She runs a Boston biotech built on a single, stubborn idea: that one brain molecule called KCC2 could restore what spinal cord injury, epilepsy and chronic pain take away.
Co-founder, President & CEO · Axonis Therapeutics
Joanna Stanicka spends her days on a molecule most people have never heard of. KCC2 is a chloride transporter that helps the brain and spinal cord apply their own brakes - the inhibition that keeps signals from spiraling into seizures, spasticity and pain. When it fails, the nervous system loses control. Stanicka, the co-founder, president and chief executive of Axonis Therapeutics, is trying to turn that biology into a pill.
Axonis, based at 75 Kneeland Street in Boston, is a clinical-stage company with a deliberately narrow focus. Its lead candidate, AXN-027, is a first-in-class oral small molecule designed to potentiate KCC2. The company began first-in-human trials in 2024, aiming at focal epilepsy and neuropathic pain first, with an eye toward spinal cord injury and other central nervous system conditions. The pitch is unusual in its ambition: a single oral drug that could address paralysis, pain and spasticity at once, three co-morbidities that today are treated separately, if at all.
"We are the only company on the market developing an oral drug for all three debilitating co-morbidities: paralysis, pain and spasticity," Stanicka has said. That claim is the reason investors paid attention. In October 2024, Axonis closed an oversubscribed $115 million Series A, co-led by Cormorant Asset Management and venBio Partners. It pushed the company's total funding past $157 million, a figure that includes an earlier seed round of more than $34 million and roughly $8.7 million in non-dilutive support from the National Institutes of Health and the U.S. Department of Defense.
The money buys time to answer the only question that matters in drug development: does the mechanism work in people. Axonis has said the Series A proceeds are meant to carry AXN-027 through clinical proof-of-concept and to fund next-generation compounds targeting KCC2 in adjacent indications, including psychiatric and neurodevelopmental disorders. Stanicka's own summary of the mission is characteristically plain. Her stated vision, as Axonis puts it, is "laser-focused - to make the KCC2 drug a reality for patients."
"To see a healthy person become completely dependent and in pain within a few months, then see them relieved so quickly with the right treatment was life-changing."
The interest in medicine started early and personally. When Stanicka was ten, growing up in Poland, her cousin Ania developed aggressive rheumatoid arthritis. Stanicka watched her go from a healthy child to someone dependent and in constant pain within months - and then watched the reverse happen after Ania joined a clinical trial. The recovery was fast enough to feel like proof. That experience, she has said, was what pointed her toward research.
At nineteen she left Poland on a 40-hour bus journey to Ireland, where she built the foundations of a scientific career at University College Cork. She completed her undergraduate studies there, won an Irish Cancer Society Research Scholarship, and earned a Ph.D. in cancer biology, staying on for postdoctoral work in cell biology. Cancer, not the brain, was her first subject.
The pivot came in Boston. Recruited into the laboratory of Professor Zhigang He at Boston Children's Hospital and Harvard Medical School, Stanicka moved into neuroscience, running genome-wide CRISPR screens on neuroregeneration - the search for ways to coax a damaged central nervous system into repairing itself. It was there, alongside research fellow Shane Hegarty, that she began studying how to restore function after spinal cord injury. The two would go on to co-found Axonis with Corey Goodman, a co-founder of venBio Partners, and Bob Yant, who sustained a severe spinal cord injury in 1981 and has spent decades raising money for research into it.
Postdoctoral research fellow in Zhigang He's lab at Boston Children's Hospital / Harvard Medical School, studying CNS repair and neuroregeneration.
Co-founds Axonis Therapeutics with Shane Hegarty, Corey Goodman and Bob Yant. Joins as Chief Scientific Officer and is promoted to CEO within six months.
Leads Axonis to an oversubscribed $115M Series A co-led by Cormorant and venBio. Lead drug AXN-027 enters first-in-human trials.
In biotech, "Golden Tickets" from major pharma companies are incubator prizes that hand young startups lab space, mentorship and credibility. Stanicka has collected several, alongside broader honors.
Bars scaled to total funding raised under Stanicka's leadership. Figures per Axonis and public reporting.
Stanicka often frames the company's purpose around a patient population rather than a market. "There are about 300,000 chronically injured SCI patients in the US, and about 70 to 80 percent experience either pain, spasticity or both," she has said. For most of them, there is no oral drug designed to address the underlying loss of inhibition in their nervous system.
That gap is the reason Axonis exists, and it explains why Stanicka moved from a career studying disease at the bench to one spent raising capital, hiring teams and steering a molecule through the clinic. The move from scientist to CEO is one of the hardest transitions in biotech, and she made it fast - inside six months of joining the company she helped found.