Intellia Therapeutics Inc. (NASDAQ: NTLA) on Thursday released longer-term follow-up data from the ongoing Phase 1 study of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).Results were presented at the International ATTR Amyloidosis Annual Meeting for Patients and Doctors. The results were simultaneously published in the New England Journal of Medicine.DataDeep, durable and consistent TTR reductions continue to be observed. Across ...

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]

Ocugen (NasdaqCM:OCGN) 2025 Conference September 25, 2025 09:30 AM ET Company ParticipantsShankar Musunuri - CEO, Co-founder & Chairman of the BoardHuma Qamar - Chief Medical OfficerNoneWe're good to go. Okay. Good afternoon, and thanks for joining us to have a conversation with Shankar Moussunari, CEO, and Huma Kumar, CFO I mean, CMO of Ocugen. Ocugen is a biotech company focused on discovery, development, and commercializing novel gene and cell therapies. The company's proprietary gene therapy platform ha ...

CHARLESTOWN, Mass., Sept. 25, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation investigational gene therapy intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 26th Annual Scientific Meeting of Neuromuscular Study Group (NMSG), September 26-28, 2025, in Stresa, Italy. Pr ...

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 ...

Summary of uniQure's AMT-130 Conference Call Company and Industry - **Company**: uniQure - **Industry**: Gene therapy for Huntington's disease Key Points and Arguments Clinical Trial Results - **Pivotal Data Announcement**: uniQure announced pivotal data from the Phase I/II clinical trials of AMT-130 for Huntington's disease, showing a statistically significant 75% slowing of disease progression at 36 months as measured by the Composite Unified Huntington's Disease Rating Scale (CUHDRS) [4][18] - **Total Functional Capacity (TFC)**: The study also demonstrated a 60% reduction in disease progression on the TFC, a key measure of a patient's ability to live independently [14][19] - **Neurofilament Light Chain (NFL)**: CSF neurofilament light chain levels were below baseline at 36 months, indicating a reduction in neurodegeneration [16][19] Regulatory and Commercial Plans - **Biologics License Application (BLA)**: uniQure plans to submit a BLA for AMT-130 in the first quarter of 2026, with a request for priority review [5][18] - **FDA Engagement**: A pre-BLA meeting with the FDA is scheduled to discuss the data and the content of the BLA [18][34] Patient Population and Market Opportunity - **At-Risk Population**: Approximately 200,000 Americans are at risk for Huntington's disease, with about 100,000 being genetically identifiable [26] - **Diagnosed Patients**: There are around 40,000 symptomatic HD patients in the U.S., with about 20,000 currently diagnosed [26][27] - **Initial Treatable Patients**: An estimated 6,000 patients could be considered initially treatable at launch [27] Treatment Characteristics - **Durable Treatment**: AMT-130 is designed as a durable, once-administered treatment, which is crucial for a slowly progressing disease [6] - **Targeted Delivery**: The treatment allows for precision-based delivery to affected brain regions, maximizing therapeutic concentrations and minimizing systemic exposure [7] - **Mechanism of Action**: AMT-130 targets the first exon of the Huntington gene, suppressing both the full-length mutant protein and toxic splice isoforms [7] Safety Profile - **Well Tolerated**: AMT-130 has shown a manageable safety profile, with no new treatment-related serious adverse events reported since December 2022 [17][19] - **Common Adverse Events**: Most adverse events were related to study procedures, such as headache and procedural pain [17] Expert Perspectives - **Clinical Impact**: Dr. Sarah Tabrizi emphasized the potential of AMT-130 to significantly impact the quality of life for patients and families affected by Huntington's disease [24] - **Long-term Benefits**: The slowing of disease progression could allow patients to maintain independence and work longer, potentially preventing symptoms from occurring [41] Future Considerations - **Payer Engagement**: Early engagement with payers is critical to educate them on the value of AMT-130 and its impact on patients' lives [51] - **Labeling and Eligibility**: Discussions are ongoing regarding the potential labeling of AMT-130, including whether it will be restricted to symptomatic patients [80] Additional Important Content - **Statistical Analysis**: The study's primary analysis showed a 75% reduction in disease progression with a p-value of 0.003, indicating strong statistical significance [18] - **Longitudinal Data**: The data from the Enroll-HD study provided a robust external control for comparison, enhancing the credibility of the results [55] This summary encapsulates the critical findings and strategic plans discussed during the conference call regarding AMT-130 and its implications for Huntington's disease treatment.

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 ...