orphan-drugs
(2)
Shorla Oncology
Shorla Oncology is a specialty pharmaceutical company based in Clonmel, Ireland, with a U.S. base in Cambridge, Massachusetts, that develops and commercializes innovative oncology drugs for orphan and pediatric cancers. Founded in 2018 by Sharon Cunningham and Orlaith Ryan, Shorla focuses on indications where existing treatments are limited, in shortage, or poorly suited to patients - reformulating and reinventing cancer therapies into easier-to-administer forms such as oral liquids and ready-to-dilute vials. The company markets four FDA-approved products in the U.S. and is advancing an early-stage pipeline addressing rare cancers.
Mendra
Mendra is an AI-native biopharmaceutical company based in San Francisco that acquires, develops, and commercializes therapies for rare and ultra-rare diseases with high unmet need. Founded in 2025 and launched publicly in January 2026 with an $82 million Series A, the company is run by veterans of BioMarin, Palantir, and other rare disease programs. Rather than discovering compounds from scratch, Mendra systematically scans, scores, and acquires shelved or under-resourced rare disease assets from academia, small biotechs, and big pharma, then uses AI to accelerate patient identification, clinical trial enrollment, and global market access.