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BioWorld - Sunday, December 28, 2025
Home » Topics » Gene therapy, BioWorld Science

Gene therapy, BioWorld Science
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Cancer

Structural variants activate contiguous oncogenes

Jan. 12, 2023
The alteration of the genome by mutations that...
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Eye and DNA illustration
Ocular

Sparingvision's gene therapy SPVN-06 to move into clinical studies for retinitis pigmentosa

Dec. 2, 2022
Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.
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Ocular

Opus Genetics cleared to enter clinic with OPGX-001 gene therapy for LCA5

Dec. 2, 2022
Opus Genetics Inc. has received FDA clearance for its IND application for a first-in-human phase I/II trial of OPGX-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). The trial is due to start in the U.S. early next year (ClinicalTrials.gov Identifier NCT05616793).
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Liver organoid
Drug Design, Drug Delivery & Technologies

Emulate launches new AAV transduction application for Liver-Chip

Sep. 20, 2022
Emulate Inc. has launched a new adeno-associated virus (AAV) transduction application for the Liver-Chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks.
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