Companies in #Pain

Korro Bio is a clinical-stage biopharmaceutical company developing genetic medicines based on RNA editing for rare and highly prevalent diseases. Its proprietary OPERA (Oligonucleotide Promoted Editing of RNA) platform uses GalNAc-conjugated oligonucleotides to recruit endogenous adenosine deaminase acting on RNA for precise, transient single-base edits, an approach designed to avoid the permanent DNA cuts associated with gene editing. The company's lead programme, KRRO-121, is in the Phase 1/2 ANCHOR trial in healthy volunteers for hyperammonemia associated with urea cycle disorders and hepatic encephalopathy; KRRO-111 is in late preclinical development for alpha-1 antitrypsin deficiency, targeting both the lung and liver manifestations of the disease, with a regulatory submission and first-in-human entry guided for the first half of 2027. Korro also has discovery programmes in neurologic and pain indications and a collaboration with Novo Nordisk covering cardiometabolic disease. The company is headquartered in Cambridge, Massachusetts and trades on the Nasdaq Global Market under the ticker KRRO.

Bresso, Italy-based CNS biopharmaceutical company developing evenamide, a glutamate modulator in Phase 3 for treatment-resistant schizophrenia, and marketing Xadago (safinamide) for Parkinson's disease.

Roswell, GA-based rare disease biotech developing therapies for pain, osteoarthritis, spasticity, and mitochondrial disorders, with a strategic focus on underserved CNS indications.

Discovery-based pharmaceutical company (Shionogi & Co., TYO:4507, Osaka, Japan, founded 1878) with US subsidiary marketing Fetroja (MDR Gram-negative antibiotic), Radicava ORS (ALS), and Xocova (COVID-19 antiviral).