Biotechnology startup Axonis Therapeutics has leveraged the unique microgravity environment of the International Space Station (ISS) to advance research into neurological diseases. The company successfully grew 3D human brain organoids in space, a feat that is difficult to achieve on Earth due to the effects of gravity on cell growth. This breakthrough has accelerated the testing of gene therapies for conditions such as Alzheimer’s, Parkinson’s, and spinal cord injury.
Overcoming Terrestrial Limitations
On Earth, gravity causes cells to grow in two-dimensional monolayers, making it challenging to culture multiple neuron subtypes together and create complex 3D brain models. Axonis Therapeutics sought to overcome these limitations by utilizing the ISS National Laboratory to rapidly grow 3D human brain organoids, providing a more mature and realistic model for testing therapeutics.
Shane Hegarty, co-founder and chief scientific officer at Axonis Therapeutics, noted, “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.”
Rapid Organoid Formation in Microgravity
Axonis was awarded a Technology in Space Prize to conduct research on the space station. The team co-cultured mature neurons and astrocytes on the ISS, and within 72 hours, the cells self-assembled into 3D brain organoids. This rapid self-assembly is a significant advantage of microgravity, as it allows for the creation of complex neural structures that more closely mimic the human brain.
Viral Vector Gene Therapy
The team utilized a viral vector, modified to be harmless to humans, to deliver gene therapy to the neurons within the organoids. The viral vector was loaded with a fluorescent protein gene, and the successful expression of this gene in the neurons, indicated by glowing green cells, demonstrated the effectiveness of the delivery mechanism. This success significantly de-risked Axonis’ therapeutic approach and helped move it closer to clinical trials.
Financial Boost and Future Directions
The ISS National Lab-sponsored research has also enhanced Axonis' visibility and credibility, facilitating the attraction of seed investors. Axonis recently completed a $115 million Series A financing campaign, which was oversubscribed, indicating strong investor confidence. The company plans to use this funding to advance other therapeutics, including treatments for epilepsy and pain, through clinical proof-of-concept studies in patients.
