CSL and uniQure Win 2023 Prix Galien USA Award

LEXINGTON - Global biotechnology leader CSL (ASX: CSL) and uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the companies have received the 2023 Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX.

CSL received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX in November 2022. It is the first and only gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.

CSL is humbled and honored to receive a Prix Galien award for HEMGENIX,' said Dr. Bill Mezzanotte, Head of Research and Development, CSL. 'We thank The Galien Foundation for acknowledging the enormous promise and hope that HEMGENIX offers to eligible people living with hemophilia B.'

The multi-year clinical development of HEMGENIX was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment.

'HEMGENIX represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for eligible people living with hemophilia B,' said Matt Kapusta, Chief Executive Officer, uniQure. 'It gives us great pride that after many years of development, this important gene therapy is now being made available by CSL to patients in geographies around the world.'

HEMGENIX also has been granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area, and the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA), as well as authorization by Health Canada.

The Prix Galien USA is America's preeminent prize acknowledging the leading-edge of scientific advances in life sciences.

About Hemophilia B

Hemophilia B is a life-threatening rare disease caused by a mutation on the F9 gene, resulting in low levels of functional clotting factor IX. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.

About HEMGENIX

HEMGENIX is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.

About CSL

CSL (ASX:CSL; USOTC:CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL - including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor - provides lifesaving products to patients in more than 100 countries and employs 30,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

About uniQure

uniQure's mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B - a historic achievement based on more than a decade of research and clinical development - represents a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases.

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