Core Viewpoint - uniQure N.V. has announced a public offering of 5,789,473 ordinary shares priced at $47.50 per share, aiming to raise approximately $300 million in gross proceeds before expenses [1] Group 1: Offering Details - The offering includes pre-funded warrants for certain investors to purchase 526,316 ordinary shares at the public offering price minus a nominal exercise price [1] - A 30-day option has been granted to underwriters to purchase up to 947,368 additional ordinary shares at the public offering price [1] - The offering is expected to close on or about September 29, 2025, pending customary closing conditions [1] Group 2: Management and Underwriters - Leerink Partners, Stifel, Guggenheim Securities, and Van Lanschot Kempen are acting as bookrunning managers for the offering, with H.C. Wainwright & Co. as the lead manager [2] Group 3: Company Background - uniQure is focused on gene therapy, with a significant milestone achieved through the approval of its gene therapy for hemophilia B, representing over a decade of research [5] - The company is advancing a pipeline of gene therapies targeting severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [5]

A new gene therapy has slowed the progression of Huntington’s disease by as much as 75%, according to the results of a newly announced trial. We explain what this could mean for patients https://t.co/rfIICk3QQ1 ...

Core Insights - Intellia Therapeutics Inc. has released long-term follow-up data from its Phase 1 study of nexiguran ziclumeran (nex-z) for treating hereditary ATTR amyloidosis with polyneuropathy, showcasing significant TTR reductions and clinical improvements [1][2][6] Data Summary - A one-time dose of 0.3 mg/kg or higher resulted in a mean serum TTR reduction of 92% at 24 months, with an absolute serum TTR level of 17.3 g/mL [2] - Among 12 patients with 36 months of follow-up, the mean serum TTR reduction was 90%, corresponding to a serum TTR level of 20 g/mL [2] - Favorable trends indicating stability or improvement were observed in most patients after a single dose of nex-z [3] Clinical Measures - Stability or improvement was assessed using various clinical and biomarker measures, including NIS, mNIS+7, mBMI, QoL-DN questionnaire, NfL, and PND score [4] - Of the 18 patients assessed at 24 months, 72% showed clinically meaningful improvements of ≥4 points in mNIS+7, including those who had previously progressed on Alnylam's Onpattro [5] Secondary Endpoints - Mean values for secondary endpoints mBMI, QoL-DN, and NfL trended towards disease improvement, with 89% of patients showing improvement or stability in PND scores through 24 months [6] - Nex-z was generally well tolerated, with mild to moderate infusion-related reactions being the most common treatment-related adverse events [6][7] Future Outlook - Patient screening for the Phase 3 MAGNITUDE-2 trial is progressing rapidly, with enrollment expected to complete in the first half of 2026 [8] - Intellia plans to submit a biologics license application (BLA) for ATTRv-PN by 2028 [8]

Core Insights - Shares of uniQure N.V. (QURE) surged 247.7% following the successful results of its pivotal study for AMT-130, a gene therapy for Huntington's disease [1][6] Study Results - The pivotal phase I/II study met its primary endpoint, showing a statistically significant 75% slowing of disease progression on the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 36 months for the high dose of AMT-130 [3][6] - The study also achieved a key secondary endpoint with a 60% slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months [5][6] - Patients receiving the high dose of AMT-130 demonstrated an 88% slowing on the Symbol Digit Modalities Test, a 113% slowing on the Stroop Word Reading Test, and a 59% slowing on the Total Motor Score compared to external controls [7] Biomarker and Safety Profile - The study indicated a decrease of 8.2% in cerebrospinal neurofilament light protein, a biomarker associated with neurodegeneration in Huntington's disease [8] - AMT-130 was reported to have a manageable safety profile, with adverse events primarily mild in severity [9] Future Plans - Based on the positive data, uniQure N.V. plans to engage with the FDA to submit a biologics license application for AMT-130, targeting the first quarter of 2026 [10] Clinical Pipeline - In addition to AMT-130, uniQure N.V. has other candidates in early to mid-stage development for conditions such as refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis, and Fabry disease [14]

