HQ Team
July 29, 2023: Alexion, AstraZeneca Rare Disease, an arm of AstraZeneca group, has signed a $1 billion pact with Pfizer for gene therapy projects.
Alexion will purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for up to one billion dollars, plus tiered royalties on sales, according to an AstraZeneca statement.
Alexion plans to close the transaction in the third quarter of 2023.
The accord covers a portfolio of preclinical gene therapy programmes and enabling technologies from Pfizer Inc.
“The agreement furthers Alexion and AstraZeneca’s commitment to advancing next-generation genomic medicines with the addition of complementary pipeline assets and innovative technologies,”
Capsids
Alexion will get access to a number of novel adeno-associated virus capsids following the agreement.
The capsids have been shown to be an effective mechanism for delivering therapeutic gene cargo for gene therapy and gene editing.
A capsid is the protein shell of a virus, enclosing its genetic material. It consists of several oligomeric (repeating) structural subunits made of protein called protomers.
These new resources build on Alexion and AstraZeneca’s combined capabilities in genomic medicine, according to the statement.
Genomic medicine
AstraZeneca had recently acquired LogicBio, with the objective of developing new genetic therapies with improved safety and efficacy profiles.
The pact “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,” said Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease.
There are more than 7,000 known rare diseases, and around 80% of rare diseases are believed to be caused by a genetic mutation, according to AstraZeneca.
Genomic medicines are designed to 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. Rare diseases affect more than 30 million Americans, according to the National Association for Rare Disorders.
