The National Health Surveillance Agency (Anvisa) authorized the Oswaldo Cruz Foundation (Fiocruz) to begin clinical trials on humans of GB221, an advanced gene therapy product aimed at treating Spinal Muscular Atrophy (SMA) Type 1 (SMA1), the most serious form of the disease.
The announcement was made during a meeting of the Executive Group of the Health Economic-Industrial Complex (Geceis) of the federal government, this Monday (24), in São Paulo. Approved quickly by Anvisa in a priority analysis process, the study positions Brazil as a leader on the topic in Latin America.
The GB221 therapy was developed by the US company Gemma Biotherapeutics, Inc. (GEMMABio). Fiocruz, in addition to participating in the clinical development of the therapy, signed a technology transfer agreement with the company, through the Institute of Immunobiological Technology (Bio-Manguinhos), paving the way for the national production of a gene therapy to be carried out for the first time.
The Fiocruz strategy for Advanced Therapies aims to provide the country with the scientific-technological, production and assistance bases necessary to make products available within the scope of the Unified Health System (SUS).
With the initiative, the Ministry of Health (MS) advances in national support for research and development of gene therapies, one of the most innovative frontiers in precision public health, with a focus on the SUS. The project aimed at developing gene therapies for rare diseases led by Fiocruz has already received investments of R$122 million from the Ministry of Health (MS). The Strategy also has financial support from the Financier of Studies and Projects (Finep), which invested resources of around R$50 million in infrastructure for the production of advanced therapies.
Since the production of the vaccine for Covid-19, Bio-Manguinhos/Fiocruz has mastered the viral vector technology used for this gene therapy.
The project is part of the Ministry of Health’s initiatives to strengthen the Health Economic-Industrial Complex (Ceis) and, at the same time, is part of the effort to expand the National Care Policy for People with Rare Diseases. By acting on these two fronts, the initiative strengthens the SUS with concrete technological development, which can overcome the challenges posed by new technologies to the financial sustainability of public health.
“As an institution that has worked for 125 years in defense of life, it is exciting for Fiocruz to put its scientific capacity at the service of children in our country. The clinical study that has just been authorized could transform the lives of children with spinal muscular atrophy in an unprecedented way using gene therapy techniques, a cutting-edge field in world science”, stated the president of Fiocruz, Mario Moreira.
The director of Bio-Manguinhos, Rosane Cuber, assessed that this partnership symbolizes a decisive step for the country.
“The inclusion of gene therapies in our portfolio represents a unique opportunity to strengthen Brazil’s scientific and technological sovereignty. We always act in line with regulatory and scientific rigor, because we know that each advance represents real hope for thousands of Brazilian families.”
Clinical trials
Considered rare and manifesting itself in the first months of life, Type 1 SMA is caused by an alteration in the SMN1 gene, responsible for producing a protein essential for the functioning of neurons related to movement. The absence of this protein causes the progressive loss of muscle strength and can compromise the survival of children in the first years of life.
