QURE News and Press, uniQure N.V. From 05/02/23

LEXINGTON, Mass. and AMSTERDAM, May 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be deliver...

2023-03-21 07:43:24 ET A top FDA official said Monday that the agency needs to consider accelerated approval, a regulatory avenue commonly used to expedite the market entry of cancer drugs, for gene therapies. Dr. Peter Marks, who heads the FDA's biologics unit, said that the agency...

uniQure press release ( NASDAQ: QURE ): FY GAAP EPS of -$2.71 beats by $0.98 . Revenue of $106.48M (-79.7% Y/Y) beats by $83.27M . As of December 31, 2022, the Company had cash and cash equivalents and investment securities of $392.8 million. The Company expects cash a...

~ Achieved U.S. and European Commission marketing approvals of first and only gene therapy for adults with hemophilia B ~ ~ Advancing development of AMT-130 for patients with Huntington’s disease – clinical update from U.S. Phase I/II trial expected in seco...

~ Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds, reduce or eliminate the need for prophylactic therapy and generate elevated and sustained factor IX levels for years after a one-time infu...

CSL's HOPE-B Data Published in the New England Journal of Medicine, Demonstrating Efficacy and Durability of HEMGENIX® (etranacogene dezaparvovec-drlb) PR Newswire Data follow the historic approval of the first gene therapy for hemophilia B, which reduces the rate of annu...

~ Historic approval represents the first gene therapy in Europe to treat hemophilia B and provides a new treatment option for patients that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels ~ ...

Summary Hemgenix was approved by FDA. uniQure appears undervalued based on the value of milestones and royalties due to the company. During Q2, uniQure will release data on AMT-130, which is a binary event with an asymmetric risk reward. Summary Partner CSL obtaine...

Dutch biotech uniQure N.V. ( NASDAQ: QURE ) traded higher pre-market Tuesday in reaction to a licensing agreement with Apic Bio for APB-102, a gene therapy targeted at a rare form of neurodegenerative disorder, amyotrophic lateral sclerosis (ALS). The FDA has already cleared an invest...

~ License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~ ~ APB-102 and uniQure’s c9orf72-ALS program have the potential to address most inherited forms of ALS ~ ...