My name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens, OH, USA, a graduate degree in Business Management from Universidad Andina Simon Bolivar, Quito, Ecuador, and the Ph.D. degree in computer applications from the University of Alicante, Spain.Disclosure: I collaborate professionally with Edgar Torres H, who is also an author on Seeking ...

Core Insights - U.S. stocks exhibited mixed performance, with the Dow Jones index declining by over 50 points on Thursday [1] - Taysha Gene Therapies Inc received Breakthrough Therapy designation from the FDA for TSHA-102, leading to a significant share price increase of 50.4% to $4.78 [1] Company Highlights - Taysha Gene Therapies Inc (NASDAQ:TSHA) saw its price target raised from $8 to $10 by Needham analyst Gil Blum, maintaining a Buy rating [1] - Bakkt Holdings Inc (NYSE:BKKT) experienced a 37.2% increase in share price to $46.81 following the completion of its Loyalty Business sale [4] - Canaan Inc – ADR (NASDAQ:CAN) shares surged by 29.8% to $1.35 after announcing a significant sales order for Bitcoin mining machines [4] - Ondas Holdings Inc (NYSE:ONDS) gained 25.5% to $9.18 after securing an initial order of 500 Wasp drones for U.S. defense market distribution [4] - Fair Isaac Corp (NYSE:FICO) shares rose 19.6% to $1,811.61 after launching the FICO Mortgage Direct License Program [4] - AST SpaceMobile Inc (NASDAQ:ASTS) jumped 12.3% to $63.98 after completing final assembly and testing of its BlueBird 6 satellite [4] - Joby Aviation Inc (NYSE:JOBY) rose 9.3% to $17.72, reflecting positive market sentiment [4] - Applied Digital Corp (NASDAQ:APLD) shares increased by 7.7% to $26.93, with crypto-linked stocks trading higher amid a rise in Bitcoin [4]

Taysha Gene Therapies (NASDAQ:TSHA) jumped ~39% on Thursday after the U.S. FDA granted its Breakthrough Therapy designation for the company's lead candidate TSHA-102 as a treatment for Rett syndrome, a rare neurodevelopmental disorder. Additionally, the gene therapy developer stated that the ...

Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all 12 patients treated in Part A of the REVEAL Phase 1/2 trials Finalized FDA alignment on REVEAL pivotal trial protocol and SAP following resolution of remaining clinical and statistical queries to IND application amendment; on track to begin patient enrollment in Q4 2025 Key pivotal trial design elements remain unchanged, including 6-month interim analysis to potentially expedite BLA ...

Core Insights - Recent market movements have highlighted significant price changes in several companies, attracting investor attention Company Summaries - **Palisade Bio, Inc. (PALI)**: Experienced an 83.77% price surge to $1.57, supported by a trading volume of 143,677,281. The company raised $120 million through an upsized underwritten public offering, selling 171 million shares at $0.70 each, to advance its novel therapeutics for autoimmune, inflammatory, and fibrotic diseases [1][6] - **Ouster, Inc. (OUSTZ)**: Saw a 76.55% price increase to $0.09, with a volume of 150,580. This reflects growing confidence in Ouster's high-resolution lidar technology, which serves various industries including automotive and robotics [2][6] - **Ryvyl Inc. (RVYL)**: Formerly known as GreenBox POS, experienced a 72.64% price jump to $0.511, supported by a substantial volume of 231,813,941. The company focuses on blockchain-based payment solutions, showcasing resilience and innovation in the technology sector [3] - **Klotho Neurosciences, Inc. (KLTOW)**: Saw a 50.11% price increase to $0.14, with a volume of 134,508. The company is advancing gene therapy product candidates, and its ALS therapy received Orphan Drug Designation from the U.S. FDA, contributing to a rise in shares [4] Industry Overview - The top gainers reflect diverse industries, including biotechnology, healthcare, technology, and financial services, underscoring the dynamic nature of the market and varied investment opportunities [5]

