A new biotech aims to get RNA drugs into the brain

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The latest biotechnology company hoping to get genetic medicines into the brain has now launched, with backing from more than half a dozen investment firms.

Aerska, a Dublin-based startup whose name comes from a Gaelic proverb, on Wednesday announced it has raised $21 million in seed funding. Age1, Backed VC and Speedinvest co-led the funding round, while other early-stage investors like Blueyard, Norrsken VC and Ada Ventures participated.

Aerska’s research focuses on RNA interference, a drugmaking technique that treats illnesses by blocking the genetic information the body uses to create harmful or undesirable proteins. Specifically, Aerska is trying to silence genes that drive brain diseases. It’s an ambitious goal. So far, RNA interference technology has been most successful when directed at disease targets in the liver.

The brain, meanwhile, is encased in a protective shield that keeps out larger molecules, including genetic medicines.

Over the past decade or so, a new class of biotechs has spawned boasting technologies designed to surmount that tricky “blood-brain barrier.” One, Aliada Therapeutics, formed in 2021 with seed funding from RA Capital Management and the venture arm of Johnson & Johnson. Built around a J&J technology, Aliada operated for about three years before AbbVie swooped in and bought it for $1.4 billion.

AbbVie’s acquisition added to an already hot area of dealmaking. Eisai, the Japan-based pharmaceutical firm, in April of last year agreed to work with longtime partner BioArctic on a preclinical drug made with the latter’s patented “BrainTransporter” platform. Then, a few months later, Roche licensed a technology from Sangamo Therapeutics.

Even more recently, GSK wagered billions of dollars on barrier-crossing tools from ABL Bio while Biogen purchased Alcyone Therapeutics for a technology meant to conveniently send RNA drugs known as “antisense oligonucleotides” to the brain

Those deals followed other, similar pacts between Biogen and Denali Therapeutics and Eli Lilly and Entos Pharmaceuticals.

“Neurological diseases remain one of the greatest challenges in medicine, with limited options to alter the course of disease,” said Jack O’Meara, Aerska’s CEO and one of its three co-founders, in a statement. The company’s aim, O’Meara added, is to “enable precise, durable gene silencing” in the CNS — or central nervous system — by “integrating brain shuttles with RNA therapeutics.”

Another Aerska co-founder, Stuart Milstein, helped lead brain delivery efforts at Alnylam Pharmaceuticals, a pioneering company in the RNA interference field. The biotech’s research head, Mike Perkinton, previously led discovery work and served as an executive director in AstraZeneca’s neuroscience division.

Aerska’s “antibody-oligo conjugate” platform is meant to deliver RNA interfering medicines throughout the brain, where it can be taken in by neurons and subdue certain genes.

Additionally, the company is investing in data science to “advance a precision medicine strategy” in neurology, with an initial focal point being genetic forms of Alzheimer’s disease and Parkinson’s.

“Delivery across the blood-brain barrier remains the bottleneck for genetic medicines in neurology,” said Alex Brunicki, a partner at Backed VC and an Aerska board member. Aerska’s platform therefore positions the company to be “at the forefront of CNS therapeutics.”

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter.

The latest biotechnology company hoping to get genetic medicines into the brain has now launched, with backing from more than half a dozen investment firms.

Aerska, a Dublin-based startup whose name comes from a Gaelic proverb, on Wednesday announced it has raised $21 million in seed funding. Age1, Backed VC and Speedinvest co-led the funding round, while other early-stage investors like Blueyard, Norrsken VC and Ada Ventures participated.

Aerska’s research focuses on RNA interference, a drugmaking technique that treats illnesses by blocking the genetic information the body uses to create harmful or undesirable proteins. Specifically, Aerska is trying to silence genes that drive brain diseases. It’s an ambitious goal. So far, RNA interference technology has been most successful when directed at disease targets in the liver.

The brain, meanwhile, is encased in a protective shield that keeps out larger molecules, including genetic medicines.

Over the past decade or so, a new class of biotechs has spawned boasting technologies designed to surmount that tricky “blood-brain barrier.” One, Aliada Therapeutics, formed in 2021 with seed funding from RA Capital Management and the venture arm of Johnson & Johnson. Built around a J&J technology, Aliada operated for about three years before AbbVie swooped in and bought it for $1.4 billion.

AbbVie’s acquisition added to an already hot area of dealmaking. Eisai, the Japan-based pharmaceutical firm, in April of last year agreed to work with longtime partner BioArctic on a preclinical drug made with the latter’s patented “BrainTransporter” platform. Then, a few months later, Roche licensed a technology from Sangamo Therapeutics.

Even more recently, GSK wagered billions of dollars on barrier-crossing tools from ABL Bio while Biogen purchased Alcyone Therapeutics for a technology meant to conveniently send RNA drugs known as “antisense oligonucleotides” to the brain

Those deals followed other, similar pacts between Biogen and Denali Therapeutics and Eli Lilly and Entos Pharmaceuticals.

“Neurological diseases remain one of the greatest challenges in medicine, with limited options to alter the course of disease,” said Jack O’Meara, Aerska’s CEO and one of its three co-founders, in a statement. The company’s aim, O’Meara added, is to “enable precise, durable gene silencing” in the CNS — or central nervous system — by “integrating brain shuttles with RNA therapeutics.”

Another Aerska co-founder, Stuart Milstein, helped lead brain delivery efforts at Alnylam Pharmaceuticals, a pioneering company in the RNA interference field. The biotech’s research head, Mike Perkinton, previously led discovery work and served as an executive director in AstraZeneca’s neuroscience division.

Aerska’s “antibody-oligo conjugate” platform is meant to deliver RNA interfering medicines throughout the brain, where it can be taken in by neurons and subdue certain genes.

Additionally, the company is investing in data science to “advance a precision medicine strategy” in neurology, with an initial focal point being genetic forms of Alzheimer’s disease and Parkinson’s.

“Delivery across the blood-brain barrier remains the bottleneck for genetic medicines in neurology,” said Alex Brunicki, a partner at Backed VC and an Aerska board member. Aerska’s platform therefore positions the company to be “at the forefront of CNS therapeutics.”