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uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-210, a product candidate for the treatment of Parkinson's disease; AMT-260 for temporal lobe epilepsy; AMT-240, a preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease; and AMT-161 for the treatment of amyotrophic lateral sclerosis. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

uniQure N.V.

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~ ACHIEVED ALIGNMENT WITH THE FDA ON THE AMT-130 STATISTICAL ANALYSIS PLAN AND CMC REQUIREMENTS TO SUPPORT A PLANNED BLA SUBMISSION IN THE FIRST QUARTER OF 2026;

UNIQURE ANNOUNCES SECOND QUARTER 2025 FINANCIAL RESULTS AND HIGHLIGHTS OF RECENT COMPANY PROGRESS

UNIQURE TO HOST EARNINGS CALL ON TUESDAY, JULY 29, 2025 AT 8:30 A.M. ET UNIQURE TO HOST EARNINGS CALL ON TUESDAY, JULY 29, 2025 AT 8:30 A.M. ET

UNIQURE TO ANNOUNCE SECOND QUARTER 2025 FINANCIAL RESULTS

~PROVEN BIOTECH EXECUTIVE TO LEAD COMMERCIALIZATION OF AMT-130 IN HUNTINGTON'S DISEASE ~ LEXINGTON, MASS. AND AMSTERDAM, JUNE 11, 2025 (GLOBE NEWSWIRE) -- UNIQURE N.V.

UNIQURE ANNOUNCES APPOINTMENT OF KYLIE O'KEEFE AS CHIEF CUSTOMER AND STRATEGY OFFICER

~ ALIGNMENT WITH FDA CONTINUES TO SUPPORT ACCELERATED APPROVAL PATHWAY ~ ~ BLA SUBMISSION PLANNED FOR FIRST QUARTER OF 2026 ~ ~ CONFERENCE CALL TODAY AT 8:30 A.M. ET ~ LEXINGTON, MASS.

UNIQURE PROVIDES REGULATORY UPDATE ON AMT-130 FOR HUNTINGTON'S DISEASE

NORWOOD, MASS., MAY 19, 2025 (GLOBE NEWSWIRE) -- CORBUS PHARMACEUTICALS HOLDINGS INC. (NASDAQ: CRBP), A CLINICAL-STAGE COMPANY FOCUSED ON ONCOLOGY AND OBESITY, TODAY ANNOUNCED THE APPOINTMENT OF RACHELLE JACQUES AS CHAIR OF ITS BOARD OF DIRECTORS, EFFECTIVE MAY 15.

CORBUS PHARMACEUTICALS NAMES INDUSTRY VETERAN RACHELLE JACQUES AS BOARD CHAIR

~ AMT-130 GRANTED BREAKTHROUGH THERAPY DESIGNATION BY FDA ~ ~ INITIAL SAFETY DATA FROM THIRD COHORT OF PHASE I/II STUDY SHOW AMT-130 CONTINUES TO BE GENERALLY WELL-TOLERATED, WITH NO TREATMENT-RELATED SERIOUS ADVERSE EVENTS ~ ~ HELD TYPE B FDA MEETINGS IN THE FIRST AND SECOND QUARTERS OF 2025 TO ADVANCE BLA PREPARATIONS FOR AMT-130; REGULATORY UPDATE EXPECTED IN THE SECOND QUARTER OF 2025 ~ ~ AMT-260 CLINICAL DATA FROM FIRST PATIENT TO BE PRESENTED AT EPILEPSY THERAPIES & DIAGNOSTICS DEVELOPMENT SYMPOSIUM ON MAY 29, 2025 ~ ~ CASH, CASH EQUIVALENTS AND CURRENT INVESTMENT SECURITIES OF APPROXIMATELY $409.0 MILLION AS OF MARCH 31, 2025 EXPECTED TO FUND OPERATIONS INTO SECOND HALF OF 2027 ~ ~ UNIQURE TO HOST INAUGURAL EARNINGS CALL AT 8:30 A.M. ET ~ LEXINGTON, MASS.

UNIQURE ANNOUNCES FIRST QUARTER 2025 FINANCIAL RESULTS AND HIGHLIGHTS OF RECENT COMPANY PROGRESS

~ UNIQURE TO HOST INAUGURAL EARNINGS CALL ON FRIDAY, MAY 9, 2025 AT 8:30 A.M. ET ~ ~ UNIQURE TO HOST INAUGURAL EARNINGS CALL ON FRIDAY, MAY 9, 2025 AT 8:30 A.M. ET ~

UNIQURE TO ANNOUNCE FIRST QUARTER 2025 FINANCIAL RESULTS

~ BREAKTHROUGH THERAPY DESIGNATION BASED ON CLINICAL EVIDENCE FROM PHASE I/II TRIALS SHOWING MEANINGFUL SLOWING OF DISEASE PROGRESSION ~

UNIQURE ANNOUNCES FDA BREAKTHROUGH THERAPY DESIGNATION GRANTED TO AMT-130 FOR THE TREATMENT OF HUNTINGTON'S DISEASE

~ ANNOUNCED ALIGNMENT WITH THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) ON KEY ELEMENTS OF THE ACCELERATED APPROVAL PATHWAY FOR AMT-130 IN HUNTINGTON'S DISEASE; INITIATED PREPARATIONS FOR A POTENTIAL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION ~

UNIQURE ANNOUNCES 2024 FINANCIAL RESULTS AND HIGHLIGHTS RECENT COMPANY PROGRESS

94 PERCENT OF PATIENTS ELIMINATED FACTOR IX PROPHYLAXIS AND REMAINED FREE OF CONTINUOUS PROPHYLAXIS THROUGH FOUR YEARS POST-TREATMENT MEAN FACTOR IX ACTIVITY LEVELS WERE SUSTAINED AT NEAR NORMAL LEVELS OF 37% THROUGH FOUR YEARS POST-TREATMENT, REINFORCING THE EFFICACY OFHEMGENIX IN THE TREATMENT OF HEMOPHILIA B PHASE 3 HOPE-B DATA SHOWED THAT A ONE-TIME TREATMENT WITHHEMGENIX PROVIDED LONG-TERM BLEED PROTECTION AS MEAN ADJUSTED ANNUALIZED BLEEDING RATE (ABR) FOR ALL BLEEDS WAS REDUCED BY APPROXIMATELY 90% FROM LEAD-IN AS COMPARED TO YEAR FOUR KING OF PRUSSIA, PA. , FEB. 7, 2025 /PRNEWSWIRE/ -- GLOBAL BIOTECHNOLOGY LEADER CSL (ASX:CSL; USOTC:CSLLY) TODAY ANNOUNCED THE FOUR-YEAR RESULTS FROM THE PIVOTAL HOPE-B STUDY CONFIRMING THE LONG-TERM DURABILITY AND SAFETY OF A ONE-TIME INFUSION OF HEMGENIX(ETRANACOGENE DEZAPARVOVEC-DRLB) FOR ADULTS LIVING WITH HEMOPHILIA B.

CSL BEHRING'S GENE THERAPY HEMGENIX (ETRANACOGENE DEZAPARVOVEC-DRLB) FOUR YEARS POST-INFUSION DATA CONTINUE TO SHOW SUSTAINED EFFICACY AND SAFETY IN ADULTS WITH HEMOPHILIA B

uniQure N.V.

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uniQure N.V.

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