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About Ultragenyx Pharmaceutical

Ultragenyx is a leader in the rare disease space, with a mission to bring transformative treatments to patients with severe, life-altering genetic conditions. The company has successfully commercialized products such as Crysvita® (burosumab) for XLH and Dojolvi® for long-chain fatty acid oxidation disorders (LC-FAOD), while advancing a deep pipeline of gene therapy, mRNA, and other biologic candidates. Its strategy focuses on leveraging multiple technology platforms to address high-unmet-need diseases, supported by strategic partnerships and a global commercial footprint.

About Abeona Therapeutics

Abeona Therapeutics is dedicated to delivering transformative cell and gene therapies for patients with severe rare diseases. The company has achieved a significant milestone with the commercial launch of ZEVASKYN® and maintains a diverse pipeline spanning preclinical to Phase 1/2 development, primarily targeting inherited retinal diseases and neurological conditions. Its strategy is supported by in-house manufacturing capabilities at The Elisa Linton Center for Rare Disease Therapies and key partnerships with Ultragenyx and Taysha Gene Therapies.

Therapeutic Areas