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Drug intelligence / Profile preview
ZA high dose
- Development stage
- Unknown
- Lead developer
- QLT
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
ZA high dose is the high-dose formulation of zuretinol acetate (also known as BBP-551, EB-109, EB-116, QLT-091001, or RGX-001), a synthetic small-molecule retinoid (9-cis-retinyl acetate) developed for the treatment of inherited retinal diseases (IRD). It is administered orally as a solution and acts as an ester prodrug of 9-cis-retinol, which is oxidized to 9-cis-retinal to replace deficient 11-cis-retinal in the visual cycle. By complexing with opsin to form isorhodopsin, it initiates the phototransduction cascade and prevents photoreceptor degeneration. The drug is primarily developed for Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) caused by biallelic recessive mutations in the RPE65 (retinoid isomerase) or LRAT (lecithin:retinol acyltransferase) genes. Originally developed by QLT and its successor Novelion Therapeutics, the program was licensed to Retinagenix Holdings (a BridgeBio company) and subsequently acquired by Eluminex Biosciences in January 2022.
- Other names
- 9-cis-retinol acetate9-cis-retinyl acetatezuretinolzuretinol acetatezuretinol acetate (high dose)zuretinol acetate oral solution (high dose)
02
Targets
RHO (Rhodopsin)
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