A biotech company has solved one of the challenges faced by gene therapy for neurodegenerative diseases, but with former Novartis executives at the helm, 80 million USD To support its research.
AviadoBio’s leading project is gene therapy for frontotemporal dementia. The London-based startup plans to use Series A financing to advance the candidate to human testing and continue to work on research on preclinical assets, including potential gene therapies for amyotrophic lateral sclerosis.
AviadoBio’s technology is based on research in the laboratory of Christopher Shaw, a professor of neurology and neurogenetics at the Institute of Psychiatry, Psychology and Neuroscience at King’s College London. In the startup’s financing announcement, Shaw stated that neurodegenerative diseases initially only appear in a limited area, but the disease will eventually spread to the entire nervous system. Although modifying the genes at the root of the disease can be cured, the challenge lies in realizing the widespread distribution of therapies.
“We have proven that precise micro-administration of neural networks will provide a wide range of CNS expression and provide safe and effective treatments,” said Shaw, co-founder of AviadoBio and the company’s chief scientific and clinical consultant. “We are directly using insights into the causes of diseases to design treatments that have the potential to cure patients without effective treatments. I believe AviadoBio has the potential to transform neurodegenerative diseases from palliative to preventive.”
Frontotemporal dementia is a brain disease that affects the frontal and temporal lobes of the brain. There is no FDA-approved treatment that can change the course of the disease. In some cases, frontotemporal dementia is caused by mutations in the gene encoding precursor granulin, a protein that regulates the immune activity of the brain. AVB-PGRN, AviadoBio’s frontotemporal dementia drug candidate, is designed to deliver functional genes throughout the central nervous system to restore precursor granulin to normal levels. According to the company, the therapy is delivered to cells via an adeno-associated virus, is administered in the thalamus of the brain, and uses the organ’s neuronal network to maximize its distribution.
AviadoBio faces competition in pursuing the treatment of frontotemporal dementia. Passage Bio has advanced its gene therapy drug candidates to phase 1/2 testing. The gene therapy provided by AAV is injected into the cerebrospinal fluid to increase the level of granulin precursors to overcome the lack of this protein. At the same time, Alector’s drug candidate for frontotemporal dementia is an antibody, not a gene therapy.The drug is Developed in cooperation with GlaxoSmithKline, Designed to bind to proteins that cause the degradation of granule protein precursors. The plan is currently in the late stage of clinical development.
AviadoBio is incubated by F-Prime Capital and Johnson & Johnson Innovation-JJDC. The startup is led by CEO Lisa Deschamps, who is the former senior vice president and chief commercial officer of Novartis Gene Therapy. She oversees the commercialization of Zolgensma, a gene therapy for spinal muscular atrophy.
The new capital was made after the initial seed funding of $16.5 million. The Series A financing was led by New Enterprise Associates and Monograph Capital. Participants also include LSP and seed investors Advent Life Sciences, Dementia Discovery Fund, F-Prime Capital, JJDC and Lifers, a medical research charity.
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