Brazil’s drug authority Anvisa has authorized the research foundation Fiocruz to begin clinical trials in humans with GB221, an advanced gene therapy product for the treatment of type-1 spinal muscular atrophy (SMA1), the most severe form of the disease.

Quickly approved by Anvisa under priority review, the study positions Brazil at the forefront of the field in Latin America. 

The GB221 therapy was developed by the US company Gemma Biotherapeutics, Inc. In addition to participating in the clinical development of the therapy, Fiocruz signed a technology transfer agreement with the company, paving the way for the unprecedented Brazilian production of a gene therapy.

The foundation’s strategy for advanced therapies aims to ensure that the country has the scientific and technological foundations to offer products to the Brazilian public health care network, the SUS.

With this initiative, the Brazilian Ministry of Health is increasing national support for research and development of gene therapies, one of the most innovative frontiers in precision public health care, with a focus on the SUS.

The project led by Fiocruz has received investments of BRL 122 million from the Ministry of Health. The strategy also has financial support from the Brazilian Innovation Agency (Finep), which has invested BRL 50 million in infrastructure for the production of advanced therapies.

Type-1 SMA

Considered rare and manifesting in the first months of life, type-1 SMA is caused by a mutation in the SMN1 gene, which is responsible for producing a protein essential for the functioning of motor neurons. 

The absence of this protein causes progressive muscle weakness and can compromise the survival of children in the first years of life.