Evolving Gene Editing for Rare Disease Drug Development

Innovative approaches to gene editing hold the potential to transform the landscape of therapy for ultra-orphan conditions, which often lack effective treatments due to their rarity. Danaher is partnering with leading academic and clinical research institutes focused on the development of a novel platform approach to gene editing aimed at accelerating the delivery of therapies and creating economies of scale to treat ultra-orphan conditions.

Through our partnerships, we are ensuring the scalability of these advancements, creating efficient manufacturing models that facilitate broader access for patients in need. Additionally, we are formulating comprehensive regulatory and clinical strategies to navigate beyond N=1 complexities. Our work aims to enhance the therapeutic options available for affected individuals and establish a framework that may be applied to future research and development in the field of rare diseases.

In this presentation, you will learn about:

Developing novel gene editing to accelerate therapies for ultra-orphan conditions
Creating scalable models for efficient manufacturing and broader patient access
Formulating regulatory and clinical strategies to address rare disease challenges