Skip to main content

Life Sciences

Tackling Rare Diseases With Gene Therapy



A couple of weeks ago, I shared some statistics that I learned from an FDA blog post about rare diseases. While those numbers will make you cringe, Pfizer will give you hope. They, along with Spark Therapeutics, are teaming up to develop treatments for rare diseases, like hemophilia, through gene therapy.

Gene therapy is a treatment method that involves genes being inserted into a patient’s cells. This kind of innovative, personalized approach exhibits the incredible and meaningful contribution that R&D pharmaceutical companies continue to make in the new era of precision medicine.

Leave a Reply

Your email address will not be published. Required fields are marked *

This site uses Akismet to reduce spam. Learn how your comment data is processed.

Eugene Sefanov

Director, Industry and Regional Marketing

More from this Author

Follow Us