Science & Innovation
PIPELINE RICH IN POSSIBILITIES
Built on deep neuroscience expertise, AviadoBio is developing targeted, one-time gene therapies to precisely deliver genetic medicines to disease-relevant cells and pathways within the nervous system, restoring function and hope through advanced science.
Our precision platforms are focused on
- Blood-brain barrier (BBB)-penetrant delivery approaches including intrathalamic, intravitreal and intravenous routes of administration
- Engineered AAV vectors built for durable therapeutic expression across complex neural systems such as the brain and retina
- vMiX™ RNA silencing system for targeted RNA modulation of gain-of-function targets & disease pathways
AviadoBio’s pipeline spans frontotemporal dementia (FTD), retinal dystrophies including retinitis pigmentosa and geographic atrophy, tauopathies including Alzheimer’s disease and amyotrophic lateral sclerosis (ALS).
Pioneering Science
Prof Chris Shaw, Chief Scientific and Clinical Advisor and Co-Founder, discusses how AviadoBio’s gene therapy approach is designed to target FTD-GRN.
Our Approach
POWERED BY PRECISION™
Validated targets. Engineered payloads. Integrated delivery. Unified to deliver maximum benefit for patients.
Payload
Engineer gene supplementation and gene silencing payloads with cell-and tissue-selective regulatory control
Target
Select validated drivers of neuronal degeneration (CNS and retina) with substantial unmet medical need and burden
Delivery
Combine capsid innovation with fit-for-purpose routes of administration to optimize biodistribution, safety, and efficacy for each program
INVESTIGATIONAL GENE THERAPY AVB-101
AVB-101 is an investigational one-time therapy designed to deliver a functional copy of the GRN gene directly to the brain, thereby potentially restoring progranulin levels and delaying or even halting disease progression in participants with FTD-GRN. AVB-101 is administered using a neurosurgical procedure directly to the thalamus.
AviadoBio has developed a one-time surgical procedure to deliver gene therapy directly to the region of the brain called the thalamus.
Alex Bloom, PhD
Chief Technology Officer
Alex Bloom, PhD
FRONTOTEMPORAL DEMENTIA
Frontotemporal dementia (FTD) is a devastating form of early-onset dementia and is characterized by a rapid decline in executive function, behavior and/or language, and typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.
AMYOTROPHIC LATERAL SCLEROSIS
Retinitis Pigmentosa
Retinitis Pigmentosa (RP) is a leading cause of visual disability and blindness, affecting over 1.5 million people worldwide. Progressive symptoms include night blindness, reduction in peripheral and central vision, commonly called “tunnel vision,” and reduced ability to see details and colors.
