The Promise of Gene Therapy Offers More Hope for Rare Disease Cures in 2022

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While the field of gene therapy is still relatively new, it’s moving at a breakneck pace. In fact, 1,745 gene therapies are currently in development around the world! Technological advancements in gene editing, modulation, and replacement technologies have helped to advance this new breed of therapies, raising hope for cures for many of these rare diseases in the near future.

5 Questions with Kayla Valdes, Ph.D. Senior Manager, Horizon Therapeutics

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Dr. Kayla Valdes is a Senior Manager at Horizon Therapeutics, a biopharmaceutical company focused on researching, developing, and commercializing medicines that address critical needs for people impacted by rare diseases. Prior to joining Horizon, Dr. Valdes worked at the National Institutes of Health as a Senior Program Manager on the Cure Sickle Cell Initiative. She was also the Associate Director of Scientific Programs at the Drug Information Association (DIA), where she developed DIA’s global content strategy by providing scientific expertise in therapeutic drug development and regulatory policy.