Sustained visual improvements in LHON patients treated with AAV gene therapy
A multinational study led by the LHON Study Group has revealed sustained visual improvements and a favorable safety profile ...
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Sensorion Announces Completion of Enrollment of The First Cohort of The Audiogene Phase 1/2 ...
The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the ...
Krystal Biotech: Blockbuster Ambitions For Topical Gene Therapy
Krystal Biotech's VYJUVEK, a groundbreaking gene therapy for DEB, achieved $83.8M in Q3 2024 sales. Read why I rate KRYS ...