RNA medicines to treat, delay, and prevent diseases of the brain
Targeted delivery of RNA medicines to the brain. By silencing the genes that cause harm, we aim to preserve minds, protect memories, and enable our loved ones to live healthier, longer lives.
Precision medicine for the brain
Built on three pillars:
Next-generation RNA medicines
Systemic administration and targeted brain delivery
Precision and prevention to transform CNS treatment
RNAi can redefine CNS treatment
RNAi offers unprecedented potency, durability, and precision for hitting disease targets at their origin, intracellularly. In recent years, RNAi has transformed treatment of liver and cardiometabolic diseases, but the full potential of RNAi for CNS diseases has yet to be realised.
Less invasive, targeted delivery of RNA
Major strides have been made in brain shuttle technologies, enabling the delivering of antibody and protein drugs across the blood-brain barrier (BBB).
We’re building on this foundation through partnerships and internal innovation to accelerate delivery of RNA medicines to the brain.
prevention
Ensuring patients get the right medicine at the right time
Precision medicine transformed cancer care, and we believe a similar opportunity exists for many neurological disorders. By combining target biology, data-driven patient stratification, and biomarker-guided development, we aim to bring precision medicine to CNS diseases.
It's a path that could transform how we treat brain disorders—one patient, one gene, one memory at a time.
Devoted to making better brain medicines
World experts in RNAi, brain shuttles and CNS drug development have come together to expand treatment options for those suffering from neurological diseases.

Jack O’Meara
Jack O’Meara

Stuart Milstein
Stuart Milstein

Dave Hardwicke
Dave Hardwicke

Alycia Shoultz
Alycia Shoultz

Andrew Cregan
Andrew Cregan

David Coughlan
David Coughlan

Duygu Yilmaz
Duygu Yilmaz

Michael Perkinton
Michael Perkinton

Robyn McAdam
Robyn McAdam

Ollie King
Ollie King

Amélie Bowden-Feytout
Amélie Bowden-Feytout

Mallik Putta
Mallik Putta

Sam Handelman
Sam Handelman

Marta Dragoshi
Marta Dragoshi

Jack O’Meara - Board Member
Jack O’Meara - Board Member

Stuart Milstein
Stuart Milstein

Alex Brunicki
Alex Brunicki

Alex Colville
Alex Colville

Andrea Zitna
Andrea Zitna

Jason Whitmire
Jason Whitmire

Uli Stilz
Uli Stilz

Mark Wolters
Mark Wolters

Peter Hoehn
Peter Hoehn

Lisa Taylor Ash
Lisa Taylor Ash

Dominic Walsh
Dominic Walsh

Lynne Hughes
Lynne Hughes
Standing on the shoulders of giants
Brain-shuttled RNAi has been decades in the making.
- 1991
- 1998
- 2018
- 2021
- 2023
- 2024
- 2025
Transferrin (TfR) receptor used for drug delivery
Scientists from Alkermes demonstrate the utility of using the TfR receptor as a means to deliver drug payloads to the brain.
RNAi is discovered
Andrew Fire and Craig Mello uncover RNA interference in the nematode worm—revealing a new way to silence genes.
ReferenceFirst RNAi medicine approved
Alnylam’s Onpattro uses LNP-coated RNAi, delivered to liver cells by IV infusion for the treatment of hATTR amyloidosis
ReferenceFirst BBB crossing enzyme replacement therapy approved.
JCR’s pabinafusp alfa (Izcargo) is approved in Japan for Hunter syndrome, validating TfR brainshuttle technology for delivering protein/enzymereplacement cargo to the CNS.
ReferenceRNAi delivered to the CNS
First human validation of siRNA knockdown in the CNS via spinal cord injection. Stuart Milstein, Aerska Co-Founder, initiated work on this advancement to deliver siRNAs to the brain with his Alnylam colleagues.
ReferenceTrontinemab shows rapid, deep amyloid clearance at low doses
Roche showed that its TfR “Brain shuttle” antibody cleared amyloid fast and deeply using much lower doses than standard (‘non-shuttled’) mAbs.
ReferenceAerska founded
Aerska launches to unlock precision RNAi delivery to the brain, building on decades of innovation in RNAi, BBB shuttles, and bioconjugation chemistry.
Aerska founded
Aerska launches to unlock precision RNAi delivery to the brain, building on decades of innovation in RNAi, BBB shuttles, and
Our Values
Walk on Air
Ambition spurs us to tackle problems no one has solved (yet). We keep our sights on breakthroughs that will change patients’ lives. We take calculated risks, guided by a vision for the world we want to see rather than a fear of failing.
Time is Neurons
Patients deserve our urgency and precision. Getting to the clinic quickly relies on focus, efficient decision making, and scientific excellence. Urgency isn’t frenetic, it’s purposeful. Saving time today means saving neurons tomorrow.
Win or Learn
Sometimes experiments fail. We share our misses openly, extract lessons, and move forward. Every result is a data point, and every day is a school day.
We Before Me
Inspired by our namesake, our success relies on one another. We have the curiosity to seek other perspectives and the humility to adopt them. Open communication and healthy debate sharpens our thinking, but once a decision is made, we advance as one team.