A two-dozen-person Belgian biotech took an old, stubborn drug target - and built a cleaner way to hit it. Now it is in the clinic.
The logo doubles as a microtubule diagram. Fitting, for a company obsessed with how nerves move their cargo.
HDAC6 has been on the wall of drug-hunters for two decades. It is a cytoplasmic enzyme that sits at the crossroads of how cells move cargo, clear waste, and handle stress. Block it in the right way and damaged nerves start behaving again. Block it the wrong way and you poison everything else in the process. For years, the wrong way was the only way anyone knew.
Augustine Therapeutics is the company betting that the right way finally exists. It is small - around 24 people spread across an HQ in Leuven, a foothold in Cambridge, Massachusetts, and a research subsidiary in Copenhagen. It is young, founded in 2019. And as of 2025 it is doing the one thing that separates a biotech story from a biotech company: it is dosing actual patients.
HDAC6 is a well-known target. We've got a breakthrough approach.
- Augustine Therapeutics, company taglineThe earlier generation of HDAC6 inhibitors leaned on a chemical group called a hydroxamic acid - a kind of molecular grappling hook. It worked. It also grabbed onto things it shouldn't, which is a polite way of saying it was too toxic to take every day for the rest of your life. For a cancer course measured in weeks, you can live with that. For a chronic disease measured in decades, you cannot.
That is the tension at the center of this company. The diseases that HDAC6 could help - inherited neuropathies, neurodegeneration, cardio-metabolic disorders - are chronic by definition. The tools to drug it were built for acute use. A breakthrough target, locked behind a safety problem.
A target that works in a petri dish and poisons a patient is not a drug. It is a footnote.
- The chronic-disease problem, in one sentenceAnd the patients were not abstract. Charcot-Marie-Tooth disease - CMT - is one of the most common inherited neurological disorders on earth, affecting roughly 3 million people. It slowly weakens hands, feet, and legs. It is progressive. And in 2025, it still had no approved drug that changes its course. Not a better drug. No drug.
The science traces back to Prof. Ludo Van Den Bosch at the VIB-KU Leuven Center for Brain and Disease Research, who showed that inhibiting HDAC6 could rescue the failing transport machinery inside damaged nerves. Augustine spun out of that work in 2019 to do something specific with it: design HDAC6 inhibitors that skip the toxic hydroxamate warhead entirely.
The result is a proprietary non-hydroxamate, non-hydrazide chemotype. In plain terms, a different chemical handle that grips HDAC6 selectively - shutting down the catalytic activity that drives disease while leaving the enzyme's other, useful jobs intact. Selective enough, the bet goes, to be taken safely for years.
Augustine's HDAC6 inhibitors are purposefully designed to selectively inhibit HDAC6 while preserving its beneficial non-catalytic functions.
- Augustine TherapeuticsThe bet was not subtle. It was: the reason no one has a chronic HDAC6 drug is the chemistry, not the biology - so fix the chemistry. Investors apparently agreed. So, eventually, did Eli Lilly, which rarely shows up to co-invest alongside venture funds at Series A.
Augustine isn't building a single drug. It's building a platform around HDAC6 and pointing it at three very different problems - each with a molecule tuned to where the disease lives in the body.
A peripherally-restricted, selective HDAC6 inhibitor - the first of its kind in the clinic for Charcot-Marie-Tooth disease. First patient dosed in May 2025.
An orally bioavailable, peripherally-restricted follow-on aimed at cardio-metabolic disorders - a far larger patient population.
A central-acting, blood-brain-barrier-penetrant candidate for neurodegenerative diseases such as ALS.
Three molecules, one enzyme, and a map of the human body drawn by where each drug is allowed to go.
In March 2025, Augustine closed an oversubscribed Series A of EUR 77.7 million (USD 84.8 million), co-led by Novo Holdings and Jeito Capital. The round built on a EUR 17.5M first closing led by Asabys Partners, and pulled in AdBio Partners, V-Bio Ventures, PMV, the CMT Research Foundation, Newton Biocapital, the Gemma Frisius Fund, VIB - and Eli Lilly. Total raised to date sits near EUR 89 million.
Big Pharma rarely co-invests with venture funds at a Series A. Lilly did here. Read that twice.
- On the company they keepTwo rounds, one trajectory. The bar that matters most is the one labeled "first patient" - and it isn't on this chart.
Sources: company announcements & GlobeNewswire, 2024-2025. Bars scaled to total raised.
Founded on Prof. Ludo Van Den Bosch's discovery that HDAC6 inhibition rescues nerve transport.
Led by Asabys Partners. Gerhard Koenig, PhD, joins as Executive Chairman in June.
The drug-development veteran steps from chair into the operating seat.
Co-led by Novo Holdings and Jeito Capital, with Eli Lilly participating.
The first selective HDAC6 inhibitor enters the clinic for Charcot-Marie-Tooth disease.
Rie Schultz Hansen, PhD appointed CSO; a late-breaking cardio-metabolic poster heads to the ADA Scientific Sessions.
Strip away the chemistry jargon and the mission is almost domestic: build drugs people can take for years without the cure becoming its own problem. Augustine's stated aim is selective HDAC6 inhibition that preserves the enzyme's good behavior - delivering disease-modifying therapies across neuromuscular, neurodegenerative and cardio-metabolic disease.
The leadership reflects the bet. CEO Gerhard Koenig brings drug-development mileage. CSO Rie Schultz Hansen anchors the science from Copenhagen. Chief Business Officer Andy Hu works the partnerships. CFO Virginie Cartage minds the runway. Around them, a team that fuses VIB-KU Leuven research depth with people who have moved molecules through the clinic before.
The diseases are chronic. So the drug has to be, too. That single constraint shapes every molecule they make.
- Why "selective" is the whole companyA safe, selective, oral HDAC6 inhibitor would not be one drug. It would be a key that fits several locks - CMT first, then cardio-metabolic disease, then neurodegeneration. That is the difference between a single rare-disease asset and a platform. The Phase I readout for AGT-100216 is the first real test of whether the lab's promise survives contact with human biology.
Back to where we started: a known target, in a quiet lab, getting a second chance. The difference now is that the second chance is no longer a hypothesis on a whiteboard. It is in a patient's bloodstream, being measured. The rest of the story depends on a number Augustine doesn't have yet - and is, right now, working to find out.
No verified company YouTube channel or product-demo video was found at publication; links above are the primary public record. Figures are drawn from company announcements and may be approximate.