AviadoBio Reported Positive Gene Therapy Trial Results
Preliminary data from the ASPIRE-FTD trial indicates increased progranulin levels in patients treated for FTD-GRN.
Updated on Oct. 9, 2026 in Alzheimer’s
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AviadoBio has released preliminary clinical trial data showing dose-dependent increases in cerebrospinal fluid progranulin levels in patients with FTD-GRN. The ASPIRE-FTD study, which has dosed 17 patients, reported a 90% increase in progranulin in the fourth dose cohort.
Why it matters
Patients with FTD-GRN suffer from a significant deficiency of progranulin, a protein essential for brain cell function. Restoring these levels could address the root cause of the condition by bypassing the blood-brain barrier.
The ASPIRE-FTD trial involves 17 patients across 20 global sites. The fourth dose cohort demonstrated a 90% increase in CSF progranulin over baseline, while serum neurofilament light chain levels remain at or below baseline in patients with 52-week data.
The players
AviadoBio
This London-based biotechnology company focuses on the development of gene therapies for neurological conditions.
The details
The experimental therapy AVB-101 is administered via MRI-guided infusions directly into the thalamus during a neurosurgical procedure. No serious adverse events related to the treatment have been reported, and the trial requires no immunosuppression for participants.
Timeline
12 weeks: Timeframe used to measure progranulin increase in clinical trial cohorts.
52 weeks: Timeframe used to measure serum neurofilament light chain levels in patients.
The Big Picture
This development represents a significant progression in gene therapy research as evidenced by the ASPIRE-FTD trial. The results mark a potential shift in how neurodegenerative conditions are approached through direct protein restoration.
Participants in this trial are undergoing a surgical procedure that avoids the need for immunosuppression medications. While promising, the therapy remains in the clinical trial phase and is not yet available for general medical treatment.
The takeaway
Gene therapy continues to evolve as a potential method to treat the underlying protein deficiencies of neurodegenerative diseases. Researchers will continue monitoring long-term efficacy and safety outcomes as enrollment progresses.
Further reading
For more on the latest research in this field, visit our Alzheimer’s section.
More information
View the clinical trial study details for comprehensive information on this research.
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