Science & Delivery
PIPELINE RICH IN POSSIBILITIES
AviadoBio has developed a next-generation AAV gene therapy platform focused on controlling gene expression and is complemented by a suite of unique delivery solutions to deliver gene therapies directly to the brain and spinal cord. We are also building additional platforms and capabilities as well as proprietary RNA silencing and subpial delivery technologies. Our neuroanatomy-led approach to viral vector distribution seeks to deliver our therapies to the right place to maximize their potential.
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, adeno-associated virus (AAV) gene therapy for patients with frontotemporal dementia (FTD) with disease-causing mutations in the progranulin (GRN) gene. AVB-101 is designed as a one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore progranulin levels throughout the brain. AVB-101 is designed to be administered intrathalamically to facilitate transport of the viral vector and the progranulin protein by exploiting the neuronal networks of the brain to maximize biodistribution.
“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.
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.