Eloxx is evaluating several of its eukaryotic ribosomal selective glycoside (ERSG) molecules in IND-enabling studies for use in the treatment of inherited retinal diseases with an initial focus on Usher Syndrome
Preclinical studies have demonstrated:
dose-dependent restoration of missing protein of Usher Syndrome nonsense mutations;
encouraging pharmacokinetics in the retina by intravitreal injection;
a favorable tolerability profile at high doses in sensitive species
Eloxx also made a presentation at the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit on
“We are very pleased with the preclinical results for several of our ERSG compounds demonstrating that they are suitable for intravitreal injection, well-tolerated in a sensitive model species, active against Usher Syndrome mutations and capable of reaching the retina. We look forward to advancing IND-enabling studies in inherited retinal diseases, with an initial focus on Usher Syndrome,” said Dr.
In a Poster presentation at the ARVO Annual Meeting on
- Eloxx has screened multiple compounds from its ERSG library of read-through agents for potential use in the treatment of inherited retinal disorders with an initial focus on Usher Syndrome, beginning with USH2A.
- Multiple Eloxx compounds in preclinical studies have demonstrated:
- Dose-dependent activity against Usher mutations.
- Restoration of missing Usher Syndrome protein.
- Favorable tolerability profile at high doses in sensitive species; preserved electroretinogram (ERG) and no compound-related histopathological changes.
- Encouraging pharmacokinetics demonstrating retina exposure by intravitreal injection.
- IND-enabling studies ongoing.
- Next steps include evaluation of patient-derived cell models and sustained release formulations.
At the request of the Foundation Fighting Blindness, Dr.
Eloxx compounds are investigational agents not approved by any regulatory agency for therapeutic use.
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Contact:
Barbarar@eloxxpharma.com
(203) 274-2825
SOURCE:
Source: Eloxx Pharmaceuticals