In a swift succession of actions, China’s Center for Drug Evaluation (CDE) has added two more rare disease drugs to its patient-centered rare disease drug development pilot program, known as the Care Plan. This follows three other approvals within a 10-day period. The latest inclusions are Ractigen Therapeutics’ RAG-17 and Shanghai Vitalgen BioPharma Co., Ltd’s VGR-R01.
RAG-17: A siRNA Therapy for Amyotrophic Lateral Sclerosis (ALS)
RAG-17 is a superoxide dismutase (SOD1)-targeted small interfering RNA (siRNA) therapy developed through Ractigen Therapeutics’ SCAD intelligent chemical assisted delivery platform. The drug is indicated for the treatment of amyotrophic lateral sclerosis (ALS) and falls into the Pre IND stage before clinical trial implementation, categorized as stage B. Ractigen Therapeutics is expected to develop and validate a Professional Quality of Life Scale for ALS patients (ALS-QoL), translate and localize the Chinese version of the ALSRFS-R scale, and use these clinical outcome assessment (COA) indicators as efficacy evaluation indicators. Digital health technology is also planned for clinical application during the process.
VGR-R01: Gene Replacement Therapy for Crystalline Retinal Degeneration (BCD)
VGR-R01 is a gene replacement therapy targeting CYP4V2 mutations, intended for the treatment of crystalline retinal degeneration (BCD). CYP4V2, a member of the P450 enzyme family, is highly expressed in retinal pigment epithelial cells (RPE). BCD is an autosomal recessive progressive retinal degeneration disease closely associated with CYP4V2 mutations. VGR-R01 can be administered via subretinal injection, delivering target genes based on AAV vectors and expressing CYP4V2 protein in RPE cells to reconstruct the cellular fatty acid metabolism pathway and improve RPE cell function. Its filing belongs to the Key pre research stage, categorized as Stage C. A superior efficacy trial design will be adopted, with clinical outcome assessment including patient reported outcome (PRO) and performance outcome (PerfO).-Fineline Info & Tech
