Cell therapy weekly: FDA exemptions and Breakthrough Therapy designations
This week: The US Food and Drug Administration (FDA; MD, USA) has granted a special exemption for an iPSC-derived cell therapy targeting Parkinson’s disease, along with Breakthrough Therapy designations for a CAR-T therapy for a pediatric brain tumor and a gene therapy for Huntington’s disease.
The news highlights:
- Special exemption for Parkinson’s disease cell therapy
- Pediatric brain tumor CAR-T therapy receives FDA Breakthrough Therapy designation
- FDA Breakthrough Therapy designation for Huntington’s disease gene therapy
Special exemption for Parkinson’s disease cell therapy
The FDA has granted XellSmart Biopharmaceutical (Suzhou, China) a special exemption to support a clinical trial of XS-411 Injection, the company’s allogeneic, off-the-shelf iPSC-derived dopaminergic neural progenitor cell therapy for Parkinson’s disease. This follows an Investigational New Drug submission in January 2025. In parallel, China’s National Medical Products Administration has approved XS-411 to enter Phase I clinical trials.
Additionally, the FDA has cleared XellSmart’s XS228, an allogeneic, off-the-shelf, clinical-grade iPSC-derived cell therapy for amyotrophic lateral sclerosis (ALS), to begin Phase I trials, marking it as the first-in-class regenerative neural cell therapy for the disease.
“XellSmart is committed to developing universal, off-the-shelf, allogeneic, clinical-grade iPSC-derived regenerative cell therapies to address central nervous system diseases with significant unmet clinical needs,” expressed Michael Lee, founder and CEO of XellSmart. “The FDA’s unconditional approval of the [Investigational New Drug applications] for XellSmart’s XS411 and XS228 products, targeting Parkinson’s disease and ALS, paves the way for these therapies to advance into clinical trials in the United States and lays the foundation for future commercialization in the global market.”
Pediatric brain tumor CAR-T therapy receives FDA Breakthrough Therapy designation
BCB-276, an autologous B7-H3 CAR T-cell therapy developed by BrainChild Bio (WA, USA) for children and young adults with diffuse intrinsic pontine glioma, has received FDA Breakthrough Therapy designation. The designation follows encouraging survival data from the BrainChild-03 Phase I trial, where BCB-276 showed promise in treating this high-grade brain tumor. With the designation in place, BrainChild Bio is now preparing to launch a Phase II trial, expected to begin in late 2025.
Michael Jensen, Founder and Chief Scientific Officer of BrainChild Bio, commented on the significance of this pivotal moment:
“This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating [central nervous system] brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases.”
FDA Breakthrough Therapy designation for Huntington’s disease gene therapy
uniQure biopharma B.V.’s (Amsterdam, Netherlands) AMT-130, an adeno-associated virus-based gene therapy for Huntington’s disease, has been granted FDA Breakthrough Therapy designation. The therapy was eligible for the designation after data from ongoing Phase I/II trials showed that AMT-130 significantly slowed disease progression.
“It’s a powerful recognition of the promise of AMT-130 and the important progress we’ve made,” said Walid Abi-Saab, Chief Medical Officer of uniQure. “We deeply value the FDA’s continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington’s disease patient community as quickly as possible.”
The FDA has also awarded AMT-130 Regenerative Medicine Advanced Therapy, Orphan Drug and Fast Track designations.