Financial Data and Key Metrics Changes - The company is targeting three Biologics License Applications (BLAs) in the next three years, starting with OCU 400 in 2026 for retinitis pigmentosa [2][4] - The company has a cash runway of approximately $50 million, with a recent $20 million injection into the balance sheet and a potential additional $30 million option [52][53] Business Line Data and Key Metrics Changes - OCU 400 is in Phase 3 trials, with a primary endpoint focused on baseline changes in lux levels and secondary endpoints including low luminance visual acuity [9][17] - OCU 410 for Stargardt disease is in a Phase 2/3 registration trial with an adaptive design, aiming for a reduction in lesion size and improvement in visual acuity [31][35] Market Data and Key Metrics Changes - The company is the only one targeting a broad indication for retinitis pigmentosa, potentially addressing 98-99% of patients with unmet medical needs [6][10] - The geographic atrophy (GA) program is positioned against existing approved drugs, with a focus on both structural and functional outcomes [40][41] Company Strategy and Development Direction - The company aims to become a global leader in gene therapies for blindness diseases, focusing on three key programs [46][47] - The divestment of the neocard asset is intended to allow the company to concentrate on gene therapies, with a valuation of over $100 million for the divested assets [48][50] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming BLA submissions and the potential for significant patient impact globally [4][28] - The company is preparing for regulatory submissions in both the US and Europe, with EMA agreeing to the US trial design [27][28] Other Important Information - The company has established safety and efficacy in prior trials, with no serious adverse events reported [16][35] - The company is efficiently run compared to many biotechs, with a focus on maximizing resources for its gene therapy programs [52] Q&A Session Summary Question: What is the progress on the OCU 400 study and when will top-line data be available? - The data will be available next year, and the company is on track to file for the BLA in 2026 [23][24] Question: What is Stargardt disease and what is the status of the OCU 410 program? - Stargardt disease is an inherited retinal disease primarily affecting the pediatric population, and the OCU 410 program is in a Phase 2/3 trial with promising early data [30][31] Question: How does the company plan to address the competitive landscape for geographic atrophy? - The company is focusing on both structural and functional outcomes, with promising data from Phase 1 and Phase 2 trials [40][41] Question: What is the company's strategy regarding the divestment of the neocard asset? - The divestment allows the company to focus on gene therapies, with a significant valuation for the divested assets that could enhance shareholder value [48][50] Question: What is the current strength of the company's balance sheet? - The company has a cash runway of approximately $50 million, bolstered by recent funding [52][53]

Core Insights - Solid Biosciences Inc. is presenting data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a gene therapy for Duchenne muscular dystrophy, at the NMSG Annual Meeting in Italy [1][2] - The company emphasizes the favorable profile of SGT-003, particularly its liver tolerability and early biomarker signals, supported by a steroid-only immunomodulation regimen [2] - Solid Biosciences has established over 25 licensing agreements for the use of its next-generation capsid, AAV-SLB101, which is designed for targeted gene therapy [2] Company Overview - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy [6][7] - The company aims to improve the lives of patients affected by devastating rare diseases, with a mission rooted in personal experiences with Duchenne [7] Product Details - SGT-003 is an investigational gene therapy featuring a differentiated microdystrophin construct and the proprietary AAV-SLB101 capsid, designed to enhance muscle transduction while minimizing liver targeting [4] - The microdystrophin construct includes R16/17 domains, which may improve blood flow to muscles, potentially reducing muscle breakdown and fatigue [4] Clinical Trial Information - INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial assessing the safety, tolerability, and efficacy of SGT-003 in pediatric patients with Duchenne [5] - The trial is multinational, enrolling participants from the United States, Canada, the United Kingdom, and Italy [5]

Core Viewpoint - uniQure N.V. has initiated a $200 million underwritten public offering of its ordinary shares and pre-funded warrants, with a 30-day option for underwriters to purchase an additional 15% of the total shares sold [1][2]. Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs, including gene therapies for hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [5]. - The company has achieved significant milestones in genomic medicine, particularly with its gene therapy for hemophilia B, which is based on over a decade of research and clinical development [5]. Offering Details - The public offering is subject to market conditions, and there is no assurance regarding its completion or the final terms [1]. - The offering is being conducted under an automatically effective shelf registration statement filed with the SEC [3]. - A preliminary prospectus supplement and accompanying prospectus will be filed with the SEC and will be available for free on the SEC's website [3]. Underwriters - The bookrunning managers for the offering include Leerink Partners, Stifel, Van Lanschot Kempen, and Guggenheim Securities [2].

Uniqure (QURE) stock soared nearly 300% on Wednesday after the Dutch biotechnology company announced a major breakthrough in Huntington’s disease. Investors are cheering QURE shares this morning because the Nasdaq-listed firm has secured new financing worth $175 million from Hercules Capital as well. More News from Barchart QURE stock is now trading roughly 460% above its year-to-date low in early April. www.barchart.com Why Did the Clinical Data Light a Fire Under QURE Stock? Uniqure shares are rall ...

PresentationGood day, and welcome to the top line results for AMT-130 in Huntington's disease. [Operator Instructions] As a reminder, this call may be recorded. I would now like to turn the call over to Chiara Russo, Senior Director, Investor Relations.Chiara Russo Thank you. This morning, uniQure announced pivotal data on patients treated with our investigational gene therapy, AMT-130 in our ongoing Phase I/II clinical trials in Huntington's disease, taking place in the U.S., EU and the U.K. This 3-year u ...

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 ...