Gene Therapy News Monitoring

IL2RG Gene Therapy for X-SCID

Pre-clinical validation of SIN-EFS-IL2RG.co vector-based gene therapy for X-SCID CHINA, March 13, 2025 /⁨EINPresswire.com⁩/ -- X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects …

Atsena Therapeutics Receives FDA Fast Track Designation for ATSN-201 Gene Therapy in X-Linked Retinoschisis

Atsena Therapeutics has been granted Fast Track designation by the FDA for ATSN-201, its investigational gene therapy designed to treat X-linked retinoschisis (XLRS), a rare inherited retinal disease with no approved treatments. A Milestone in XLRS Gene …

Wesley Center for Immunotherapy Researchers at UH Seidman Cancer Center Honored with Top Abstract Award at 2025 ...

A cutting-edge breakthrough in car T-cell therapy manufacturing has garnered significant attention within the scientific community. A research team from the Wesley Center for Immunotherapy at the University Hospitals Seidman Cancer Center in Cleveland, …

Advances in Gene Therapy, Neuroregeneration at the Forefront of MDA 2025

Muscular Dystrophy Association (MDA), established in 1950, and is set to convene for its 75th meeting. Looking ahead to this year’s event, Sharon Hesterlee, PhD, chief research officer at MDA, praised the breadth of research to be presented. With …

MeiraGTx partners with cryptic AI startup Hologen to advance Parkinson's gene therapy

Once vaunted as a cure, gene therapy has hit hard times

New Clinical Trial at UTHealth Houston Tests Gene Therapy Vector That Delivers Drug Aimed at Stopping Overactive Bladder ...

A modified herpes virus that targets spinal cord nerve cells to treat neurogenic bladder in people with spinal cord injury is underway in a first-in-human clinical trial by UTHealth Houston at TIRR Memorial Hermann. Neurogenic bladder, also called …

Children's National research chief eyes gene therapy as 'tip of the iceberg'

At Children's National Hospital in Washington, D.C., research is key to advancing innovation in pediatric care. Catherine Bollard, MD, senior vice president and chief research officer of its research institute, told Becker's that the future is …

Around the Helix: Cell and Gene Therapy Company Updates – March 12, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, …

FDA Awards Fast Track Designation to ATSN-201 Gene Therapy for XLRS

The US Food and Drug Administration (FDA) has granted Fast Track designation to ATSN–201 for the treatment of X-linked retinoschisis (XLRS), marking the third FDA designation for the novel gene therapy.1 Announced by Atsena Therapeutics, on March 12, 2025, …

The most expensive drug ever approved: A gene therapy that cures butterfly skin and could cost $20 million per patient

When Mía Millán was born at Linares Hospital in the early hours of August 11, 2017, one of the first things that caught her parents’ attention was a large, bright red spot on her right leg: “The doctors said it could be an angioma,” recalls Ana González. …

Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

DURHAM, N.C., March 12, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug …

Mutated DNA Restored to Normal in Gene Therapy Advance

Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene. This was the first time a mutated gene has been restored to normal. The small study of nine patients announced …

Life With Hemophilia, Reimagined: Ryan's Gene Therapy Journey

He didn’t know it then, but when Ryan was little, his sister would dedicate her birthday wish each year to one request: that Ryan’s hemophilia would be cured.   Maybe all that wishing paid off. At age 22, his doctor at Children's Hospital Los Angeles …

Children's Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating ...

Philadelphia March 11, 2025 – In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), …

Calidi’s Rtnova platform delivers transient gene therapy payloads to tumors

Immuno-oncology Calidi Biotherapeutics Inc. has released promising preclinical results for its systemic Rtnova platform showing its ability to successfully deliver transient gene therapy payloads to targeted tumors. Moreover, the company’s tumor-specific …

Sickle cell gene therapy frees teen from life of pain

A Fayetteville teen is making history in North Carolina after undergoing a new gene therapy for sickle cell disease. Aaron Scott was diagnosed with sickle cell at birth. Sickle cell is a genetic blood disorder that effects the protein which effects the …

Roche Absorbs $2.4B Impairment in Overhaul of Spark Gene Therapy Unit

Roche is currently enacting a “fundamental reorganisation” of its gene therapy subsidiary Spark Therapeutics amid broader shifts in its pharmaceuticals business. The move, first revealed in the company’s financial report published in January 2025, comes …

Medera Completes Patient Dosing in Phase 1/2a MUSIC-HFrEF Trial of SRD-001 Gene Therapy for Heart Failure with Reduced ...

BOSTON, March 11, 2025 (GLOBE NEWSWIRE) -- Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics, today announced …

Digital Marketing Agency is Awarded Trademark: Turning Clicks Into Clients®

PHILADELPHIA, PA, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Results Driven Marketing, LLC proudly announces that it holds the official trademark for Turning Clicks Into Clients®, as conferred by the United States Patent & Trademark …

fija partners with softstack in smart contract auditing

Fija enhanced smart contract security and fund protection following softstack’s in-depth audit, with all identified risks addressed. FLENSBURG, SCHLESWIG-HOLSTEIN, GERMANY, March 13, 2025 /⁨EINPresswire.com⁩/ -- DeFi security is more critical than …

Vycor Medical Joins B2i Digital Venture Company Program to Build and Educate Its Investor Base

B2i Digital to Showcase Vycor Medical's Neurosurgical and Vision Rehabilitation Technologies Vycor Medical, Inc. (OTCQB:VYCO) NEW YORK, NY, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- B2i Digital is pleased to announce that Vycor Medical, …

GOGov Expands into the Transportation Sector with the Launch of the I-81 Connect Mobile App

GOGov expands into transportation with I-81 Connect, a mobile app for NYSDOT’s I-81 project. PATCHOGUE, NY, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- GOGov, a leader in local government technology solutions, is proud to announce its expansion …

JARIET to Demonstrate 64 GSPS Direct RF-Sampling at GOMACTech 2025

GOMAC is the Government Microcircuit Applications & Critical Technology Conference REDONDO BEACH, CA, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Jariet Technologies invites you to visit our booth at GOMAC Tech 2025 in Pasadena, CA …

Mainstream Fluid & Air Joins AMCA International to Further Innovation in Air Movement Industry

Mainstream Fluid & Air joins AMCA, reinforcing its commitment to innovation, industry standards, and high-performance air movement solutions. BERKELEY HEIGHTS, NJ, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Mainstream Fluid & …

WSI’s AI Market Research Survey Enters Its Second Year as an Industry Benchmark for SMB Digital Transformation

TORONTO, ONTARIO, CANADA, March 13, 2025 /⁨EINPresswire.com⁩/ -- WSI, the world's largest network of digital marketing consultants, is continuing its commitment to understanding AI adoption among small and medium-sized businesses (SMBs) as it …

KraLos and WEBOUNCER: A New Standard in Cybersecurity Led by Carsten Klein

BURGDORF, GERMANY, March 13, 2025 /⁨EINPresswire.com⁩/ -- As cyberattacks become increasingly sophisticated and frequent, KraLos GmbH, based in Burgdorf near Hanover, continues to develop cybersecurity solutions. With their patented cybersecurity …

Amitek Expands IT Support Services Across Berkshire and Reading

WOKINGHAM, BERKSHIRE, UNITED KINGDOM, March 13, 2025 /⁨EINPresswire.com⁩/ -- Amitek, a leading provider of managed IT solutions, has announced the expansion of its specialised IT support services tailored for businesses in Berkshire and Reading. …

ETA Track Plus Unveils Smart-Trip AI Report & Advanced GPS Fleet Tracking for Smarter Fleet Management in Dallas, Texas

ETA Track Plus, unveils its latest innovation—the Smart-Trip AI Report, a comprehensive GPS fleet tracking system. DALLAS, TX, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- ETA Track Plus, a leader in GPS tracking technology and fleet …

Integral BioSystems to Present Data on its Flagship Drug Delivery Platforms May 4-7 at ARVO 2025 in Salt Lake City, Utah

Boston area drug delivery firm Integral BioSystems LLC will present key research data May 4-7, 2025 at the Salt Palace Convention Center, Salt Lake City, Utah. BEDFORD, MA, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Integral BioSystems …

Data Foundation CEO Nick Hart Joins Forbes Technology Council; Advocates for Next Steps on America's Data Strategy

Inaugural article outlines vision for the future of U.S. data infrastructure as a catalyst for innovation and economic growth WASHINGTON, DC, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Dr. Nick Hart, President and CEO of the Data …

TECNO launches the new Camon 40 series MWC 2025

TECNO, a force in mobile innovation, continues to redefine the industry with groundbreaking AI capabilities, ultra-thin engineering, and futuristic designs. BARCELONA, SPAIN, March 13, 2025 /⁨EINPresswire.com⁩/ -- TECNO, a force in mobile …

UnifyCloud's CloudAtlas AI Factory Now Available on Microsoft Azure Marketplace

UnifyCloud simplifies AI adoption with CloudAtlas AI Factory, now accessible through the Microsoft Azure Marketplace for rapid AI proof-of-concept development. REDMOND, WA, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- UnifyCloud, a global …

Industrial Heat Pumps: A Key Solution for Sustainable Heating and Cooling

Increasing energy efficiency drives demand for industrial heat pumps, which capture and reuse waste heat, reducing energy use and operational costs. WILMINGTON, DE, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- According to a new report …

Revolutionizing Philanthropy: AI-Powered Platform Bridges Donors with Community Needs

Northern Cincinnati Foundation launches revolutionary AI platform, connecting donors to community needs with smart matching, impact analytics & seamless giving. CINCINNATI , OH, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- In a …

Custom Audio Systems Designed to Enhance Any Atmosphere

SLIDELL, LA, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Audio plays a key role in shaping the atmosphere of a space. Whether used in homes, businesses, event venues, or entertainment spaces, a well-designed audio system enhances the …

Global Product Analytics Market Share Registering CAGR of 22.7% Projected to Reach USD 76.7 Billion by 2031

WILMINGTON, DE, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Allied Market Research published a new report, titled, " Global Product Analytics Market Share Registering CAGR of 22.7% Projected to Reach USD 76.7 Billion by 2031 ." The …

St. John's Episcopal School Unveils State-of-the-Art SmartLab

Innovative Learning Environment Enhances STREAM Education for Students MCALLEN, TX, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- St. John's Episcopal School proudly hosted a ribbon-cutting ceremony on Thursday, January 30th, to …

Ateios Systems Unveils Gen 3 RaiCore™ Electrodes, Driving the Next Era of PFA-Free, High-Performance Batteries

Gen 3 RaiCore™ electrodes deliver higher energy density, enhanced conductivity, and 5X faster manufacturing—all PFA-free. NEWBERRY, IN, UNITED STATES, March 13, 2025 /⁨EINPresswire.com⁩/ -- Ateios Systems has unveiled their newest electrode …