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Boehringer Ingelheim Pharma Inc. is a Ridgefield, CT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
VintaBio is a life sciences company that specializes in customized process development, GMP manufacturing, and quality control services for cell and gene therapies.
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.
Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions.
Regernon Pharmaceuticals Inc. is a Tarrytown, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.