Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company that focuses on developing innovative RNA-modulating drug candidates to address rare and ultra-rare premature stop codon diseases. These diseases stem from point mutations disrupting protein synthesis from messenger RNA, resulting in reduced or eliminated protein production from the affected allele and contributing to severe phenotypes in these genetic conditions. The company's read-through therapeutic development strategy aims to prolong mRNA half-life and enhance protein synthesis by enabling cytoplasmic ribosomes to read through premature stop codons, ultimately producing full-length proteins. The lead product candidate, ELX-02, is a small molecule drug designed to restore the production of fully functional proteins. This candidate is in the early stages of clinical development, with a focus on addressing cystic fibrosis and cystinosis. It's important to note that ELX-02 is an investigational drug that has yet to receive approvals from any global regulatory body. Established in 2013, Eloxx Pharmaceuticals is based in Waltham, MA, with research and development operations in Rehovot, Israel. Recently, the company secured a $2.00M Post-IPO Equity investment on 19 September 2023. While the specific investors for this round of funding are unspecified, this infusion of capital signifies continued support and confidence in Eloxx Pharmaceuticals' mission to address rare and ultra-rare diseases through innovative RNA-modulating drug candidates.