phase I clinical study
Dyne Reports on Response Predictive Biomarkers for Neurodegenerative Disorder Genetic Medicine
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The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.
In a Phase Ia trial in ovarian cancer, a strong neutralizing antibody response correlated with better disease control and longer survival.
Leucid Bio Begins Phase I Trial of CAR T-Cell Therapy in NKG2DL-Expressing Solid Tumors
The study tests the autologous CAR T-cell therapy LEU011 in patients who express NKG2DL in at least 10 percent of tumor cells.
HuidaGene Shares Early Data From First-in-Human Trial of Macular Degeneration RNA-Editing Therapy
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Researchers found the therapy downregulated VEGF expression and improved retinal thickness in neovascular age-related macular degeneration patients.
Capsida Gets FDA Clearance to Test Gene Therapy in Patients With Rare Epileptic Condition
The firm will test CAP-002 in a Phase I/IIa trial involving patients with syntaxin-binding protein 1 developmental and epileptic encephalopathy.
Apr 29, 2025
Apr 10, 2025
Apr 10, 2025