With the MEB Science Day focusing on platform technologies, it’s clear there’s strong interest both from innovators and from regulators looking to streamline implementation. Platform approaches could help reduce the development time and ease regulatory complexity for advanced therapies, while also opening up opportunities to develop treatments for (ultra-)rare diseases.
In that context, the recent NEJM article, Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease (link), offers a excellent example of what’s possible. They describe how a modular CRISPR platform enabled the rapid design, production, and in vivo delivery of a personalized therapy, all within just six months. Delivered under compassionate use, establishing a clear pathway to market authorization for remains a significant challenge.
To me this raises several questions:
What defines a truly modular gene-editing platform?
Which elements (e.g., vector backbone, guide design, delivery system) can be reused, and what still requires case-by-case customization?
Can these modular platforms scale beyond ultra-rare indications?
What are the main barriers — manufacturing, cost, or regulatory — to broader application?
How do we move from experimental use to formal market authorization?
Could regulatory frameworks evolve to accommodate individualized or semi-bespoke therapies under a platform-based approval model?
Would love to hear your thoughts: How can we turn these one-off successes into a pathway to patient treatment?