A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Exclusion Criteria

• •Prior treatment with gene or cell-based therapy at any time.
• •Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.
• •Has left ventricular ejection fraction \< 50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy.
• •Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.
• •Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during this study.
• •The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic/allergic, behavioural disease, infection, unhealed injury, malignancy, concomitant illness, extenuating circumstance, or requirement for chronic drug treatment that, in the opinion of the Investigator:
• •1. Creates unnecessary risks for undergoing gene transfer;
• •2. Might compromise the participant's ability to comply with the protocol-required testing or procedures; or
• •3. Might compromise the participant's well-being, safety, or clinical interpretability.
• •Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
• •Has signs of clinically significant symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
• •Has contraindications for IT administration of the product or for lumbar puncture, such as anatomical abnormalities, bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or significant clotting abnormalities).
• •Demonstrates cognitive or developmental delay or impairment that could confound assessment of motor development in the opinion of the Investigator.
• •Total serum anti-AAV9 antibody titers of \> 1:50 as determined by ELISA within 14 days of Day 1.