Space-Grown Brain Organoids Help Advance New Neurological Treatment

Startup Leverages ISS National Lab to Test Viral Vector That Delivers Gene Therapy

KENNEDY SPACE CENTER, Fla., Nov. 21, 2024 /PRNewswire/ -- Did you know some viruses don't cause illness and can instead be used to treat disease? Biotechnology startup Axonis Therapeutics reprogrammed a virus to carry a novel gene therapy to neurons to treat neurological conditions like Alzheimer's, Parkinson's, and spinal cord injury. The company needed a way to test the therapeutic in a mature human brain model, which is difficult to produce on Earth. To address this challenge, Axonis leveraged the International Space Station (ISS) National Laboratory to quickly grow 3D human brain organoids and test the therapeutic.

The team's groundbreaking research is featured in the latest issue of Upward, official magazine of the ISS National Lab. In the article, Axonis Therapeutics co-founder and chief scientific officer Shane Hegarty says, "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." 

Viruses infect cells in a host and insert genetic material into them. In viruses that cause disease, the genetic material takes over cells and damages them. However, viruses can be reprogrammed to carry helpful genetic material. Axonis has modified a virus that is not harmful to humans to act as a viral vector. This vector targets neurons and delivers gene therapy designed to prevent neuron degeneration and spur regeneration. Importantly, it must be delivered only to neurons and no other cells in the body.

The viral vector worked in mice but required testing in a mature human brain model. On Earth, gravity forces cells to grow in 2D monolayers, and it is hard to culture multiple neuron subtypes together. The Axonis team learned about researchers who sent mature liver cells to space, and the cells spontaneously assembled into a 3D liver organoid. The team wondered if mature central nervous system (CNS) cells would do the same.

Axonis was awarded a Technology in Space Prize—funded by the Center for the Advancement of Science in Space™, which manages the ISS National Lab, and Boeing in partnership with the MassChallenge startup accelerator program—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—a feat not possible on Earth. The viral vector was loaded with a fluorescent protein gene instead of the gene therapy, and the team was excited to see glowing green neurons, meaning the viral vector had worked. These successful results significantly de-risked Axonis' therapeutic and helped move it closer to clinical trials.

Hegarty said the company's ISS National Lab-sponsored research also helped put Axonis on the map and made it easier for the startup to attract seed investors. Axonis recently announced the completion of a successful $115 million Series A financing campaign that was oversubscribed (the company received investor interest beyond its fundraising target). The company will use the funding to advance another of its therapeutics—this one to treat epilepsy and pain—through clinical proof-of-concept in patients.

To learn more about Axonis' exciting ISS National Lab-sponsored investigation, read the Upward feature "From Viral Villain to Gene Therapy Hero."

Download a high-resolution image for this release: Space-Grown Brain Organoid.

About the International Space Station (ISS) National Laboratory:
The International Space Station (ISS) is a one-of-a-kind laboratory that enables research and technology development not possible on Earth. As a public service enterprise, the ISS National Laboratory® allows researchers to leverage this multiuser facility to improve quality of life on Earth, mature space-based business models, advance science literacy in the future workforce, and expand a sustainable and scalable market in low Earth orbit. Through this orbiting national laboratory, research resources on the ISS are available to support non-NASA science, technology, and education initiatives from U.S. government agencies, academic institutions, and the private sector. The Center for the Advancement of Science in Space™ (CASIS™) manages the ISS National Lab, under Cooperative Agreement with NASA, facilitating access to its permanent microgravity research environment, a powerful vantage point in low Earth orbit, and the extreme and varied conditions of space. To learn more about the ISS National Lab, visit our website.

As a 501(c)(3) nonprofit organization, CASIS accepts corporate and individual donations to help advance science in space for the benefit of humanity. For more information, visit our donations page.

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SOURCE International Space Station National Lab