Recently, Suzhou GenAssisit Therapeutics Co.,Ltd. (hereinafter referred to as “GenAssist”) announced that its first base editing product, GEN6050, has submitted a pre-investigational new drug (Pre-IND) application to the FDA and has been accepted.
GEN6050 is an in vivo base editing drug targeting exon 50 skipping for the treatment of Duchenne Muscular Dystrophy (DMD). The drug aims to achieve exon 50 skipping through base editing, restoring the expression of dystrophin that a protein is associated with muscle atrophy. DMD is a rare disease with a relatively high incidence, affecting one in every 4,000 newborn boys. According to LEIDEN DMD data, exon 50 skipping can be used to treat 4% of DMD patients with large segment deletions [1]. Currently, the approved drugs for DMD include glucocorticoids and antisense oligonucleotide (ASO) drugs targeting exon skipping of 45, 51, and 53. There is currently no drug in clinical development specifically targeting exon 50 skipping for dystrophin.
Moreover, as of now, there are no in vivo base editing drugs for other indications that have entered clinical trials globally. As one of the earliest companies engaged in the development of in vivo base editing drugs, GenAssist has established a comprehensive in vivo and in vitro evaluation system for base editing drugs and is developing a series of products for DMD.
A Pre-IND meeting typically refers to a voluntary meeting where the applicant communicates with the regulatory authority before initiating clinical trials (Investigational New Drug – IND application). The main purpose of the meeting is to review CMC and non-clinical trial studies and discuss whether existing study results are sufficient to support the initiation of clinical studies.
About GenAssist
GenAssist is a globally leading innovative pharmaceutical research and development company, founded in July 2020. The company is headquartered in Suzhou, China, with subsidiaries in Shanghai, China, and Boston, USA. GenAssist holds the global rights to the TAM base editor technology, which originates from the Institute of Nutrition and Health of the Chinese Academy of Sciences and is licensed by Westlake University. The mission of GenAssist is to transform cutting edge science into products that benefit patients.
