Dive Brief:
- 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.
Dive Insight:
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.