AstraZeneca’s Alexion Buys Pfizer Gene Therapies for Rare Diseases

Alexion, AstraZeneca’s rare disease subsidiary, is acquiring a portfolio of preclinical gene therapy programs from Pfizer. The deal will bring a number of novel adeno-associated virus (AAV) capsids to Alexion and help build on AstraZeneca’s capabilities in genomic medicine.

Under the agreement, Alexion will purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total of up to $1 billion, plus tiered royalties on sales. Alexion plans to close the transaction in Q3 2023.

Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease, 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.”

AAV capsids are used for delivering therapeutic gene cargos for gene therapy and gene editing. In a press release AstraZeneca says, “These new resources build on Alexion and AstraZeneca’s combined capabilities in genomic medicine, recently strengthened with the acquisition of LogicBio, with the objective to develop new genetic therapies with improved safety and efficacy profiles. Additionally, Alexion will seek to welcome talent from Pfizer associated with the portfolio.”

Alexion acquired LogicBio in October of 2022. LogicBio’s proprietary GeneRide platform is a gene editing approach, while its SAAvy technology is a gene delivery capsid platform. At the time of the acquisition, Dunoyer said, “The proposed acquisition of LogicBio is a significant development for our growing research in genomic medicine.”

AstraZeneca today also announced the appointment of Sharon Barr as executive VP, BioPharmaceuticals R&D. Barr will succeed Mene Pangalos, who is retiring and will step down from his role early next year, after almost fourteen years with the company after a 35-year career.

Barr has been senior VP, Head of Research and Product Development of Alexion, AstraZeneca’s rare disease group, since 2013. She will now be responsible for discovery through to late-stage development across Cardiovascular, Renal & Metabolism and Respiratory & Immunology. She will report to CEO, Pascal Soriot and become a member of AstraZeneca’s senior executive team as of August 1.

According to the FDA, there are more than 7,000 known rare diseases, and around 80% of these are believed to be caused by a genetic mutation. Gene therapies treat or cure these diseases by addressing the malfunctioning gene. This can be done through addition, alteration, or inactivation of the gene to help the body fight the disease.

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals. A pioneer in rare diseases for nearly 30 years, Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology. Alexion is headquartered in Boston, Massachusetts and has offices around the globe, serving patients in more than 50 countries.

Dunoyer said, “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.”

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