Nasal gene therapy offers new hope for brain injury patients

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Age 2 to 65 years, at the time of enrollment. 2. Documented diagnosis of chronic hypoxic-ischemic encephalopathy (HIE), confirmed by medical history, MRI findings, or neonatal records. 3. Stable medical condition for at least 6 months prior to enrollment (no major surgeries or hospitalizations related to HIE within that period). 4. Baseline Gross Motor Function Measure (GMFM-66 or GMFM-88) score between 40% and 70%, representing moderate functional impairment. 5. Completion or active receipt of intranasal 15-gene AAV9-PHP.eB therapy at a participating international clinical site under local physician supervision. 6. Parent(s) or legal guardian(s) willing and able to provide written informed consent for participation in the observational registry and data sharing. 7. Access to clinical follow-up and ability to participate in scheduled assessments or data submissions at 3, 6, 12, 18, and 24 months after treatment. Exclusion Criteria: 1. Active systemic infection, immune deficiency, or ongoing use of immunosuppressive agents (other than short-term rapamycin used per treating physician's protocol). 2. Known positive anti-AAV9 neutralizing antibody titer at baseline exceeding threshold values that may preclude effective vector transduction (if testing performed locally). 3. Uncontrolled seizure activity exceeding five episodes per day at baseline despite medical therapy. 4. Known or suspected malignancy, severe hepatic or renal dysfunction, or other conditions that would confound safety monitoring. 5. Previous gene therapy or investigational stem cell therapy within the past 12 months. 6. Known pregnancy or breastfeeding in post-pubertal female participants. 7. Any condition that, in the opinion of the local investigator or registry sponsor, may interfere with participation, data reliability, or patient safety.