Suzhou GenAssist Therapeutic Co.,Ltd recently announced its pre-IND application of their first base editing product, GEN6050 and the acceptance by the FDA.

GEN6050 is an in vivo base editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene. It is designed to restore the expression of dystrophin protein by base editing.

DMD is a rare disease with a high incidence rate, with one DMD patient diagnosed per 4000 newborn boys. According to LEIDEN DMD data, exon 50 skipping can be used to treat 4% of DMD patients with large deletions ^[1]. Nowadays, DMD patients are often treated with marketed drugs such as glucocorticoids and ASOs, which target exon skipping at exon 45, 51, and 53. Until now, there is no approved drug targeting exon 50 skipping in the dystrophin gene, even in clinical stage. In addition, no AAV-delivered in vivo base editing drugs for any other indication have entered clinical trials globally. As one of the pioneering companies developing in vivo base editing drugs, Suzhou GenAssist Therapeutic has already established a comprehensive evaluation system for base editing drugs. Moreover, the Company is developing base editing products for more DMD mutations.

The Pre-IND meeting usually refers to a voluntary meeting where the sponsor submits and communicates with the regulatory authority before conducting clinical trials of investigational new drugs (IND). The main purpose of the meeting is to review pharmacological study and non-clinical study, and to discuss whether existing research results are sufficient to support clinical research.