Achieving advancement in ATMP regulation through disruptive innovation of European healthcare systems

Partnerships and transformation are a large part of our evolving biotech landscape. Not surprisingly, they were two common themes at this year’s Meeting on the Med in Barcelona.  As an example, attendees discussed how the lack of harmonization across EU member states has led to fragmented access to advanced therapy medicinal products (ATMPs), which varies from country to country. Chris Fox, president of Novartis Gene Therapies, used her company’s product, ZOLGENSMA®, as an example. It’s approved for use in 48 countries but is only accessible in 35. 

A new means to fight stem cell transplant infections

When attending a large event such as Advanced Therapies Week, you are able to reach outside your regular workspace and learn about numerous developments happening in other spaces. One in particular that I learned of is an exciting advance in a new ex vivo cell therapy from Smart Immune. It's designed to help those who receive allogenic bone marrow transplants as part of treatment for cancer, such as leukemia, lymphoma, or myeloma, which can leave a patient in an immunosuppressive state.

Downstream challenges in mRNA production

In my previous post, I brought up important questions that should be asked when beginning to design a scalable manufacturing process to meet specific use requirements. In this post, some of the difficulties encountered in process development are reviewed.

Production paths to mRNA

My last post gave a short introduction to the manufacture of in vitro transcribed (IVT) messenger RNA (mRNA) for vaccines and therapies, outlining the steps from preclinical to clinical stages of production. In this post, I’ll touch on manufacturing considerations, beginning with the upstream process for IVT mRNA.

Both immunotherapy and regenerative medicine are experiencing explosive growth.  It was a pleasure to learn about the advancements in these fields at two excellent conferences, the CAR-TCR Summit organized by Hanson Wade, and the Cell and Gene Meeting on the Mesa sponsored by the Alliance of Regenerative Medicine.