Cell therapy weekly: advances in personalized stem cell therapy for Parkinson’s disease
This week: A collaborative research project looks to advance the development of a gene therapy for a fatal lysosomal storage disorder and a trial assessing a personalized stem cell therapy for Parkinson’s disease demonstrates positive interim results. Plus, the US Food and Drug Administration (FDA; MD, USA) granted supplemental approval for Casgevy in pediatric sickle cell disease and β-thalassemia.
The news highlights:
- Collaborative research to develop lysosomal storage disorder gene therapy
- Advances in personalized stem cell therapy for Parkinson’s disease
- FDA expands Casgevy age range to include younger patients
Collaborative research to develop lysosomal storage disorder gene therapy
Biotechnology company Apertura Gene Therapy (NY, USA) announced that it has entered into a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Human Genome Research Institute to evaluate the systemic delivery of an investigational gene therapy for Niemann-Pick Disease Type C1 (NPC1).
NPC1, a rare, fatal, autosomal recessive lysosomal storage disorder, results from a mutation in the NPC1 gene. This disorder leads to neurodegeneration in early childhood, progressive cerebellar ataxia, dementia and often death in adolescence.
The research will use Apertura’s TfR1 CapX™, a proprietary AAV capsid designed to efficiently cross the blood–brain barrier by targeting transferrin receptor 1, enabling systemic delivery to the brain and spinal cord.
“Systemic delivery of AAV gene therapies by intravenous administration has significant advantages over other methods of administration due to lower complexity and risks,” said Forbes D. Porter, Senior Investigator of the Section on Molecular Dysmorphology at NICHD and a Principal Investigator of the agreement. “Our goal is to identify a therapy that significantly slows neurodegeneration in individuals with NPC1, and this collaboration will allow us to explore the potential therapeutic efficacy of systemic administration of this novel AAV capsid.”
Advances in personalized stem cell therapy for Parkinson’s disease
Scripps Research (CA, USA) presented encouraging interim clinical data at the International Society for Stem Cell Research meeting (ISSCR; Montréal, Canada; July 8–11, 2026) from its ongoing Phase I/IIa trial evaluating an autologous iPSC-derived dopamine neuron precursor transplantation for sporadic Parkinson’s disease. This is the first Phase I/IIa trial using this approach to transplant therapy into the dopamine-depleted brain region.
Interim results were reported for eight patients who were evaluated 12 months after transplantation.
“The greatest challenge for autologous cell therapy has been developing reliable methods to qualify every patient’s cells for treatment,” explained Jeanne Loring (Scripps Research), who presented the findings. “We developed genomic analysis approaches that address cell line heterogeneity and identify mutations that can arise as iPSCs divide, helping establish a rigorous framework for manufacturing personalized therapies.”
FDA expands Casgevy age range to include younger patients
The FDA has approved the gene therapy Casgevy for patients aged 2 years and older who have sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia.
“With today’s decision, pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,” said Karim Mikhail, Acting Director of the FDA’s Center for Biologics Evaluation and Research. “The FDA is committed to prioritizing and speeding up the review of products that address critical U.S health priorities through expedited review programs, including the FDA Commissioner’s National Priority Voucher Pilot Program. These initiatives are designed to advance therapies for diseases with significant unmet medical needs, enabling faster access to innovative treatments while upholding the FDA’s rigorous gold-standard requirements for safety and effectiveness.”
