The successful space-based research has not only advanced Axonis' therapeutic closer to clinical trials but also attracted significant investor interest, culminating in a $115 million oversubscribed Series A financing campaign.

To validate their therapy in a mature human brain model, Axonis utilized the International Space Station (ISS) National Laboratory to grow 3D human brain organoids, a process complicated by Earth's gravity.

Inspired by previous research that showed liver cells could form 3D organoids in space, Axonis aimed to see if central nervous system (CNS) cells would exhibit similar behavior.

Initial tests with the viral vector included loading it with a fluorescent protein gene, resulting in glowing green neurons, which confirmed the vector's effectiveness.

The viral vector, which had previously shown success in mice, required a more complex human model for further validation, leading to the ISS research.

This innovative research is featured in the latest issue of Upward, the official magazine of the ISS National Lab, highlighting insights from co-founder Shane Hegarty.

Axonis was awarded the Technology in Space Prize to conduct their ISS research, successfully co-culturing mature neurons and astrocytes to form 3D brain organoids within 72 hours.

These promising results have de-risked Axonis' therapeutic approach, moving it closer to clinical trials and mitigating associated risks.

The new funding will support the advancement of another therapeutic aimed at treating epilepsy and pain through clinical proof-of-concept studies.

Biotechnology startup Axonis Therapeutics is pioneering a gene therapy approach using a reprogrammed viral vector that specifically targets neurons to treat conditions like Alzheimer's and Parkinson's disease.

Co-founder Shane Hegarty emphasized the unique advantages of conducting research in microgravity, which can unlock new scientific potential.

Overall, Axonis' research has enhanced its reputation and positioned the company for future growth in the biotech sector.