# AstraZeneca’s Alexion agreement with Pfizer for preclinical gene therapy portfolio 

- Link: https://www.directorstalkinterviews.com/astrazenecas-alexion%c2%a0agreement-with-pfizer-for-preclinical-gene-therapy-portfolio%c2%a0/4121124350
- Published: 2023-07-28T07:40:47+01:00
- Author: Amilia Stone

**[AstraZeneca plc](https://www.directorstalkinterviews.com/ftse-company/astrazeneca/azn.l)**(
[LON:AZN](https://www.directorstalkinterviews.com/ftse-company/astrazeneca/azn.l))
Alexion has announced that it has entered a definitive purchase and licence agreement
for 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.

As part of the agreement, the transaction will bring to Alexion a number of novel
adeno-associated virus (AAV) capsids. AAV capsids have been shown to be an effective
mechanism for delivering therapeutic gene cargos for gene therapy and gene editing.
^(1) 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.

> **Marc Dunoyer, Chief Executive Officer, 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. 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 more than 7,000 known rare diseases, and around 80% of rare diseases are
believed to be caused by a genetic mutation.^(2,3) 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.^(4)

**Financial considerations**

Under the agreement, Alexion will purchase and licence the assets of Pfizer’s early-
stage rare disease gene therapy portfolio for a total consideration of up to $1bn,
plus tiered royalties on sales.

Alexion plans to close the transaction in Q3 2023, subject to the satisfaction of
closing conditions.

**Alexion** 

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare
diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.
As a leader in rare diseases for nearly 30 years, Alexion is focused on serving 
patients and families affected by rare diseases and devastating conditions through
the discovery, development and commercialisation of life-changing medicines. Alexion
focuses its research efforts on novel molecules and targets in the complement cascade
and its development efforts on haematology, nephrology, neurology, metabolic disorders,
cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has
offices around the globe and serves patients in more than 50 countries.

**References**

1.   Wang D, et al. Adeno-associated virus vector as a platform for gene therapy
delivery. _Nat Rev Drug Discov_. 2019 May;18(5):358-378.

2.   FDA. Rare Diseases at FDA. Available at: https://www.fda.gov/patients/rare-
diseases-fda. Accessed July 2023.

3.   European Commission. Expert Group on Public Health. Rare diseases. Available
at: https://health.ec.europa.eu/non-communicable-diseases/expert-group-public-health/
rare-diseases_en. Accessed July 2023.

4.   FDA. How Gene Therapy Can Cure or Treat Diseases. Available at: https://www.
fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases. 
Accessed July 2023.

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