There are many elements to make a successful gene therapy
The key ones are the capsid, the therapeutic payload, and the manufacturing process.
The capsid is the ‘envelope’ that delivers the therapeutic payload to its right tissue in the body. The therapeutic payload is the genetic message or ‘letter’ put inside the capsid envelope that tells the cells in the tissue what protein to make and how much, essentially fixing the defect underlying the disease. The manufacturing process is how the capsid and therapeutic payload are put together properly as a final product before being given to a patient. For gene therapies, the manufacturing process is so important that experts say “the process is the product.
