Travere Therapeutics

Orphan Technologies Ltd. is a clinical-stage biopharmaceutical company based in Rapperswil, Switzerland, focused on developing innovative therapies for patients with rare disorders, particularly classical homocystinuria (HCU). Incorporated in 2016, the company aims to alleviate the challenges faced by individuals suffering from HCU, a genetic condition that leads to serious cardiovascular, skeletal, neurologic, and ophthalmologic complications. Its lead drug candidate, OT-58, is an optimized enzyme replacement therapy designed to effectively lower homocysteine levels through a targeted mechanism. This therapy not only addresses the symptoms of classical homocystinuria but also holds potential for therapeutic applications in other related diseases. Orphan Technologies operates as a subsidiary of Travere Therapeutics, Inc.

Perlara, PBC is a biotechnology company based in San Francisco, California, that focuses on discovering personalized treatments for rare and common diseases. Incorporated in 2014, Perlara operates as a Public Benefit Corporation and utilizes a unique drug discovery platform that leverages CRISPR-engineered animal models, including yeast, nematodes, fruit flies, and zebrafish. This innovative approach allows for whole-organism phenotypic screening, enabling the rapid and cost-effective identification of orphan drug candidates for over 5,000 rare genetic diseases. The company’s lead programs target Niemann-Pick Type C, a lysosomal storage disorder, and NGLY1 Deficiency, a congenital disorder of glycosylation. Perlara’s pipeline also includes candidates for additional lysosomal storage disorders, mitochondrial diseases, and peroxisomal biogenesis disorders, thereby contributing significantly to advancements in precision drug discovery.

Manchester Pharmaceuticals is a specialty pharmaceutical company dedicated to the identification, development, FDA approval, and commercialization of innovative therapeutic modalities aimed at addressing the unique needs of patients with ultra-rare diseases. By focusing on rare diseases, the company seeks to provide effective treatment options for conditions that often lack adequate therapies, thereby improving patient outcomes in this underserved segment of healthcare.