For the first time in the world, CRISPR Prime Editing technology will be tested in SMA disease
1For the first time in the world, CRISPR Prime Editing technology will be tested in SMA disease
Üsküdar University Transgenic Cell Technologies and Epigenetics Application and Research Center (TRGENMER) tried CRISPR Prime Editing gene editing technology in SMA disease for the first time in the world. TRGENMER Director Dr. Lecturer Cihan Taştan stated that with this study, they increased the production of SMN protein, which is low in SMA Type 1 patients, and noted that they aim for a clinical phase trial at the end of 2023. TRGENMER, which carries out studies to produce similar and inexpensive drugs in the national drug concept to be used in the treatment of rare diseases, especially SMA, announced that SMA gene therapy studies are progressing positively.
'SYMPTOMS CAN REDUCE'
TRGENMER Director Dr. Lecturer Cihan Taştan stated that for the first time in the world, CRISPR Prime Editing technology was tested in SMA disease and said, “We proved that we can reverse the low SMN protein production that causes SMA disease, with the gene engineering we have done in the SMN2 gene, and increase the production of SMN protein. In the past weeks, we have also published our preliminary reports showing that our SMA gene therapy studies based on CRISPR Gene Editing Technology, for which the 2020 Nobel Prize was won, are progressing positively.” Taştan continued his words as follows: “While SMN protein production is approximately 10 percent in SMA type 1 patients, we increased this rate to 30 percent with CRISPR and 75 percent with AAV gene therapy in the controlled experiments we established in the laboratory, which are very good rates for reducing the symptoms of SMA disease. ”
'WE ARE TARGETING THE END OF 2023'
Cihan Taştan said that they aim to start testing SMA gene therapy approaches in SMA animal models by the end of 2022, which are structurally similar to two different genetic therapy drugs Zolgensma and Spinraza, which are currently FDA-approved and available for sale, and even provide more permanent and long-term treatment. Stating that they aim to switch to clinical phase trials in children with SMA by the end of 2023, Taştan noted that they made a project application to the Turkish Health Institute Presidency (TÜSEB).