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Alzheimer'S & Dementia : the Journal of the Alzheimer'S Association
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AVB-101 gene therapy for frontotemporal dementia (FTD-GRN) showed good safety and tolerability in the first patients. Early results suggest this treatment is promising for FTD-GRN, with ongoing trials for higher doses.

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Area of Science:

  • Neuroscience and Neurology
  • Gene Therapy
  • Neurodegenerative Diseases

Background:

  • Frontotemporal dementia (FTD) due to progranulin mutations (FTD-GRN) is a debilitating neurological disorder.
  • Current treatment options for FTD-GRN are limited, highlighting the need for novel therapeutic strategies.
  • AVB-101, an adeno-associated virus 9 vector gene therapy, aims to restore progranulin (PGRN) levels in the brain.

Purpose of the Study:

  • To evaluate the safety and tolerability of AVB-101 in patients with FTD-GRN.
  • To assess the preliminary efficacy of AVB-101 through safety measures and biomarker analysis.
  • To report initial safety findings from the first cohort of the ASPIRE-FTD Phase 1/2 trial.

Main Methods:

  • Phase 1/2, open-label, ascending dose study (NCT06064890) evaluating AVB-101.
  • One-time, bilateral intrathalamic infusion of AVB-101 guided by real-time MRI.
  • Safety assessments included adverse events, MRI, clinical/laboratory evaluations, and plasma/CSF Neurofilament light (NfL) protein levels.

Main Results:

  • AVB-101 was well-tolerated in the initial cohort of three patients.
  • No significant safety concerns, including hemorrhage, edema, or inflammation, were observed on serial MRIs.
  • No serious adverse events or AEs related to AVB-101 or the procedure were reported; transient NfL elevation was noted.

Conclusions:

  • Preliminary data from the ASPIRE-FTD trial indicate a favorable safety profile for AVB-101 gene therapy.
  • The intrathalamic administration procedure for AVB-101 was also well-tolerated.
  • Recruitment for higher dose cohorts is ongoing, suggesting continued interest and potential for AVB-101 in FTD-GRN.