Accelerating Innovation: CRISPR and mRNA Biotech Platforms

Here’s a brief update on two platforms with progress in the past couple of weeks: CRISPR and mRNA. Both are examples of technologies I described in a previous article I wrote. The thesis of that article was that platform technology will accelerate the pace of innovation in treating – and curing – disease.

There is a Platform Effect: once the technology is working and accepted, then innovating on top of that platform means having a head start in terms of science, technology, or regulatory policy. Sometimes all three.

1. The UK’s Medicines and Healthcare Products Agency authorized (on November 16th) the first-ever CRISPR therapy. It is for patients with sickle cell disease and beta thalassemia.  An FDA decision is expected in December (for sickle cell) and in March 2024 (for beta thalassemia).

   a. Benefits: Avoid severe pain (from sickle cell disease) and frequent transfusions (for beta thalassemia).
   
   b. How it works: A patient’s stem cells are removed; edited in a lab using CRISPR gene editing; and returned after the patient undergoes a conditioning treatment to receive the edited cells.
   
   c. The Platform Effect of CRISPR: There are multiple CRISPR approaches being explored. With one approval in a major market, look for the use of CRISPR to treat other genetic diseases.
   
   Meanwhile, a lot still needs to be figured out. Some challenges include: ethical considerations of editing a genome; concerns about unintended (off-target) edits; long-term impact; as well as pricing and reimbursement. This article in Nature considers price expectations and alternatives (e.g., bone marrow transplant).
   

2. Japan’s Ministry of Health approved the first-ever self-amplifying mRNA (sa-mRNA) vaccine. It is for COVID-19.

a. Benefits: Preventing disease and reducing the spread of disease.

b. How it works: mRNA provides directions for creating the desired protein. Because sa-mRNA replicates the mRNA, there is the potential for an enhanced response -- which could mean more or longer protection at lower doses.

c. The Platform Effect of sa-mRNA: Look for the use of sa-mRNA in vaccines against other diseases.

Dr. Drew Weissman, who recently became a Nobel laureate for his pioneering work in mRNA, called self-amplifying mRNA a “next generation mRNA technology.”