In an astonishing leap for medical science, biotechnology startup Axonis Therapeutics is utilizing the extraordinary environment of the International Space Station (ISS) to develop innovative treatments for debilitating neurological conditions like Alzheimer's, Parkinson's, and spinal cord injuries. While you might be familiar with how some viruses can lead to illness, it turns out that reprogramming them can instead hold the key to healing!

Axonis has ingeniously modified a non harmful virus to serve as a viral vector, capable of delivering a novel gene therapy specifically to neurons, preventing degeneration, and promoting regeneration of damaged nerve cells. This groundbreaking research is featured in the latest edition of Upward, the official magazine of the ISS National Lab.

Shane Hegarty, co-founder and chief scientific officer of Axonis, explains, “Conducting research in space opens up endless possibilities. The unique microgravity conditions allow us to explore scientific boundaries that are impossible to achieve on Earth.”

Traditionally, growing mature human neurons for experimental purposes presents significant challenges due to our planet’s gravitational forces, which restrict cell growth to two-dimensional layers. However, previous experiments with liver cells in space demonstrated that mature cells could spontaneously assemble into complex three-dimensional organoids — a breakthrough that spurred Axonis to investigate whether central nervous system (CNS) cells would exhibit similar behavior.

Thanks to a prestigious Technology in Space Prize, funded by the Center for the Advancement of Science in Space™ and Boeing in partnership with MassChallenge, Axonis was able to embark on this ambitious research aboard the ISS. The results were nothing short of spectacular: within just 72 hours of co-culturing mature neurons and astrocytes in microgravity, the cells self-organized into 3D brain organoids, a feat previously thought impossible on Earth.

To confirm the efficacy of their viral vector, Axonis introduced a fluorescent protein gene, resulting in vibrant green neurons—proof that their approach was a resounding success! This crucial experiment has not only minimized the risks associated with Axonis' therapeutic but has also positioned the company for the next step toward clinical trials.

Hegarty further shared that the ISS National Lab-sponsored research has significantly elevated the company's profile, leading to a successful $115 million Series A funding round, which exceeded their projections. With this new capital, Axonis plans to further develop their therapies aimed at treating epilepsy and pain, moving closer to real-world applications.

This astonishing research demonstrates that the final frontier of space is not just about exploration but also holds phenomenal potential for healing and human health. To discover more about Axonis's thrilling journey into the cosmos and its implications for neurological health, check out the detailed feature “From Viral Villain to Gene Therapy Hero” in Upward magazine.

Stay tuned as we witness the dawn of a new era in medicine, driven by innovation and the limitless possibilities that await us among the stars!