P-Pharm: Product for spinal muscular atrophy

Main article: Spinal muscular atrophy (SMA)

2023: Drug Development Agreement

R-Pharm Group and Kazan Federal University announced the signing of an agreement on the development of an innovative gene therapy drug. According to the agreement, scientists from KFU will develop a drug for the treatment of spinal muscular atrophy, the customer of which is the R-Pharm group of companies, which announced this on May 11, 2023.

This drug is based on the use of adeno-associated virus as a vector to deliver a functional copy of the SMN1 gene to motor neurons. Due to this, the specific protein begins to be produced in sufficient quantities capable of preventing degeneration and death of neurons, which can provide a significant improvement in the condition of patients.

Anastasia Batrak, Vice President for Strategic Marketing and Product Portfolio Development at R-Pharm, noted that innovative drugs are one of the company's priorities.

SMA is a severe disease that has a negative impact on the quality of life of patients and their families. Since the beginning of 2023, SMA has been included in the expanded neonatal screening program. This allows you to detect the disease in a timely manner and start 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.

Albert Rizvanov, head of the Center for the Superiority of Personalized Medicine at the Institute of Fundamental Medicine and Biology of KFU, also noted that SMA belongs to orphan diseases. Despite the fact that the incidence of orphan diseases is low, in general there are more than 7,000 such diseases, and together they affect 6-8% of the world's population.

The development of CFCs has the potential to reduce the cost of SMA treatment, making it more accessible to more patients. In addition, the drug will help relieve the health care system, as it will reduce the number of patients in need of long-term care and support. It is important to note that the successful implementation of the agreement between R-Pharm and Kazan Federal University will be an example of effective cooperation between academic science and industry in the development of innovative medicines for the treatment of rare diseases, "said Albert Rizvanov.