KNA-123

KNA-123 is an experimental gene therapy developed by Kinea Bio for the treatment of Duchenne muscular dystrophy (DMD). The therapy utilizes two viral vectors, each encoding a portion of the midi-dystrophin protein fused to inteins. Once delivered into muscle cells, these intein-tagged protein fragments are joined together to form a full-length midi-dystrophin protein. This approach enables delivery of a larger and potentially more effective version of dystrophin compared to micro-dystrophin therapies. Preclinical studies in mice have demonstrated that this strategy can achieve superior efficacy at lower doses than existing mini-dystrophin approaches, suggesting potential for improved outcomes in DMD patients[1][2].

Other names

midi-dystrophindual-AAV midi-dystrophin gene therapyKinea Bio midi-dystrophin