GenAssist Therapeutics GEN6050X Exon 50 Skipping Drug Early Phase 1 Clinical Trial

Receipt of glucocorticoids for 6 months and a stable daily dose for at least 12 weeks prior to study entry

• The participant is able to walk independently and completes the 10-meter walk test without assistance.
• Participant is able to complete time to stand from supine independently in less than 30s.
• The participant is able to cooperate with motor assessment testing.
• Ability to tolerate muscle biopsies under anesthesia with no contraindications to these procedures.

• Participants are in the active period of viral infection, including infections such as TORCH virus, Epstein-Barr(EB) virus, and severe acute respiratory syndrome coronavirus 2 (SARS-COV-2).
• Received a live attenuated vaccine within 3 months prior to receiving GEN6050X, or was exposed to an influenza (or other inactivated) vaccine within 30 days prior to receiving GEN6050X, or received systemic antiviral, anti-infective, and/or interferon therapy.
• Serological tests found HIV, Hepatitis B Virus(HBV), hepatitis C virus(HCV), and syphilis infection.
• Severe infection (e.g., pneumonia, pyelonephritis, or meningitis) within 4 weeks prior to receiving gene therapy.
• With clear symptoms of cardiomyopathy, echocardiography shows that the left ventricular ejection fraction is less than 40%.
• Need for continuous or intermittent assisted support from a ventilator.
• Diagnosed with autoimmune disease or receiving related treatment for autoimmune disease.
• The following indicators are abnormal in laboratory biochemical testing:
• γ-glutamyl transpeptidase (GGT) above the 2-fold upper limit and total bilirubin above 1.5 times the upper limit, cystatin C (cystatin C) > 1.27 mg/L, hemoglobin (Hgb) < 100 or >200 g/L; Leukocytes (WBC) > 18.5×10^9/L or platelet ≤ 125×10^9/L.
• The titer of AAV9 neutralizing antibody determined by cell suppression assay > 1:50.
• Patients have received any gene therapy (e.g., adeno associated virus(AAV) gene therapy), cell therapy (e.g., stem cell transplantation), in vivo editing, or ex vivo editing therapy (e.g., CRISPR-Cas9, TALEN) in the past.
• Participant has any contraindication to immunosuppressive therapy.
• Has a medical condition or extenuating circumstance that, in the opinion of the principal investigator, is unsuitable for participation in the clinical trial.
• The family does not wish to disclose the patient's study participation to the attending physician and other medical providers.

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