Cell therapy weekly: breakthrough in bioprinted adrenal tissues
This week: Preclinical data demonstrated the potential of bioprinted adrenal tissues for primary adrenal insufficiency, Broken String Biosciences (Cambridge, UK) and BioLizard (IL, USA) partnered to develop an AI tool to improve gene editing, and Genascence Corporation (CA, USA) received Regenerative Medicine Advanced Therapy designation for its gene therapy for knee osteoarthritis.
The news highlights:
- Preclinical data on bioprinted adrenal tissues
- AI-powered gene editing
- Gene therapy for knee osteoarthritis
Preclinical data on bioprinted adrenal tissues
At ENDO 2025 (12-15 July; San Francisco, CA, USA), Aspect Biosystems (Vancouver, Canada) presented preclinical data demonstrating that the company’s bioprinted tissue therapeutics (BTTs) can restore adrenal function in animal models of primary adrenal insufficiency.
BTTs containing human adrenal cells were implanted into adrenalectomized mice and successfully produced cortisol in response to adrenocorticotropic hormone. The BTTs maintained function for over six months and improved survival rates in the mice.
“Our research shows that Aspect’s adrenal BTTs successfully replicate healthy human adrenal gland function by releasing cortisol in a pattern that follows the natural daily rhythms of hormone release and by responding appropriately to a stimulus that mimics a stress response,” said Sam Wadsworth, Chief Scientific Officer at Aspect Biosystems. “These results demonstrate that our off-the-shelf, implantable cell therapy can work in harmony with the body’s physiology and has real potential to serve as a functional cure for primary adrenal insufficiency.”
AI-powered gene editing
Broken String Biosciences and BioLizard have partnered to create SafeGuide, an AI tool that improves the selection of guide RNAs (gRNAs) for gene editing technologies like CRISPR. This collaboration aims to solve a critical challenge in gene therapy development.
The tool helps scientists identify optimal gRNAs that direct CRISPR precisely to the correct DNA locations, potentially reducing development time, cutting costs and decreasing reliance on expensive laboratory testing. The technology combines Broken String’s INDUCE-seq platform, which precisely maps where DNA editing occurs, with BioLizard’s AI expertise to create algorithms that can predict which gRNAs will be both effective and safe.
Felix Dobbs, CEO at Broken String Biosciences stated: “This collaboration with BioLizard will set a new standard for safety in genome editing. By using AI to predict and distinguish natural and unnatural DNA break patterns, human intervention is significantly reduced, while safety increases.”
Gene therapy for knee osteoarthritis
The FDA has granted Regenerative Medicine Advanced Therapy designation to Genascence’s gene therapy for knee osteoarthritis, GNSC-001. The gene therapy utilizes an AAV vector to deliver an optimized human interleukin-1 receptor antagonist, which blocks interleukin-1 signaling, one of the key mediators of osteoarthritis pathogenesis.
Thomas Chalberg, founder and CEO of Genascence, stated: “With the [Regenerative Medicine Advanced Therapy] designation for GNSC-001, we look forward to working closely with the FDA as we seek to accelerate late-stage clinical development of GNSC-001 so we can bring a new treatment option to people suffering from this incapacitating, disabling disease.”