Alexion Completes Purchase and License Agreement of Rare Disease Gene Therapy Portfolio from Pfizer

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The AstraZeneca rare disease group hopes to use these new gene therapy programs to bolster its work on genomic medicine.

On Sept. 20, 2023, Alexion, AstraZeneca’s rare disease group, completed the purchase and license agreement for a portfolio of preclinical rare disease gene therapy programs and enabling technologies from Pfizer. The acquisition cost AstraZeneca up to $1 billion, with the addition of tiered royalties on sales.Alexion hopes to create new genetic therapies, improving their safety and efficacy; the portfolio will bolster Alexion and AstraZeneca’s current work on genomic medicine.

Alexion entered into the agreement with Pfizer near the end of July 2023. The profile included sever novel adeno-associated virus (AAV) capsids, which have demonstrated to be effective for delivering therapeutic gene cargos for gene therapy and gene editing. Alexion reported that several Pfizer employees associated with the portfolio will join Alexion’s team as employees.

Alexion was formed as a group within AstraZeneca that focuses on rare diseases, following AstraZeneca’s acquisition of Alexion Pharmaceuticals in 2021. Alexion centers its research on novel molecules, focusing on the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology.


In a July 28, 2023 AstraZeneca press release, Marc Dunoyer, chef executive office at Alexion, said, “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. We look forward to continuing our work to develop enhanced platforms and technologies with broad therapeutic application while integrating best-in-class expertise to accelerate promising therapeutics into the clinic.”

Source: AstraZeneca