Eloxx Pharmaceuticals develops small molecule RNA-modulating therapies that target premature stop codon mutations, which disrupt protein synthesis in over 1,800 rare and ultra-rare diseases. Their lead candidate, ELX-02, aims to restore full-length protein production in conditions such as cystic fibrosis and cystinosis, addressing the critical lack of functional proteins in affected patients.
Funding
$2M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare and ultra-rare genetic diseases are caused by premature stop codon mutations, which interrupt the normal protein synthesis process, leading to reduced or absent production of functional proteins. There is a significant unmet need for therapies that can address these mutations and restore protein production.
Solution
Eloxx Pharmaceuticals is developing small molecule RNA-modulating therapies designed to treat rare and ultra-rare diseases caused by premature stop codons. Their approach focuses on enabling the ribosome to read through these premature stop codons, thereby restoring the production of full-length, functional proteins. Eloxx utilizes its Turbo-ZM™ platform and Ribosome Modulating Agents (RMAs) along with Eukaryotic Ribosomal Selective Glycosides (ERSGs) to target nonsense mutations implicated in various rare diseases and ribosomal mutations driving cancer. The lead product candidate, ELX-02, is currently in clinical development for conditions such as Alport Syndrome, Cystic Fibrosis and Cystinosis.
Target Audience
The primary target audience includes patients with rare and ultra-rare genetic diseases caused by premature stop codon mutations, as well as the physicians and researchers who treat and study these conditions.
Features
- Turbo-ZM™ platform for the discovery and development of RNA-modulating therapies.
- Ribosome Modulating Agents (RMAs) designed to promote read-through of premature stop codons.
- Eukaryotic Ribosomal Selective Glycosides (ERSGs) for targeting ribosomal mutations.
- ELX-02, a small molecule drug candidate in clinical development.
- Focus on restoring full-length protein production in patients with premature stop codon diseases.