Space research accelerates gene therapy for neurological disorders

Biotechnology startup Axonis Therapeutics has leveraged the unique microgravity environment of the International Space Station (ISS) to develop and test advanced neurological treatments. The company reprogrammed a harmless virus to deliver gene therapy directly to neurons, addressing conditions such as Alzheimer's, Parkinson's, and spinal cord injuries. Axonis used the ISS to create mature 3D human brain organoids for testing, overcoming challenges faced on Earth.

The research, detailed in the latest issue of 'Upward', the ISS National Lab's official magazine, highlights the company's innovative approach. "Doing research in space is not something you'd ever think about normally, but the opportunity to leverage microgravity conditions can unlock a lot of untouched potential by pushing the boundaries of science in a unique environment," explained Axonis co-founder and chief scientific officer Shane Hegarty.

Viruses are traditionally associated with causing disease, but Axonis demonstrated their potential as tools for healing. The company re-engineered a non-harmful virus into a viral vector capable of delivering gene therapy specifically to neurons, promoting regeneration and preventing degeneration. The key challenge was ensuring precision delivery to neurons without affecting other cells.

While the viral vector showed promise in mouse models, human brain testing posed a significant hurdle. On Earth, gravity limits cell cultures to flat, two-dimensional growth, complicating efforts to replicate mature, functional brain tissue. Inspired by space-based experiments where liver cells spontaneously formed 3D organoids, the Axonis team sought to apply similar methods to central nervous system (CNS) cells.

Awarded a Technology in Space Prize funded by the Center for the Advancement of Science in Space (CASIS) and Boeing, Axonis conducted its groundbreaking research aboard the ISS. In just 72 hours, mature neurons and astrocytes self-assembled into 3D brain organoids, an achievement unattainable in terrestrial labs. To test the viral vector, researchers replaced the gene therapy payload with a fluorescent protein gene. Glowing green neurons confirmed successful gene delivery, demonstrating the viral vector's effectiveness.

This major achievement de-risked Axonis' therapy, accelerating its path toward clinical trials. Hegarty noted that the ISS collaboration also elevated the company's profile, attracting investor interest. Axonis recently closed an oversubscribed $115 million Series A funding round, which will help advance another promising therapy targeting epilepsy and pain to clinical proof-of-concept.

To learn more about Axonis Therapeutics' innovative work on the ISS, read the 'Upward' feature "From Viral Villain to Gene Therapy Hero."