R-Pharm group and Kazan Federal University (KFU) signed an agreement on the development of an innovative gene therapy drug for the treatment of spinal muscular atrophy (SMA). The basis of the future drug is an adeno-associated viral vector, which will deliver a functional copy of the SMN1 gene to motor neurons. This will stimulate production of the specific protein in sufficient quantities, which will prevent degeneration and death of neurons characteristic of SMA.
“Since the beginning of 2023, SMA has been included in the expanded neonatal screening program. This makes it possible to detect the disease in a timely manner and begin treatment with the latest generation of drugs. The development of Kazan scientists gives children with SMA hope for normal development, and the agreement with KFU is a step towards increasing the availability of innovative drugs in Russia,” said Anastasia Batrak, R-Pharm’s Vice President for Strategic Marketing and Product Portfolio Development.
It is expected that the drug developed by KFU will cost less than the therapies for SMA offered by foreign manufacturers. According to Albert Rizvanov, head of the Center for Excellence in Personalized Medicine at the Institute of Fundamental Medicine and Biology of the KFU, this will not only make the treatment of SMA in Russia more affordable, but will also relieve the burden on the national health system, “as it will reduce the number of patients in need of long-term care and support.”
Previously, a therapy for SMA was developed by Biocad. In 2022, the company received authorization for trials from the Ministry of Health. Forty patients take part in the tests in five Russian medical organizations.