uniQure N.V. QURE on Wednesday released topline data from the pivotal Phase 1/2 study of AMT-130 for Huntington’s disease.Huntington’s disease is an inherited condition in which nerve cells in the brain break down over time. The disease affects a person’s movements, thinking ability and mental health.The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating ...

uniQure (NasdaqGS:QURE) Update / Briefing September 24, 2025 08:30 AM ET Company ParticipantsChiara Russo - Senior Director - IRMatt Kapusta - CEO & Executive DirectorWalid Abi-Saab - Chief Medical OfficerSarah Tabrizi - ProfessorKylie O’Keefe - Chief Customer & Strategy OfficerPaul Matteis - MD & Head - Therapeutics ResearchJoseph Schwartz - Senior MD - Rare DiseasesDebjit Chattopadhyay - Senior Managing DirectorUy Ear - VP of US Healthcare - BiotechnologySalveen Richter - Biotechnology Equity ResearchElli ...

Pivotal Phase I/II AMT-130 Huntington's Disease Update September 24, 2025 Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may ...

uniQure (NASDAQ:QURE) announced on Wednesday that its one-time gene therapy AMT-130 reached the main goals in a pivotal Phase 2/3 trial for patients with Huntington’s disease, a rare neurodegenerative disorder. Citing topline data from the trial, the Dutch biotech noted that ...

~ Term of current $50 million debt outstanding extended from January 2027 to October 2030 ~ ~ Additional $100 million available subject to regulatory and financial milestones ~ ~ Additional $25 million available subject to Hercules’ approval ~ ~ Transaction provides financial flexibility and improves cost of capital ~ LEXINGTON, Mass. and AMSTERDAM, Sept. 24, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medic ...

~ Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control ~ ~ High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures ~ ~ Mean cerebrospinal fluid NfL levels were below baseline at 36 months ~ ~ AMT-130 continued to be gener ...

- Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for dysferlin-related limb-girdle muscular dystrophy - - Solid to receive an upfront payment and is eligible for certain development and sales milestones and tiered royalties on net sales -- Solid continues to expand collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date - CHARLESTOWN, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Sol ...

Screening closing ahead of expectations, with full enrollment now anticipated in 4Q 2025 (previously 1Q 2026)ARTEMIS topline data readout accelerated to 1Q 2027 REDWOOD CITY, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of intravitreal gene therapy as a potential One And DoneTM treatment to preserve sight for life, today announced it has notified sites that it plans to complete screening in ARTEMIS, its first pivotal Ph ...

BERLIN--(BUSINESS WIRE)--Bayer announced today progress for two potential therapies against Parkinson's disease (PD). A first participant received randomized treatment in the pivotal Phase III clinical trial, exPDite-2, of bemdaneprocel, an investigational cell therapy for PD. At the same time, first European participants have been randomized in REGENERATE-PD, a Phase II clinical trial of AB-1005, an investigational gene therapy. Both therapies are focused on treating moderate-stage PD and are. ...

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) is among the best fundamentally strong penny stocks to buy right now. During the first quarter, Trexquant Investment LP reduced its stake in Adverum Biotechnologies, Inc. (NASDAQ:ADVM) by 80.5%. Following the offload of 77,303 shares, the institutional investor now owns 18,694 shares of the company’s stock, valued at approximately $82,000. According to the recent 13F filing with the SEC, the firm has an ownership of 0.09%. Last month, Adverum Biotechnologies, In ...