Core Insights - uniQure N.V. is recognized as one of the best performing healthcare stocks, focusing on gene therapy for severe medical conditions [1] - The company has achieved significant clinical milestones, particularly with AMT-130 for Huntington's disease, showing a 75% slowing of disease progression over three years [2] - Financially, uniQure has raised $300 million through a public offering and secured a $175 million senior secured term loan to support its development and commercialization efforts [3] - Analysts have responded positively to uniQure's breakthroughs and funding, leading to strong buy ratings and optimistic price targets [4] Company Overview - uniQure N.V. specializes in gene therapy, with a pipeline that includes treatments for hemophilia B and Huntington's disease [1] - The company is advancing multiple gene therapy candidates targeting rare diseases, including AMT-260 for mesial temporal lobe epilepsy, AMT-162 for ALS, and AMT-191 for Fabry disease [3] Clinical Developments - Positive topline results from pivotal Phase I/II trials of AMT-130 have set the stage for a Biologics License Application (BLA) submission to the FDA in early 2026 [2] - The combination of groundbreaking data from AMT-130 and a diversified pipeline positions uniQure for potential transformative impacts on patients [4]

4D Molecular Therapeutics (NASDAQ: FDMT ) seems an attractive buy in late 2025, especially for long-term investors with a high-risk appetite and who can tolerate short-term volatility. With shares priced at $8.35 (as of September 29) and a market capitalizationI am an MBA in finance and an engineering graduate. I have also completed the CFA certification.I am involved in international trade and have been passionately tracking global equity markets for more than 7 years. I mainly focus on spotting long-term ...

4D Molecular Therapeutics (NASDAQ: FDMT ) seems an attractive buy in late 2025, especially for long-term investors with a high-risk appetite and who can tolerate short-term volatility. With shares priced at $8.35 (as of September 29) and a market capitalizationI am an MBA in finance and an engineering graduate. I have also completed the CFA certification.I am involved in international trade and have been passionately tracking global equity markets for more than 7 years. I mainly focus on spotting long-term ...

About this content About Ian Lyall Ian Lyall, a seasoned journalist and editor, brings over three decades of experience to his role as Managing Editor at Proactive. Overseeing Proactive's editorial and broadcast operations across six offices on three continents, Ian is responsible for quality control, editorial policy, and content production. He directs the creation of 50,000 pieces of real-time news, feature articles, and filmed interviews annually. Prior to Proactive, Ian helped lead the business outpu ...

Core Viewpoint - A novel gene therapy has shown significant potential in slowing the progression of Huntington's disease, marking a possible breakthrough in treatment options for this rare genetic neurodegenerative disorder [4][5]. Group 1: Disease Overview - Huntington's disease is a rare hereditary neurodegenerative disorder characterized by the gradual degeneration of nerve cells in the brain, leading to motor, cognitive, and psychiatric impairments [3]. - The disease is caused by an expansion of the CAG repeat sequence in the HTT gene, resulting in a toxic protein that progressively damages the brain [5]. - Patients typically begin to exhibit symptoms between the ages of 35 and 55, with initial symptoms including mild coordination loss and memory issues, which can escalate to involuntary movements and severe emotional disturbances [4]. Group 2: Gene Therapy Development - The gene therapy developed by uniQure, known as AMT-130, utilizes adeno-associated virus type 5 (AAV5) to deliver miRNA designed to silence the mutated HTT gene, thereby blocking the production of the toxic protein [6][8]. - In a clinical trial involving 29 early-stage Huntington's disease patients, those receiving the high-dose gene therapy experienced a 75% reduction in disease progression over three years compared to the control group [4]. - uniQure plans to apply for regulatory approval for this therapy next year based on significant clinical indicators, including reduced levels of toxic proteins in the cerebrospinal fluid of treated patients [4][6]. Group 3: Future Research Directions - CRISPR gene editing technology shows promise for potentially providing a permanent cure by targeting and editing the mutated HTT gene [9]. - Recent studies have developed new gene editing delivery tools, such as RIDE, which successfully knocked out CAG repeat sequences in mouse models, leading to a reduction in toxic protein expression and improvement in disease symptoms [10]. - Base editing techniques have also demonstrated potential in interrupting repeat expansions associated with Huntington's disease, offering new strategies for treatment [12].