- Pfizer is advancing it rare diseases therapeutic program by establishing a gene therapy platform and also by partnering with Spark Therapeutics to advance work on a hemophilia B treatment in development.
- One of the main areas of focus for rare disease development will be in the area of hematology, starting with hemophilia B.
- A top-notch researchers in the area of gene therapy—Professor Michael Linden from King's College London—has joined Pfizer to run the gene therapy research program.
This is not Pfizer's first foray into rare diseases R&D. In fact, Pfizer has a 10-pus year history in rare disease therapeutics, as well as a portfolio of 22 drugs that are approved by the FDA and other regulatory agencies worldwide. Their expertise includes hematology, but also extends to neuroscience, pulmonology, oncology and inherited metabolic disorders.