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News about "investigational gene therapy "
Galibra Neuroscience has secured both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA for its investigational gene therapy for SSADH deficiency, strengthening regulatory support for a potential disease-modifying treatment aimed at addressing the genetic cause of the rare neurological disorder.
Investigational Gene Therapy | 06/08/2026 | By News Bureau
Genprex has partnered with Roche Diagnostics to validate TROP2 biomarkers for patient selection in non-small cell lung cancer trials, aiming to accelerate precision medicine and improve treatment outcomes.
Investigational Gene Therapy | 04/08/2026 | By News Bureau
AskBio Completes Enrollment for Phase 2 Gene Therapy Trial Targeting Heart Failure
The GenePHIT study evaluating investigational therapy AB-1002 for heart failure with reduced ejection fraction has fully enrolled 173 patients, with initial results expected in early 2027.
Investigational Gene Therapy | 02/04/2026 | By News Bureau | 135
Mahzi Therapeutics Doses First Patient in Phase I/II Trial of Gene Therapy for Pitt Hopkins Syndrome
Mahzi Therapeutics has dosed the first patient in its global Phase I/II UNITE study evaluating investigational gene therapy MZ-1866 for Pitt Hopkins syndrome, a rare neurogenetic disorder caused by TCF4 mutations.
Investigational Gene Therapy | 27/02/2026 | By News Bureau | 294
Genethon Partners with AskBio on Pompe Gene Therapy
The licensing deal enables AskBio to advance the clinical development of Genethon’s investigational gene therapy for Pompe disease through an industry partnership.
Investigational Gene Therapy | 15/01/2026 | By News Bureau | 195
AskBio Reports Safety Data from AB-1003 LGMD 2I/R9 Study
Data from first cohort of five participants suggest an acceptable safety profile for AB-1003 in participants with Limb-Girdle Muscular Dystrophy (LGMD) 2I/R9, with no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment.
Investigational Gene Therapy | 13/10/2025 | By Dineshwori | 252
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