rdGCM-102

rdGCM-102 is a preclinical gene therapy candidate developed by Gencellmed for the treatment of Stargardt disease, a form of inherited retinal dystrophy. The therapy utilizes a replication-defective Herpes Simplex Virus type 1 (HSV-1) viral vector to deliver a functional copy of the therapeutic gene to the retina. The choice of an HSV-1 vector is strategic, as it possesses a significantly larger packaging capacity than conventional adeno-associated virus (AAV) vectors, allowing for the delivery of large genes such as *ABCA4*, which is the primary gene associated with Stargardt disease. rdGCM-102 aims to restore the function of the ATP-binding cassette transporter protein, thereby preventing the accumulation of toxic lipofuscin in the retinal pigment epithelium and preserving vision in affected patients.