Alexion, AstraZeneca Rare Disease, the group within AstraZeneca focused on rare diseases, has entered a definitive purchase and licence agreement for a portfolio of preclinical gene therapy programmes and enabling technologies from Pfizer.

The collaboration signifies a significant step forward in their joint dedication to advancing cutting-edge genomic medicines. By incorporating complementary pipeline assets and innovative technologies, they aim to pave the way for next-generation therapies.

A key aspect of this agreement is the addition of novel adeno-associated virus (AAV) capsids to Alexion’s repertoire. These AAV capsids have demonstrated their effectiveness in delivering therapeutic gene cargos for gene therapy and gene editing, enhancing the potential for successful treatment outcomes.

The infusion of these new resources bolsters the combined genomic medicine capabilities of Alexion and AstraZeneca, which were further reinforced by the recent acquisition of LogicBio.

Together, they are committed to developing novel genetic therapies that boast improved safety and efficacy profiles. Moreover, Alexion is eager to welcome Pfizer’s skilled professionals associated with the acquired portfolio, as they continue their quest to bring transformative treatments to patients in need.

Alexion, AstraZeneca Rare Disease CEO Marc Dunoyer 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.”

There are over 7,000 known rare diseases, with approximately 80% of them attributed to genetic mutations. To tackle these conditions, genomic medicines have emerged as a promising approach, aiming to treat or even cure these diseases by addressing the underlying malfunctioning genes. This can involve various techniques such as adding, altering, or deactivating genes to bolster the body’s ability to combat the illness effectively.

As part of their efforts to advance genomic medicines, Alexion and Pfizer have reached a significant agreement. Alexion will acquire and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio, making a total investment of up to $1bn, in addition to tiered royalties based on sales.

The transaction is scheduled to be concluded in the third quarter of 2023, contingent upon the fulfilment of closing conditions.