Perth-based biotech Miro Sciences has cleared a Therapeutic Goods Administration (TGA) audit of its manufacturing partner, Brisbane's Q-Gen Cell Therapeutics, de-risking a key step in scaling up production of its StemSmart stem cell therapy ahead of a planned phase two clinical trial for refractory Crohn's disease.
Miro’s chief scientific officer and inventor of the StemSmart technology, Dr Marian Sturm, said the inspection demonstrates the company is on target with its technology transfer and represents an important step towards TGA licensure of the product. “It is a significant milestone in our preparations for the upcoming clinical trial in refractory Crohn’s disease,” she said.
Refractory Crohn’s disease is a chronic, incurable condition affecting an estimated 6–8 million people worldwide, with around 30 per cent of patients failing to respond to conventional therapies. A similar proportion develop fistulas, abnormal tracts that can form between the bowel and other organs. A previous phase two trial in refractory Crohn’s disease suggested StemSmart was potent, safe and efficacious, with a 78 per cent overall clinical response rate and 44 per cent of patients achieving remission.
The technology has its roots in Western Australia, having been developed at Royal Perth Hospital using stem cells sourced from adult human donor bone marrow. The patented process is designed to supercharge the cells’ therapeutic activity to rein in overactive immune responses. As universal donor cells, they require no patient matching, positioning StemSmart as a potential off-the-shelf intravenous infusion.
Miro contracted Brisbane-based Q-Gen Cell Therapeutics to manufacture the therapy. Q-Gen, based at the QIMR Berghofer Medical Research Institute, is one of the largest cell therapy contract manufacturers in Australia. It holds its own TGA licence for cell therapy manufacturing, has over 25 years of industry experience, and operates 13 dedicated cleanrooms.
The focus on Crohn’s disease represents a significant strategic pivot for Miro, which spent years developing its EmtinB peptide for neurodegenerative conditions. It acquired the StemSmart technology platform in mid-2025 and rapidly reoriented its clinical focus.
In October, the company received positive feedback from the United States Food and Drug Administration following a Pre-Investigational New Drug submission meeting. It has also appointed clinical research organisation Southern Star Research to conduct the trial, which is planned to include patients in both Australia and the US, subject to regulatory approvals. Miro aims to kick off the phase two trial before the end of 2026.
Analysis
Why This Matters
- For the millions of patients worldwide with refractory Crohn's disease, if successful, StemSmart could represent a new, readily available treatment option where current therapies fail.
- The successful TGA audit reinforces the viability of Australia's contract manufacturing ecosystem for advanced cell therapies, potentially attracting more international biotech firms to partner with local facilities like Q-Gen.
- The promising 78 per cent response rate from early trials suggests the science of mesenchymal stem cells for inflammatory disease is maturing, though robust phase two data is still needed to prove the technology's therapeutic potential.
Background
Miro Sciences (formerly NeuroScientific Biopharmaceuticals) is a Perth-based biotech developing StemSmart, a patented stem cell therapy derived from adult human donor bone marrow. The therapy aims to modulate overactive immune responses responsible for severe inflammatory conditions like refractory Crohn's disease. The company pivoted to this technology in mid-2025, acquiring the platform after years of research into a neurodegenerative peptide called EmtinB. The field of mesenchymal stem cell (MSC) research for inflammatory bowel disease is still in its infancy, with external studies indicating MSCs account for more than 70 per cent of stem-cell interventions in clinical trials for the condition globally.
What to Watch
- The commencement of the phase two clinical trial in refractory Crohn's disease, which Miro Sciences targets to begin before the end of 2026.
- The formal TGA licensure of the StemSmart MSC product, for which this regulatory audit is a direct precursor.
- Whether Miro secures the necessary US regulatory approvals to expand the trial to patients in the United States alongside the Australian cohort.
Checked
12 claims in this story checked against 1 source: 12 supported.
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- SupportedMiro cleared a TGA audit of its manufacturing partner for StemSmart, advancing preparations for a Crohn’s disease trial.The Age - Latest News: “Miro Sciences, formerly NeuroScientific Biopharmaceuticals, has cleared a significant manufacturing hurdle ... after Australia’s Therapeutic Goods Administration (TGA) completed an audit of its manufacturing partner’s facilities.”
- SupportedThe company says the inspection paves the way for scaling up clinical-grade StemSmart production ahead of its planned phase two trial.The Age - Latest News: “The company says the milestone paves the way for scaling up clinical-grade StemSmart production ahead of its planned phase two clinical trial”
- SupportedThe audit was at Brisbane-based Q-Gen Cell Therapeutics and covered production of StemSmart.The Age - Latest News: “The inspection at Brisbane-based Q-Gen Cell Therapeutics covered the production of Miro’s “StemSmart” product”
- SupportedDr Marian Sturm said the inspection was a significant milestone in preparations for the upcoming refractory Crohn’s disease trial.The Age - Latest News: “It is a significant milestone in our preparations for the upcoming clinical trial in refractory Crohn’s disease”
- SupportedRefractory Crohn’s disease affects an estimated 6–8 million people worldwide.The Age - Latest News: “a lifelong, incurable condition involving chronic gut inflammation that affects an estimated 6–8 million people worldwide.”
- SupportedAround 30 per cent of patients do not respond to conventional therapies.The Age - Latest News: “Around 30 per cent of patients become refractory, meaning their disease no longer responds to conventional therapies.”
- SupportedA similar proportion of patients develop fistulas, abnormal tracts that can form between the bowel and other organs.The Age - Latest News: “A similar proportion develop fistulas — abnormal tracts that can form between the bowel and other organs or the skin.”
- SupportedA previous phase two trial suggested StemSmart was potent, safe and efficacious, with a 78 per cent overall clinical response rate.The Age - Latest News: “A previous phase two trial in refractory Crohn’s disease suggested Miro’s StemSmart MSCs were potent, safe and efficacious, reporting a hefty 78 per cent overall clinical response rate”
- SupportedIn that trial, 44 per cent of patients achieved remission.The Age - Latest News: “with an impressive 44 per cent of patients achieving remission.”
- SupportedStemSmart was developed at Royal Perth Hospital using stem cells from adult human donor bone marrow.The Age - Latest News: “Miro’s technology has its roots in Western Australia, having been developed at Royal Perth Hospital using stem cells sourced from adult human donor bone marrow.”
- SupportedQ-Gen is one of Australia’s largest cell therapy contract manufacturers, has more than 25 years of industry experience and operates 13 dedicated cleanrooms.The Age - Latest News: “Q-Gen is one of the biggest cell therapy contract manufacturers in Australia. The company holds its own TGA licence for cell therapy manufacturing, has more than 25 years of industry experience, and operates 13 dedicated cleanrooms”
- SupportedMiro acquired the StemSmart platform in mid-2025, received positive FDA feedback in October, and aims to start the phase two trial before the end of 2026.The Age - Latest News: “After acquiring the StemSmart technology platform in mid-2025”