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Ocularis Pharma, Inc. is a Riverside, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Accumulus Synergy is a global, non-profit organization that is developing a transformative data exchange platform that aims to enable enhanced collaboration and efficiency between life sciences organizations and global health authorities, while also affording users the ability to extract dynamic, data-driven insights from knowledge management. Sponsored by leading biopharmaceutical companies, Accumulus Synergy was formed in 2020 to create innovative solutions that can reduce regulatory review times and transform global data exchange. The non-profit company is creating a first-of-its-kind cloud-based platform between the biopharmaceutical industry and health authorities worldwide. The single-platform approach aims to improve speed, transparencies and efficiencies in the regulatory process by leveraging advanced technology and tools for data exchange. This will help reduce the cost of innovation and ultimately bring patients safe and effective medicines faster.
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx`s lead product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel.
Angstrom Pharmaceuticals, Inc. is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions.