Gene Therapy News Monitoring

Taysha Gene Therapies Stock Soars After FDA Grants Breakthrough Therapy Designation To Gene Therapy

Taysha also announced that it has finalized alignment with the FDA on the protocol for the trial intended to support its application for approval of TSHA-102. Shares of Taysha Gene Therapies, Inc. (TSHA) rallied 49% by Thursday morning after the U.S. Food …

<![CDATA[Expert: Navigating Costs, Regulations, and Patient Care in Gene Therapy]]>

Varshini Kanukollu, PharmD, BCOP, pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering, continued her conversation with Pharmacy Times®, discussing the significant financial and regulatory considerations pharmacists must address when …

EG 427’s HSV gene therapy reduces urinary incontinence in Phase Ib/IIa trial

French biotech EG 427 has had success with its urinary incontinence DNA therapy, EG110A, showing a more than 80% reduction in episodes. In the Phase Ib/IIa trial (NCT06596291), the lowest dose of EG110A was found to reduce the incidence of urinary …

Gene therapy startup launches with $11M to tackle aging disease of the eye

Cell and Gene Therapy CMC Trends in Biopharma Manufacturing

The cell and gene therapy landscape continues to experience unprecedented growth and innovation, with cell and gene therapy CMC trends fundamentally reshaping how biopharmaceutical companies approach the development and manufacture of these revolutionary …

How Cell and Gene Therapy Developers Can Reduce Risk of FDA Complete Response Letters With Proactive CMC Strategies

The growth of cell and gene therapies (CGTs) has brought both promise and complexity to healthcare. While these products are transforming patient care, CGTs present new and dynamic regulatory challenges for companies and health authorities, particularly …

<![CDATA[Expert: Pharmacists Guide the Future of Gene Therapy in Oncology and Hematology]]>

In an interview with Pharmacy Times®, Varshini Kanukollu, PharmD, BCOP, pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering, discussed how gene therapy offers a new treatment approach by modifying a patient’s cells to address the …

Illinois Secures Federal Funding to Expand Sickle Cell Disease Care and Gene Therapy Access

Illinois is set to boost care for individuals with Sickle Cell Disease as it joins a federal program focused on gene therapy access. The state will receive nearly $9.55 million in federal funding over the next decade. This initiative aims to enhance …

Pharma Pulse: Trump Plans Direct-to-Patient Website, Gabapentin Prescriptions Surge, and Gene Therapy Slows Huntington’s ...

Welcome to Pharma Pulse, a Pharmaceutical Commerce podcast where we bring you the latest insights shaping patient access, supply chain/logistics, data & tech, and healthcare innovation. I’m your host, and today we’re covering a new government …

Illinois announces participation in federal Cell & Gene Therapy Access Model for Sickle Cell Disease treatment

Exclusive: French startup's gene therapy dramatically reduces urinary incontinence in spinal cord injury patients

Fatal genetic disease, Huntington’s, successfully treated with new gene therapy

By Violet Hollar Correspondent  A new gene therapy developed by the University College London Huntington’s Disease Center has shown miraculous success in reducing the fatal genetic disorder’s progression by 75%, according to the BBC.  Huntington’s Disease …

<![CDATA[Novel Gene Therapy Significantly Slows Progression of Huntington Disease]]>

The phase 1/2 clinical trial (NCT04120493) of AMT-130 (uniQure) for the treatment of patients with Huntington disease has met its prespecified primary end point, with a high dose of the gene therapy candidate demonstrating statistically significant …

Magnum Opus: Street drinks in LCA5 gene therapy phase I/II data

Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by the agency – evaluating OPGx-LCA5, a gene …

Gene therapy slows Huntington's disease for first time, clinical trial…

Huntington’s disease is a genetic brain disorder that causes symptoms like uncontrolled movement and decline in thinking skills. Treatment often focuses on symptom relief, but experts are seeking more effective interventions. Recent findings of a drug …

Revolutionary gene therapy offers hope for Huntington’s Disease patients

For the first time, doctors have successfully treated Huntington’s disease, one of the most devastating genetic conditions known to medicine.  Often described as a cruel combination of dementia, Parkinson’s, and motor neurone disease, Huntington’s …

VectorBuilder Wins Two IMAPAC Awards, Cementing Leadership in Gene Therapy and CDMO Innovation

CHICAGO--(BUSINESS WIRE)--Sep 30, 2025-- VectorBuilder, a global leader in the gene delivery space, has won two prestigious awards at the Asia-Pacific Cell and Gene Therapy Excellence Awards 2025 ( APCGTEA ) and the Asia-Pacific Biologics CDMO Excellence …

Opus says gene therapy for ultra-rare disease boosted vision in teens

New hope for Huntington’s families as gene therapy shows remarkable results

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. The news has spread quickly through families affected by this condition, who have been desperately waiting for a treatment that can stop …

Diligent Robotics Expands AI Advisory Board with Distinguished Leaders in Robotics and AI

Diligent Robotics deepens advisory expertise to accelerate safe, scalable AI solutions AUSTIN, TX, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Diligent Robotics, the leader in humanoid robotics and embodied AI for healthcare, today …

Mastering Line Drawing: How Everyday Users Are Getting Back to Creative Fundamentals with Xole AI

New Photo to Line Drawing Generator Transforms Visual Workflows for Creators, Patent Professionals, and Design Teams Worldwide LONDON, UNITED KINGDOM, October 2, 2025 /⁨EINPresswire.com⁩/ -- Xole AI, a leading innovator in accessible artificial …

Trenchless Sewer Solutions: Discount Drain Cleaning Brings Innovative Repairs to Las Vegas Homeowners

Discount Drain Cleaning offers trenchless sewer repairs and free inspections, protecting Las Vegas homes efficiently. LAS VEGAS, NV, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Discount Drain Cleaning, a trusted family-owned plumbing …

BTR: Intelligence in the Gray Zone – Rethinking U.S. Strategy in the New War on Cartels

WASHINGTON, DC, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- The United States has entered a new phase in its fight against transnational organized crime. When the federal government formally designated criminal cartels as foreign …

Newsmatics Turns 30: Online News Pioneer Launches Giveaway to Celebrate Three Decades of Innovation

WASHINGTON , DC, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Newsmatics is marking three decades of innovation in news distribution and technology. From its first products in 1995, EIN News and EIN Presswire, to Perspectify, Affinity …

Imitation Game: Personhood, Theology and AI’s Inevitability

Don Peterson and Clif Guy explore how AI intersects with theology, personhood, and the responsibility to live with conscience in a digital age. OVERLAND PARK, KS, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Divided Attention delivers …

Maven Collective Marketing Named PR Daily’s ‘Top Agency of the Year’ for Accelerating Microsoft Partner Growth

Microsoft Partner Marketing Leader Celebrates 40+ Global Awards, AI-Driven Innovation, and Record-Breaking Client Results VANCOUVER, BC, CANADA, October 2, 2025 /⁨EINPresswire.com⁩/ -- Maven Collective® Marketing has been named Top Agency of the …

Zayed Sustainability Prize Announces 2026 Finalists Driving Global Impact

ABU DHABI, UNITED ARAB EMIRATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- • 33 finalists selected from 7,761 submissions across 173 countries • This year’s finalists are pioneering inclusive, tech-enabled, and locally adapted solutions that …

The 'Quantum Minute' Named Best Podcast Series In 2025

Cybercrime Magazine Editors’ Choice Award goes to Applied Quantum SAUSALITO, CA, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- The editors at Cybercrime Magazine have named the Quantum Minute as its best podcast series in 2025. The …

Screen-Free Electric Vehicles: Amazon AWS and Olympian Motors to Pilot AI Cockpits in New York

Amazon AWS and Olympian Motors join forces to bring screen-free AI cockpits to electric vehicles, starting with the Model O1 in New York Amazon.com, Inc. (NASDAQ:AMZN) BROOKLYN, NY, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Olympian …

Gene Matrix Revolutionizes U.S. Healthcare with AI-Powered Genomics Platform, Announces Major Chicago Lab Expansion

Gene Matrix prevents 30,000+ adverse drug reactions yearly with AI genomics. Chicago biotech saves healthcare $450M. Series A funding launching soon. CHICAGO, IL, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Gene Matrix Revolutionizes U. …

EVO VC & Startups Launches Global Community for High-Growth Startups and Investors

Connecting high-growth startups, growing companies, and global investors through mentorship, capital access, and community. MIAMI, FL, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- EVO VC & Startups, a global startup and investor …

ChristianaCare & hunterAI Share Fast ROI with AI Supply Chain Analytics at Chicago AHRMM Lunch & Learn

How to Watch: When: October 15, 2025 | 12:00–1:00 PM CT Location: Online via Microsoft Teams Meeting ID: 256 199 200 785 7 Password: ei252b54 SCOTTSDALE, AZ, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Chicago AHRMM invites …

Ultimate Endgamers League to Provide the First-Ever Multi-Genre Esports Ecosystem to Arizona State University

Create a space where students can compete, learn, and grow through esports, while building one of the most inclusive and diverse gaming communities in the world TEMPE, AZ, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- The Ultimate …

GrainChain Expands into Guatemala to Drive Digital Transformation in the Coffee Sector

GrainChain begins operations in Guatemala, partnering with FECCEG to digitalize coffee supply chains and expand its global footprint. MCALLEN, TX, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- GrainChain Expands into Guatemala, Bringing …

Baylor College of Medicine Migrates Blackbaud CRM to Concourse Cloud

LYNNWOOD, WA, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Concourse Hosting today announced the successful conversion and migration of Baylor College of Medicine’s fundraising systems—including a transition from Blackbaud Raiser’s Edge …

Profitable Pilates Launches Groundbreaking Mobile App to Empower Pilates Instructors Worldwide

The Profitable Pilates app serves as a comprehensive "business-in-your-pocket," putting a wealth of resources at the fingertips of owners anytime, anywhere. LAS VEGAS, NV, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Profitable …

K2G Presented First Line of AI Agents at Insurance CEE Expo 2025 in Zagreb

K2G Holdings AG unveiled its brand-new line of AI Agents at the Insurance CEE Expo in Zagreb on October 1, 2025. ZAGREB, CROATIA, October 2, 2025 /⁨EINPresswire.com⁩/ -- K2G Holdings AG (formerly kasko2go) unveiled its brand-new line of AI Agents …

CodeClouds Announces Launch of Enterprise Division to Deliver Scalable, High-Impact Digital Solutions

CodeClouds unveils new Enterprise division to meet growing demand for enterprise-grade development, integration, and digital transformation services. FORT WAYNE, IN, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- CodeClouds, a global …

Forbes Column by Jens Erik Gould Examines AI’s Limits and Why Human Expertise in Legal Ops Still Matters

Forbes column by Amalga Group's Jens Erik Gould explores AI’s limits in legal ops and why human expertise and judgment remain essential. NEW YORK, NY, UNITED STATES, October 2, 2025 /⁨EINPresswire.com⁩/ -- Jens Erik Gould, founder and CEO of …