Cell therapy weekly: biotech emerges from stealth with Parkinson’s disease program
This week: A biotechnology company specializing in autologous neuron replacement therapies for Parkinson’s disease and other neurodegenerative disorders emerged from stealth with data from their ongoing Phase Ib/IIa study, and Siren Biotechnology’s (CA, USA) investigational gene therapy for high-grade glioma received clearance of an investigator-initiated Investigational New Drug (IND) application. Plus, Gilead Sciences (CA, USA) has also acquired a biotechnology company focused on immune-mediated diseases.
The news highlights:
- Biotech emerges from stealth with Parkinson’s disease program
- T-cell engager biotechnology company acquired
- High-grade glioma gene therapy receives IND clearance
Biotech emerges from stealth with Parkinson’s disease program
Emerging from stealth, clinical-stage biotechnology company Oryon Cell Therapies (CA, USA) is on track to complete the ongoing Phase Ib/IIa clinical trial of its neuron replacement therapy program for Parkinson’s disease. After securing US$21 million, the company now has a total of US$42 million in funding, comprising both equity and grants.
The therapy involves using autologous blood cells, which are reprogrammed into pluripotent stem cells. These stem cells are then converted into dopaminergic neurons, the type that degenerates in Parkinson’s disease, and implanted directly into the putamen, the brain region primarily impacted by the disease.
At the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (17–21 March 2026; Copenhagen, Denmark), Oryon presented data from the ongoing Phase Ib/IIa study, which showed that the therapy was well-tolerated, with meaningful improvements in motor function and no need for immunosuppression.
“Oryon’s program emerged from 30 years of scientific advances in stem cell biology and neurosurgical techniques, including foundational Parkinson’s disease research conducted at Harvard University and Mass General Brigham,” said Ole Isacson, Co-founder of Oryon and Founding Director of the Neuroregeneration Research Institute at McLean Hospital/Harvard Medical School (MA, USA). “The major milestones we announced today reflect the tremendous progress we have made in moving this technology from the lab to the clinic and will allow us to accelerate it through its next stages of development.”
Breaking barriers in neurodegenerative disorders with gene therapy: a podcast with Deborah Ojutalayo
Int his podcast episode, Deborah Ojutalayo discusses gene therapies for neurodegenerative disorders, with a particular focus on targeted gene therapies for frontotemporal dementia caused by mutations in the GRN gene.
T-cell engager biotechnology company acquired
Gilead Sciences is to acquire Ouro Medicines (CA, USA) though a definitive agreement, adding OM366, an investigational BCMAxCD3 bispecific T-cell engager for the treatment of autoantibodies driven immune-mediated disease, to Gilead’s portfolio.
“This acquisition underscores our commitment to advancing transformative therapies for people living with serious autoimmune diseases,” elaborated Dietmar Berger, Chief Medical Officer of Gilead. “BCMA is a validated target with emerging data demonstrating potentially transformative outcomes in autoimmune diseases. BCMA-targeted T-cell engagers represent a differentiated approach with the potential to induce durable disease control. This novel framework complements our expanding inflammation pipeline and reflects our strategy to invest in innovative science that may redefine standards of care.”
Following this definitive agreement, Gilead is also in discussions with Galapagos NV (Mechelen, Belgium) to explore potential collaboration on research and development of the acquired Ouro Medicine assets.
High-grade glioma gene therapy receives IND clearance
The US Food and Drug Administration (FDA; MD, USA) has cleared an investigator-initiated IND application for Siren Biotechnology’s investigational gene therapy, SRN-101, to address adults with recurrent high-grade glioma.
Filed by the University of California, San Francisco (CA, USA) the cleared IND will enable the launch of a single-center, investigator-initiated Phase I study to evaluate the safety of gene therapy and provide translational data for future studies.
“This IND clearance allows us to begin evaluating this novel investigational approach in patients with recurrent high-grade glioma,” elaborated Nicholas Butowski, Professor of Neurological Surgery and Neuro-Oncology at University of California, San Francisco and who will lead the study. “This early-phase study will provide important safety and translational data to help inform future clinical development.”
