BioPharma, Pharma

Biotech startup AviadoBio emerges with $80M for neuro disorder gene therapies

AviadoBio’s gene therapies are designed to achieve widespread distribution throughout the central nervous system. The company’s lead program on track to begin human testing as a treatment for frontotemporal dementia.

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A biotech company with an answer for one of the challenges facing gene therapies for neurodegenerative disorders is out of stealth with a former Novartis executive at the helm and $80 million to support its research.

The lead program of AviadoBio is a gene therapy for frontotemporal dementia. The London-based startup plans to use the Series A round of funding to advance that candidate into human testing, and to continue work on preclinical assets that include a potential gene therapy for amyotrophic lateral sclerosis.

AviadoBio’s technology is based on research from the laboratory of Christopher Shaw, a professor of neurology and neurogenetics at King’s College London’s Institute of Psychiatry, Psychology & Neuroscience. In the startup’s funding announcement, Shaw said that neurodegenerative conditions are initially seen only in a limited area, but the disease eventually spreads throughout the nervous system. While modifying the genes at the root of the disorder can be curative, the challenge is achieving widespread distribution of a therapy.

“We have shown that precision micro dosing to neural networks will deliver broad CNS expression, providing safe and effective treatments,” said Shaw, who is AviadoBio’s co-founder and chief scientific and clinical advisor to the company. “We are directly exploiting insights into the causes of diseases to design therapies that have the potential to cure patients for whom there are no effective treatments. I believe that AviadoBio has the potential to move neurodegeneration from palliation to prevention.”

Frontotemporal dementia refers to brain disorders affecting the frontal and temporal lobes of the brain. There are no FDA-approved therapies that modify the course of the disease. In some cases, frontotemporal dementia is caused be a mutation to the gene that encodes progranulin, a protein that is regulates immune activity in the brain. AviadoBio’s frontotemporal dementia candidate, AVB-PGRN, is designed to deliver a functional gene throughout the central nervous system in order to restore progranulin to normal levels. According to the company, the therapy, which is delivered to cells via adeno-associated virus, is dosed in the thalamus of the brain and uses the neuronal networks of the organ to maximize its distribution.

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AviadoBio faces competition in the pursuit of a frontotemporal dementia treatment. Passage Bio has advanced its gene therapy candidate to Phase 1/2 testing. The AAV-delivered gene therapy, which is administered via injection into the cerebrospinal fluid, is intended to boost levels of progranulin to overcome the deficiency of that protein. Meanwhile, Alector’s frontotemporal dementia drug candidate is an antibody, not a gene therapy. The drug, which is being developed under a partnership with GlaxoSmithKline, is designed to bind to a protein that leads to the degradation of progranulin. That program is currently in late-stage clinical development.

AviadoBio was incubated by F-Prime Capital and Johnson & Johnson Innovation —JJDC. The startup is led by CEO Lisa Deschamps, former senior vice president and chief business officer of Novartis Gene Therapies, where she oversaw the commercialization of the spinal muscular atrophy gene therapy Zolgensma.

The new capital follows an initial $16.5 million seed funding. The Series A round 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 medical research charity Lifers.

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