The search for rare disease therapies

In the past decade, deals involving rare diseases have increased in size and value, supported by the growing number of opportunities based on novel therapeutic platforms.

Horizon Therapeutics has transformed into a fast-growing biotech while staying true to its guiding principles.

With its Healnet platform, Healx pairs cutting-edge AI with industry-leading drug discovery and rare disease expertise to identify new uses for known compounds. The company now has over 20 programs in development.

With its lead phase 3 candidate Nefecon in development for the treatment of primary immunoglobulin A nephropathy and a late-stage orphan focused NOX program, Calliditas Therapeutics is pioneering new rare-disease therapies.

Global biotechnology company BioMarin focuses on developing and commercializing innovative therapies through a range of modalities. The company’s diverse pipeline, spanning exploratory to marketed programs, relies heavily on its genetic and genomic expertise, manufacturing capabilities, and long-term partnering models.

Chameleon Biosciences is developing next-generation adeno-associated virus vectors that elicit minimal immune responses, allowing repeat dosing and resulting in superior efficacies. These EVADER vectors have universal application for existing adeno-associated virus vectors, and Chameleon is seeking partners to advance its lead program in severe hemophilia B and to programs that target retinal cells.

With its multi-functional drug discovery engine that combines digital and human-based platforms, such as AnalytiX and microOrgan, Vyant Bio is accelerating the drug discovery process from ID to IND.