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 ...

Core Viewpoint - uniQure N.V. has secured a $175 million non-dilutive senior secured term loan facility with Hercules Capital to enhance financial flexibility for the potential commercial launch of its investigational gene therapy AMT-130 for Huntington's disease [1][2]. Financial Details - The loan facility includes three tranches: a first tranche of $50 million to refinance existing debt, a second tranche of $100 million available upon achieving regulatory and financial milestones, and a third tranche of up to $25 million subject to Hercules' approval [3]. - The interest rate for all tranches is floating, set at the greater of 9.45% or the prime rate plus 2.45%, with a current floating rate of 9.70%, down from a previous rate of 11.95% [3]. Company Background - uniQure is a leader in gene therapy, focusing on transformative therapies for severe medical conditions, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [5]. - The company has achieved significant milestones, including the approval of its gene therapy for hemophilia B, marking a major advancement in genomic medicine [5]. Partner Information - Hercules Capital is a prominent specialty finance company that provides senior secured venture growth loans to high-growth, innovative companies in technology and life sciences, having committed over $22 billion to more than 680 companies since its inception [6].

Core Insights - uniQure N.V. announced positive topline data from the pivotal Phase I/II study of AMT-130 for Huntington's disease, demonstrating statistically significant slowing of disease progression [1][2][3] Study Results - The pivotal study met its primary endpoint, showing a 75% slowing of disease progression at 36 months as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS) compared to a matched external control [3][4] - High-dose AMT-130 also achieved a statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC), meeting a key secondary endpoint [4][6] - Favorable trends were observed in additional clinical measures, including an 88% slowing of disease progression as measured by the Symbol Digit Modalities Test (SDMT) [4][6] Safety Profile - AMT-130 was generally well-tolerated with a manageable safety profile, and no new drug-related serious adverse events were reported since December 2022 [8][9] - The most common adverse events were related to the administration procedure, all of which resolved [8] Regulatory Plans - uniQure plans to submit a Biologics License Application (BLA) in the first quarter of 2026, with a potential U.S. launch later that year pending approval [1][9] - AMT-130 has received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [9] Clinical Program Overview - The Phase I/II clinical program includes two multi-center, dose-escalating studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for Huntington's disease [11] - Data from the studies will be compared to a propensity score-matched external control derived from the Enroll-HD natural history dataset [11] Background on Huntington's Disease - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 75,000 people in the U.S., EU, and the UK, with no approved therapies currently available to slow its progression [15]

- 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 ...

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Core Insights - Adverum Biotechnologies is advancing its pivotal Phase 3 trial, ARTEMIS, for Ixo-vec in wet age-related macular degeneration (wet AMD), with full enrollment expected by 4Q 2025 and data readout anticipated in 1Q 2027 [1][6]. Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to treat prevalent ocular diseases, aiming to establish functional cures to restore vision and prevent blindness [4]. - The company is developing ixoberogene soroparvovec (Ixo-vec), designed for a one-time intravitreal injection, which aims to provide long-term efficacy and reduce the need for frequent anti-VEGF treatments [3][4]. Trial Details - ARTEMIS is the first of two Phase 3 trials evaluating Ixo-vec, comparing a single administration of Ixo-vec (6E10 vg/eye) to aflibercept (2mg) administered every 8 weeks [2]. - The trial includes both treatment-naïve and previously treated patients, with the goal of demonstrating the efficacy of Ixo-vec as a potential one-time treatment for wet AMD [2][3]. Regulatory Designations - Ixo-vec has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME designation from the EMA and Innovation Passport from the UK's Medicines and Healthcare Products Regulatory Agency [3].

Core Insights - Bayer has made significant progress in developing two potential therapies for Parkinson's disease (PD) [1] Group 1: Clinical Trials - The first participant has been randomized in the pivotal Phase III clinical trial, exPDite-2, for bemdaneprocel, which is an investigational cell therapy for PD [1] - The first European participants have been randomized in the Phase II clinical trial, REGENERATE-PD, for AB-1005, an investigational gene therapy [1] - Both therapies are aimed at treating moderate-stage Parkinson's disease [1]

Group 1 - Adverum Biotechnologies, Inc. (NASDAQ:ADVM) is recognized as a fundamentally strong penny stock with significant potential for investment [1][2] - The company reported impressive progress in the ARTEMIS Phase 3 trial, with increased enrollment driven by enthusiasm from retina specialists and patients, indicating a strong pipeline and future plans [2][3] - Strategic partnership discussions are ongoing, supported by the recognition of Ixo-vec's potential as a leading therapy, and a recent vote of confidence from Frazier Life Sciences, a major investor [3] Group 2 - Trexquant Investment LP significantly reduced its stake in Adverum by 80.5%, now holding 18,694 shares valued at approximately $82,000, which reflects a 0.09% ownership [1]

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Key Takeaways Krystal Biotech gained FDA approval to expand Vyjuvek's label to DEB patients from birth.The update allows at-home administration and flexibility in managing wound dressings.Approvals in Europe and Japan, plus U.S. growth, are expected to boost Vyjuvek sales.Krystal Biotech (KRYS) announced that the FDA has approved a label update of its marketed product, Vyjuvek (beremagene geperpavec-svdt), to include dystrophic epidermolysis bullosa ("DEB") patients from birth.Shares of Krystal Biotech were ...