Gene Editing Technology Center (NuTEG)

Gene therapy is an innovative treatment and one component of what are known as advanced therapy products (ATP). For more than a decade this area has been evolving worldwide, bringing promising advances in the treatment of human diseases.

In late 2023, regulatory agencies in the US and UK approved the first CRISPR/Cas9-based gene therapy (CASGEVY™), to treat hematologic diseases such as sickle cell anemia and beta thalassemia. Today this treatment has shown excellent results and is expanding globally.

In Brazil, ANVISA has already approved 9 gene therapy products, including Zolgensma® (to treat spinal muscular atrophy, SMA), Kymriah® (for some types of leukemia and lymphoma), Roctavian® (for hemophilia A) and Luxturna® (for hereditary retinal dystrophy). In 2025, Brazil’s Unified Health System (SUS) treated two Brazilian patients with Zolgensma® for the first time.

The LNBio Gene Editing Technology Center is using advanced technologies like CRISPR/Cas9, prime editing and base editing to develop gene editing protocols in order to treat human diseases that do not yet have cures. The team has also been working to optimize encapsulation and delivery of these advanced therapy products (ATP) to develop safer and more effective treatments.