Pfizer pivots to 'externalization" of rare disease programs January 06, 2023 - Pfizer is stepping back from early-stage rare disease R&D and has begun “exploring externalization opportunities for a number of highly innovative, niche programs” as part of a rethink of its approach to orphan indications.
The company said it would "externalize" most of its early-stage rare disease programs in neurology and cardiology, and gene therapy programs not yet in clinical trials.
Pfizer will continue to develop viral vector-based gene therapies for hemophilia A, hemophilia B and Duchenne muscular dystrophy, according to Barron’s, while shifting its investment to other delivery vehicles such as lipid nanoparticles (LNPs).
“This approach will allow us to focus on programs where our innovation and investments are differentiated and where we are best positioned to generate high-impact medicines and vaccines" according to a Pfizer spokesperson
Pfizer’s current gene therapy portfolio includes three late-stage clinical programs for hemophilia A, hemophilia B, and Duchenne muscular dystrophy (DMD), as well as 12 preclinical programs investigating potential treatments for rare cardiology, endocrine, hematology, metabolic and neurology diseases.
https://www.fiercebiotech.com/biotech/pfizer-pivots-early-stage-rare-disease-rd-shifting-external-innovation-and-putting-assets