multifactorial.
With monogenic genetic diseases, gene therapy involves treating the cause of the disease by adding a copy of the working gene to replace the mutant one and/or inhibiting the toxic mutant protein.
The main advantage of AAV-based gene therapy is its longevity. Therefore, gene therapy can also be used for multifactorial diseases, where pathogenetic mechanisms are known that can be compensated by adding or blocking a gene using gene therapy methods.