GenAssist Therapeutics successfully completed a virtual meeting with the TREAT-NMD Advisory Committee for its first base editing product on September 29, 2023. Eighteen representatives, including distinguished preclinical and clinical experts in Duchenne Muscular Dystrophy (DMD) therapy, as well as DMD representatives attended this meeting. A comprehensive review and advice were received on November 13 after this meeting.
We appreciated the valuable advice and comprehensive thinking from TACT. TACT experts provide recommendation for further preclinical studies and opportunities to formally interact with the patient community. In addition, they also appreciate several appealing aspects of the base editing drug and commended us for the active engagement with regulators. Finally, the committee provide guidance for clinical trial initiation, such as outcome measurement and multinational clinical trials.
Their valuable advice will help us to further optimize our design and provide safer and more effective drug to DMD patients.
Here is the non-confidential summary for the TREAT-NMD website: https://www.treat-nmd.org/virtual-treat-nmd-advisory-committee-for-therapeutics-meeting-held-online/
