Case StudyCELL AND GENE THERAPY
Case StudyCELL AND GENE THERAPY
Advancing the future of medicine.
Gene editing holds great promise to address the 10,000 diseases for which there is no effective treatment. However, creating a fit-for-purpose regulatory environment is needed to continue therapeutic advances.
Developing fit-for-purpose gene
editing frameworks for rare disease
Objective
Gene editing treatments for rare diseases face viability challenges due to small patient populations and legacy regulatory frameworks. These regulatory approaches require updating for gene editing to fulfill its potential.
Our Approach
- Galen/Atlantica was engaged by the Alliance for Regenerative Medicine, the International Society for Cell and Gene Therapy, and Danaher Corporation.
- We worked with developers to provide the FDA with gene editing platform examples that leverage stable and reusable processes or components, de-risking this new set of modalities.
- We designed and chaired a series of discussions with the FDA, developers, and experts. The discussions identified opportunities that could yield up to 5-fold efficiency gains, collectively decreasing the time required to dose patients with a new gene-editing therapy from years down to 6 months.
Outcome
The workshop and the post-meeting debrief led to peer-reviewed publications in Cytotherapy and Nature Biotechnology and informed FDA Draft Guidance on developing gene editing therapies.
Final Report
Advancing gene editing platforms
“Galen/Atlantica has the rare ability to bring a human element and disruptive solutions to intractable problems, cutting the Gordian knots impeding progress.”
Tim Hunt
CEO of the Alliance for Regenerative Medicine (ARM)