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Actio Lands $53M Series A

BIOTECH: Platform Addresses Rare, Common Disease Targets

Actio Biosciences is advancing its novel platform that develops treatments very rare diseases that may also work for more common ailments.

The company announced this month it had raised a $55 million Series A financing led by Canaan and DROIA Ventures, with participation from existing investors Deerfield Management and EcoR1, as well as new investor Euclidean Capital.

David Goldstein, Ph.D.
CEO & Co-founder
Actio Biosciences

Actio CEO and co-founder David Goldstein, Ph.D. said the money will be used to take the company’s lead drug candidate “well into the clinic with the hope that will give proof of concept.”

Actio’s lead program is a small molecule that targets mutations associated with either neuropathies or skeletal dysplasia and are the cause of Charcot-Marie-Tooth (CMT) disease type 2C and other serious bone diseases.

In addition to possibly treating the very rare form of CMT, the molecule may also be a solution for a common ailment – overactive bladder, which CMT patients often report having.

“So we’re learning something from the rare disease about the role of the target in overactive bladder,” Goldstein said.

In mouse models, the molecule has been able to “dramatically improve” disease course in the mice after symptoms emerge and if treated early may result in “complete reversal,” he added. “And we think we understand the biology of why that happens, turns out quite unusual – not mutation in neurons but in vascular endothelial cells.”

That understanding of the biology, coupled with the mouse data shows “strong evidence” of the molecule’s potential effectiveness toward common indications of bone health and overactive bladder, Goldstein said.

From Rare to Common

Actio’s platform of discovery that allows it to translate treatments for rare disease to more common ailments uses a differentiated strategy empowered by a proprietary library of rare diseases it calls the Rare Disease Target Atlas.

The Atlas comprises of every single known Mendelian disease – about 4,000 – and includes everything that is known about the mutations from a variety of scientific and healthcare sources.

“We pull all these together and really try to identify those genes that look like they represent the very best opportunity to be able to develop a highly effective treatment for the rare disease and be able to learn from the rare disease about the target’s biology,” Goldstien said.

Actio has also established a partnership with the Rare Disease Translational Center at The Jackson Laboratory (JAX), a nonprofit biomedical research institution dedicated to understanding the molecular causes of rare diseases and their treatments. JAX is providing Actio with genetically engineered mice to conduct testing that results in high confidence of similar outcomes in humans.

‘De-Risking’ Very Rare

The Actio platform and its initial focus on rare disease with an eye on potential relevance in more common diseases allows the company to “de-risk targets of interest,” said Actio Board Member and co-founder John McHutchison, M.D., adding that it “is unlike anything else in the industry today.”

Goldstein said he and McHutchison founded Actio after becoming frustrated by the gap between the knowledge of very rare disease causes and the action in treating them – especially when it came to explaining to parents that doctors knew the cause of their child’s disease, but there were no effective treatments.

“I was particularly frustrated by that, given that some of the responsible genes for these diseases looked to me like very tractable targets for drug development efforts, but they weren’t getting a lot of attention from industry because the diseases were very, very rare,” he said, adding that most companies working on rare diseases focus on the “more common end of the spectrum,” even though very rare diseases offer targets “where there is hope for a truly transformational treatment if you worked on it.”

Beyond Actio’s lead CMT program, the company has identified two additional programs it plans to evaluate for rare genetic epilepsies and genetic skin conditions.

Actio Biosciences

Founded: 2021
CEO: David Goldstein, Ph.D.
Headquarters: Sorrento Valley
Funding: $63 million (Seed and Series A)
Employees: 21
Website: www.actiobiosciences.com
Notable: Actio’s Rare Disease Target Atlas comprises of every single known Mendelian disease – about 4,000 of them.

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