Alexey Ponomaryov’s gene therapy project for ichthyosis treatment wins provincial award
Tatarstan’s Republican Contest of Innovative Ideas and Research of Young Scientists in Medicine admitted participants from 18 to 35 years of age.
Overall, 36 papers were submitted for consideration from Kazan Federal University, Kazan State Medical University, Kazan State Medical Academy, Interregional Clinical Diagnostics Center, Emergency Medicine Hospital, Republican Clinical Oncological Hospital, and Republican Children’s Clinical Hospital. In the finals, where there were 13 participants, the contestants were invited to a face-to-face meeting with experts. Among the most popular topics were oncology, genetics, drug delivery, cardiology, and endocrinology.
Chairman of the jury, Head of the Center for Personalized Medicine, Professor of KFU Albert Rizvanov shared, “It’s cool to see people who want to change the world for the better.”
He opined that the level of finalists was very high, “As for KFU representatives, the finalists mostly dealt with genetics and gene therapy of orphan diseases and injuries of the nervous system. All works were of applied nature.”
The evaluation criteria were research level, prospects of market implementation, and qualifications of an applicant.
The expert commission decided to award the first place and 150 thousand rubles in prize money to 3rd year PhD student Alexey Ponomaryov for his project ‘Development of gene therapy for the treatment of autosomal recessive congenital ichthyosis’.
As the winner explained, ARCI is a severe untreatable congenital syndrome stemming from a mutation in the TGM1 gene and leading to a deficiency of the eponymous enzyme in epidermis. The syndrome leads to defects in skin growth and its barrier function.
“As of now, the therapy of lamellar ichthyosis is constant moisturizing and disinfection of skin. There are 3 thousand patients with ARCI in Russia and 80 thousand in the world,” said Ponomaryov.
His project is now in preclinical trials, “I plan to research cell cultures before end of year. Next year, we will study models of artificial epidermis and lab animals. If successful, this not only leads to the production of a unique gene drug without direct competitors, but also opens doors for gene therapy studies of other skin ailments.”
The prize funding will be disbursed to reagents for preclinical trials of the recovery of transglutaminase on cell cultures and animal tissues. Ponomaryov is also one of the winners of the Student Startup competition and plans to apply for the Start 1 program and Russian Science Foundation projects.
Other finalists from KFU were:
Aliya Yakubova, ‘Development of a genetic test based on PCR analysis of single-nucleotide polymorphism of rs8065080 of the gene TRPV1 for preventive diagnostics of chronic migraine and therapy choice’;
Anna Timofeeva, ‘Neuroprotective gene medication for personalized medicine’;
Rezeda Shigapova, ‘Development of adeno-associated viruses tropic to different populations of neurons for target optogenetic stimulation of neuroplasticity’.