The successful experiments conducted aboard the ISS have not only de-risked Axonis' gene therapy but have also accelerated their preparations for clinical trials.

This viral vector, which has shown success in mice, required validation in a more complex human model, making the ISS experiments crucial for further development.

The promising results from these experiments have significantly de-risked Axonis' therapeutic approach, bringing them closer to initiating clinical trials.

Recently, Axonis secured $115 million in an oversubscribed Series A funding round, which will also support the development of therapies for epilepsy and pain.

The success of their space-based research has bolstered Axonis' reputation and attracted substantial investor interest, culminating in the significant financing round.

Initial tests with the viral vector demonstrated its effectiveness by using a fluorescent protein gene, resulting in glowing green neurons that confirmed successful gene delivery.

Shane Hegarty emphasized the unique scientific opportunities presented by microgravity conditions, which can enhance research outcomes.

For more information on Axonis Therapeutics and their groundbreaking work, readers can refer to the ISS National Lab's magazine, 'Upward.'

Axonis is leveraging the unique environment of the International Space Station (ISS) National Laboratory to grow 3D human brain organoids, a process that is difficult to replicate on Earth due to gravitational constraints.

To advance their gene therapy research, the company has engineered a harmless virus into a viral vector that specifically targets neurons, promoting regeneration and preventing degeneration.

The innovative research conducted by Axonis is featured in the latest issue of 'Upward,' the official magazine of the ISS National Lab, which includes insights from co-founder Shane Hegarty.

In the U.S., nearly 600,000 individuals are diagnosed with Alzheimer's or Parkinson's disease each year, alongside approximately 18,000 new spinal cord injuries.