Case StudyCELL AND GENE THERAPY
Case StudyCELL AND GENE THERAPY
Advancing the future of medicine.
The new modalities promise progress against the 7,000 diseases for which there is no effective treatment. But navigating nascent R&D, regulatory and reimbursement pathways remains a challenge. We’ve developed the strategy for the world’s leading cell and gene therapy advocacy organization, unpacked the GLP and GMP lab opportunity for these modalities, and directly shaped the regulatory and reimbursement pathways in the US and Europe.
Addressing a critical road block to
cell and gene therapy development
Objective
Potency-assay related delays in R&D are a major source of regulatory challenges for cell and gene therapy developers. We were asked by the Alliance for Regenerative Medicine (ARM) to convene developers and the FDA to explore solutions.
Our Approach
- Led preparatory discussions with developers and senior FDA staff to give structure and definition to a previously amorphous challenge.
- Designed and chaired an all-day meeting of developers and FDA representatives to explore specific case studies and chart a path forward.
Outcome
The workshop and the post-meeting debrief were livecast to over 1,000 cell and gene developers. After the working session, both developers and regulators acknowledged the need for improved communication and transparent regulatory expectations. Developers also challenged specific regulatory requirements, including the scientific basis for performing potency assays on every stage of a therapeutic cascade.
Final Report
Addressing potency-assay related development
delays for cell and gene therapies
delays for cell and gene therapies
“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)