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 proprietary platforms, including intrathalamic and vMiX™ RNA silencing system, plus expertise in intravitreal, and blood-brain barrier (BBB)-penetrant delivery approaches, enable precise, durable therapeutic expression across complex neural systems such as the brain and retina. AviadoBio’s clinical and preclinical pipeline spans frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), inherited retinal diseases, and tauopathies, including Alzheimer’s disease.

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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.

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AviadoBio has developed a one-time surgical procedure to deliver gene therapy directly to the region of the brain called the thalamus.

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.

“What sets us apart is that we’re not just trying to do what’s easier but instead we’re challenging the status quo when it comes to next generation gene therapy development and delivery. At AviadoBio, we all take responsibility for our actions, and we know we can rely on our team members at all times to do what’s best for the company and patients.”

Alex Bloom, PhD

Chief Technology Officer

Alex Bloom, PhD

Chief Technology Officer

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.

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AMYOTROPHIC LATERAL SCLEROSIS

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease or Lou Gehrig’s disease, is a devastating multisystem neurodegenerative disease. ALS is primarily characterized by degeneration of both upper and lower motor neurons.

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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.

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Alzheimer’s disease and other tauopathies

Alzheimer’s disease and other tauopathies are a group of progressive brain disorders characterized by abnormal changes in tau, a protein that normally supports and stabilizes neurons. In these conditions, tau becomes dysfunctional, clumps together, and disrupts communication between brain cells, ultimately leading to cognitive decline, memory loss, and changes in behavior or movement.

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Science & Innovation

PIPELINE RICH IN POSSIBILITIES

Pioneering Science

INVESTIGATIONAL GENE THERAPY AVB-101

Alex Bloom, PhD

Alex Bloom, PhD

FRONTOTEMPORAL DEMENTIA

AMYOTROPHIC LATERAL SCLEROSIS

Retinitis Pigmentosa

Alzheimer’s disease and other tauopathies

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