Platform

Individualized therapies, in reach for everyone

Gene editing technologies are a powerful and proven approach to treating rare genetic diseases with recent advances unlocking the ability to deliver these therapies at scale. Improvements in guide RNA design, AI editor engineering, and delivery systems now enable rapid creation of genetic medicines tailored to specific mutations.

Aurora will develop state-of-the-art CRISPR therapies, to precisely correct disease-causing mutations — and which can be customized for other variants of the same gene just by swapping in a different guide RNA.

Aurora’s umbrella approach leverages the intrinsic programmability of CRISPR gene editors to create a pathway for treating multiple rare mutations at once. Under this model, rare mutations underlying a disease or family of related diseases are bundled together, making it possible to economically scale treatments and advance them through a single, streamlined approval pathway. 

Reinventing the route to patients

We are innovating at each step of the drug development process to make disease-modifying, mutation-specific medicines a possibility for all patients with genetic diseases.

Therapeutic design:

Our leading gene-editing expertise and hands-on clinical experience with inborn errors of metabolism allow us to design highly precise and effective genomic medicines that permanently correct the underlying cause of disease with a single course of treatment.

Clinical development and CMC:

Aurora is designing clinical pathways to engineer and advance numerous gene editing therapies in parallel. And to lower the cost of delivering personalized genetic medicines, we are establishing modular GMP workflows for rapid, economical small-batch manufacturing and quality control.

Approval and commercialization:

The FDA is establishing a regulatory pathway that enables gene-editing treatments to be approved for multiple mutations under a single label. By signaling support for the iterative development of bespoke editors, the agency has paved the way for commercially available, personalized gene-editing therapies.