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Details:
HG004 is an investigational gene replacement therapy product. It uses the recombinant AAV9 vector to deliver a functional human RPE65 gene to the retina and to restore, treat, and prevent blindness of children and adults with RPE65-Inherited Retinal Diseases.
Lead Product(s): HG004
Therapeutic Area: Rare Diseases and Disorders Product Name: HG004
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 01, 2023
Details:
HG004 is a one-time, direct-to-RPE treatment of inherited retinal disease caused by mutations in the RPE65 gene and was previously granted orphan drug designation for RPE65-IRD.
Lead Product(s): HG004
Therapeutic Area: Ophthalmology Product Name: HG004
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 07, 2023
Details:
HG004 injection is a new ophthalmic gene therapy drug designed to treat RPE65 gene mutation-related retinopathy. It has successfully obtained the IND approval from the US FDA
Lead Product(s): HG004
Therapeutic Area: Ophthalmology Product Name: HG004
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 18, 2023
Details:
HG004 is a gene replacement therapy drug which uses the recombinant non-adeno-associated virus serotype 2 vector to deliver a functional human RPE65 gene to the retina to restore, treat, and prevent blindness of children and adults with RPE65 mutation-associated IRDs.
Lead Product(s): HG004
Therapeutic Area: Ophthalmology Product Name: HG004
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 03, 2023
Details:
HG004 is an ophthalmic injection intended for the treatment of RPE65 gene mutation-related retinopathy. Based on direct preclinical comparative studies of HG004 and AAV2 in retinal function, HG004 is at least 10 times more efficient at transducing retinal pigment epithelium.
Lead Product(s): HG004
Therapeutic Area: Ophthalmology Product Name: HG004
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 27, 2023