Neurodegenerative Gene Therapy Biotech AviadoBio Exits Stealth Mode With $80M Series A

Capsule with DNA inside
Credit: iStock/© D3Damon

AviadoBio has come out of stealth mode to announce the raising of an $80M Series A round to develop gene therapies to target neurodegenerative conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).

The round was co-led by international VC’s New Enterprise Associates and Monograph Capital. Other participants in the round included LSP, seed investors Advent Life Sciences, the UK’s Dementia Discovery Fund, F-Prime Capital, Johnson & Johnson Innovation – JJDC and medical research charity LifeArc.

Based in London, AviadoBio was launched with $16.5M seed financing in 2019. The company’s science was developed in the lab of Christopher Shaw, a professor at Kings College London. Shaw and colleagues have developed a gene therapy platform to produce adeno-associated viral (AAV) vector therapies engineered to target neurological disorders such as FTD and ALS, which do not currently have viable therapies available.

“Whilst neurodegenerative conditions are focal at onset, the pathology eventually spreads throughout the nervous system. We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad central nervous system expression, providing safe and effective treatments,” said Shaw, who is also co-founder and Chief Scientific Officer of AviadoBio, in a press statement.

The startup’s most advanced therapy candidate, for FTD, has almost reached clinical trials and the plan for some of the money is to start a first in human trial. FTD is a variable neurodegenerative condition that typically shows symptoms between the age of 45 and 65 years. It differs from other forms of dementia in that the key symptoms are changes in personality, behavior and language rather than short term memory.

The FTD candidate has an intrathalamic delivery route into the brain and will target progranulin, a glycoprotein encoded by the GRN gene. Progranulin plays a role in inflammation and neuron growth and is mutated in approximately 20% of inherited and 5% of sporadic cases of FTD causing a dramatic reduction in levels around the body. The therapy will deliver copies of the unmutated GRN gene to neurons in order to raise progranulin levels and reduce symptoms in those affected.

AviadoBio also plans to invest in developing its other preclinical programs, targeting ALS and other forms of FTD via gene inactivation, and bring them closer to the clinic.

The company is led by CEO Lisa Deschamps who has spent 25 years working at Novartis and is an industry veteran. In addition to Shaw, Youn Bok Lee and Do Young Lee, both also from Kings College London, are scientific co-founders.

In the past, an issue with AAV vectors has been precise enough targeting, with off-target build up in the liver causing toxicity in some patients. However, AviadoBio has engineered its vectors to more precisely target the neurosystem.

“AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery,” says Shaw. “Precision micro dosing achieves extensive gene expression throughout the nervous system, maximizing the therapeutic potential for patients living with devastating neurological diseases.”

Neurological disorders have been notoriously difficult to treat to date, with many failed late-stage trials. Gene therapy has shown some recent success in this area, with Zolgensma for treatment of spinal muscular atrophy, a rare neurodegenerative disease of infancy where those affected rarely live beyond 2 years without treatment. This success has encouraged others to enter the space.

In addition to AviadoBio, PassageBio is also developing gene therapies to target neurological diseases including one targeting GRN mutated FTD.  Its therapy, which is injected into the cisterna magna in the brain, started Phase I trials earlier this year.

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