Pfizer announced the preliminary results of gene therapy in Phase 1b clinical trials

, Pfizer announced the preliminary results of a gene therapy, PF-06939926, designed to treat Duchenne muscular dystrophy (DMD), in a Phase 1b clinical trial
.the results showed that this gene therapy can improve the expression of antimyostrophy protein in the patient's muscles, and improve muscle function indicators.about PF-06939926PF-06939926 is a gene therapy in the study. It contains a "mini-dystrophin" genetically modified version of the "mini-dystrophin" controlled by a human muscle-specific initiater in a gland-related virus 9 (AAV9) vector. AAV9 virus vectors have the ability to target muscle tissue for the delivery of genetically modified organisms.PF-06939926 has been approved by the FDA as an orphan drug and a rare pediatric disease drug.studyIn this Phase 1b clinical trial, six patients aged 6-12 received a dose of PF-06939926 at a dose of 1E14 vg/kg (low dose) or 3E14 vg/kg (high dose).The main endpoints of the trial were safety and tolerance, and secondary and exploratory endpoints included the expression level of mini-antimyostrophy protein in myofibres and other muscle function indicators.preliminary trial results showed that after 2 months of treatment, the proportion of muscle fibers expressing mini-antimyostrophy protein in the biceps sample was 38% in patients treated with low-dose therapy and 69% in patients treated with high-dose therapy.also tested two patients for low-dose therapy for muscle function. After one year of treatment, using the NorthStar Outpatient Assessment (NSAA), the scores of the two patients improved by 4.5 points, with baseline levels of 24 and 25., the NSAA score for DMD patients in this age group remained stable or decreased with age. (Drug Information Network)