Cell therapy weekly: BLA submission planned for Huntington’s gene therapy AMT-130
This week: uniQure (Amsterdam, Netherlands) has announced plans to submit a Biologics License Application (BLA) for AMT-130 in Huntington’s disease. Ernexa Therapeutics (MA, USA) has confirmed that it remains on track to submit an IND for its induced mesenchymal stem cell therapy and is well-positioned to initiate its first-in-human Phase I clinical trial. Plus, Autolus Therapeutics (London, UK) has presented data from its ongoing CARLYSLE trial evaluating obecabtagene autoleucel (obe-cel) in severe refractory systemic lupus erythematosus.
The news highlights:
- BLA submission planned for Huntington’s gene therapy AMT-130
- Induced mesenchymal stem cell therapy IND in preparation
- Encouraging early data on Phase I Lupus CAR-T
BLA submission planned for Huntington’s gene therapy AMT-130
uniQure has announced plans to submit a BLA for AMT-130, a gene therapy for Huntington’s disease, following a recent Type B meeting with the US Food and Drug Administration (FDA; MD, USA). The company is conducting two multi-center, dose-escalating Phase I/II clinical studies to evaluate the safety, tolerability and potential effectiveness of AMT-130.
The FDA indicated that 3-year data from these studies would be sufficient to support an accelerated approval application. To demonstrate efficacy, uniQure will compare results from the first two patient cohorts against a matched external control group from the Enroll-HD natural history database – a large registry tracking Huntington’s disease progression. This comparison will follow a prespecified statistical analysis plan.
Before submitting the BLA, the FDA and uniQure will finalize the design of a confirmatory study required for full approval. The FDA has suggested using a concurrent control group receiving standard-of-care therapy rather than a sham surgical procedure, which would be less invasive for patients.
“Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” commented Matt Kapusta, CEO at uniQure. “The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study. The consistency and strength of the clinical data generated to date give us great confidence in the product’s potential to make a meaningful difference for patients. We remain focused on bringing AMT-130 to patients and families as quickly and responsibly as possible in the US and globally.”
uniQure intends to submit the BLA in the third quarter of 2026.
Induced mesenchymal stem cell therapy IND in preparation
Ernexa Therapeutics has revealed that it’s well-positioned to initiate its first-in-human Phase I clinical study by the end of 2026, which will mark its transition into a clinical-stage biotechnology company. The company has made significant progress towards a Phase I trial as it has completed manufacturing process development and has GMP production underway. Alongside this, Ernexa is advancing IND-enabling activities and preparing for an IND submission to support its stem cell therapy, ERNA-101. ERNA-101 is a modified allogeneic induced mesenchymal stem cell therapy derived from induced pluripotent stem cells, designed to selectively home to tumors and deliver a proprietary IL-7/IL-15 fusion cytokine directly into the tumor microenvironment. Ernexa’s initial focus is developing ERNA-101 for the treatment of ovarian cancer.
“ERNA-101 continues to advance toward the clinic as we execute across manufacturing, regulatory and clinical development activities,” shared Sanjeev Luther, President and CEO of Ernexa Therapeutics. “With GMP manufacturing underway, technology transfer activities progressing and our IND submission expected in the third quarter of 2026, we believe we are entering one of the most important periods in the Company’s history.”
Encouraging early data on Phase I Lupus CAR-T
University College London (UCL; UK) spinout Autolus Therapeutics presented data from its ongoing CARLYSLE trial assessing obe-cel in severe refractory systemic lupus erythematosus at the EULAR European Congress of Rheumatology (3–6 June 2026; London, UK). CARLYSLE is a Phase I trial enrolling patients aged 12 to 65 with active, severe lupus who have not responded to multiple standard treatments. While their findings are preliminary and based on a small patient cohort, they highlight the potential of CAR-T therapy as a novel approach for severe lupus.
The early data demonstrated a favorable safety profile, with no cases of immune effector cell-associated neurotoxicity syndrome and no moderate or severe cytokine release syndrome observed. Alongside these encouraging safety results, researchers observed signs of clinical benefit, suggesting that the therapy may enable an “immune reset” rather than simply suppressing the immune system.
“For patients living with severe lupus that has not responded to existing treatments, the options can be very limited. These findings provide early evidence that CAR T-cell therapy may be able to reset the immune system and drive meaningful clinical improvements after a single treatment,” explained Claire Roddie, an investigator on the trial from UCL Cancer Institute. “Although these are still early days, the results mark an important step forward in exploring how cellular therapies could transform the treatment of autoimmune diseases.”
