Gene Therapy Pipeline Reaches Over 1,000 Trials for Rare Diseases

Published on Spencer Knight via LinkedIn

There are more than 300 million people worldwide living with rare diseases, many of them with no approved treatments. Spencer Knight highlights the growing impact of gene therapy in this space, with over 1,000 active therapies in development and several already approved.

While gene therapy offers promising cures by targeting the root genetic cause, access, pricing, and commercialization still present major hurdles. Knight calls for continued efforts to ensure these treatments reach the people who need them, especially for ultra-rare genetic disorders where awareness and funding are limited.

Read the full post on LinkedIn:

👉 Spencer Knight: Approved Gene Therapies for Rare Diseases

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