HUIDAGENE THERAPEUTICS RECEIVES FDA RARE PEDIATRIC DISEASE DESIGNATION FOR HG004 TO TREAT INHERITED BLINDNESS
- Monday, August 7, 2023, 7:12
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HG004 is a one-time, direct-to-RPE treatment of inherited retinal disease caused by mutations in the RPE65 gene ~10-fold lower vector doses in multiregion, multicenter HG00402 trial than other AAV2 gene therapy clinical trials HG004 was previously granted orphan drug designation (ODD) for…
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