AstraZeneca’s Alexion to boost rare disease commitment with Pfizer’s gene therapy programs

Alexion, a subdivision of AstraZeneca focusing on rare diseases, has announced its definitive purchase and licensing agreement for a portfolio of preclinical gene therapy programmes from Pfizer Inc. The deal, a move to strengthen Alexion and AstraZeneca’s commitment to next-generation genomic medicines, is worth up to $1 billion, plus tiered royalties on sales.

This acquisition brings novel adeno-associated virus (AAV) capsids to Alexion, which have proven effective for delivering therapeutic gene cargos for gene therapy and gene editing. This agreement enhances Alexion and AstraZeneca’s combined capabilities in genomic medicine, recently bolstered with the acquisition of LogicBio Therapeutics. The ultimate goal is to develop new genetic therapies with improved safety and efficacy profiles.

Marc Dunoyer, Chief Executive Officer of Alexion, stated, “Today’s announcement represents another major step forward in Alexion and AstraZeneca’s ambition to be an industry leader in genomic medicine, which has potential to be transformative and even curative for patients with devastating diseases.”

Rare diseases are believed to be caused by genetic mutations in around 80% of cases. Genomic medicines are designed to treat or cure these diseases by addressing the malfunctioning gene, potentially through the addition, alteration, or inactivation of the gene to help the body fight the disease.

Alexion’s acquisition of Pfizer’s early-stage rare disease gene therapy portfolio is scheduled to close in Q3 2023, subject to the satisfaction of closing conditions.