Tag: Gene Therapy
-
NanoMosaic Earns FDA AMT Designation for Advanced Gene Therapy Analytics
NanoMosaic Secures FDA AMT Designation for Advanced Gene Therapy Analytics NanoMosaic has been awarded the FDA’s Advanced Manufacturing Technology (AMT) designation, a status reserved for platforms that demonstrably improve efficiency, product quality, and regulatory readiness in complex manufacturing environments. This recognition underscores NanoMosaic’s commitment to advancing gene therapy analytics and supporting developers navigating the regulatory…
-
NanoMosaic Secures FDA AMT Designation to Accelerate Advanced Gene Therapy Analytics
NanoMosaic Secures FDA AMT Designation to Accelerate Advanced Gene Therapy Analytics NanoMosaic has earned the U.S. Food and Drug Administration’s (FDA) Advanced Manufacturing Technology (AMT) designation, signaling a pivotal step for developers and manufacturers leveraging the company’s analytics platform for gene therapies. The designation aims to streamline regulatory engagement, improve product quality, and expedite the…
-
NanoMosaic Earns FDA Advanced Manufacturing Technology Designation to Accelerate Gene Therapy Analytics
Overview: A Milestone for Gene Therapy Analytics NanoMosaic has secured the U.S. Food and Drug Administration’s (FDA) Advanced Manufacturing Technology (AMT) designation for its cutting-edge analytics platform. This recognition underscores the company’s ability to streamline complex gene therapy workflows, enhance product quality, and improve FDA engagement for developers and manufacturers pursuing advanced therapies. As gene…
-
Sarepta’s ELEVIDYS Demonstrates Slowed Disease Progression in Ambulatory Duchenne Patients: Positive EMBARK Topline Three-Year Results
New EMBARK topline results highlight a meaningful impact of ELEVIDYS in ambulatory Duchenne patients Sarepta Therapeutics today reported positive topline results from three years of follow-up in the EMBARK study, evaluating ELEVIDYS, its adeno-associated virus (AAV) gene therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (DMD). The company described sustained clinical…
-
ELEVIDYS Slows Duchenne Progression: Three-Year EMBARK Positive Topline Results
Overview of EMBARK Topline Results Sarepta Therapeutics announced positive topline results from three years of follow-up in the EMBARK study evaluating ELEVIDYS, the company’s gene therapy designed for ambulatory Duchenne muscular dystrophy (DMD). The data focus on functional outcomes in a cohort of treated boys with an average age of about nine years and demonstrate…
-
ELEVIDYS EMBARK Results: Slowed Duchenne progression in Ambulatory Patients
Overview of ELEVIDYS EMBARK topline findings Sarepta Therapeutics has reported positive topline results from the three-year EMBARK study evaluating ELEVIDYS, a gene therapy designed for ambulatory patients with Duchenne muscular dystrophy (DMD). At a mean age of about 9 years, patients treated with ELEVIDYS achieved mean North Star Ambulatory Assessment (NSAA) scores that surpassed their…
-
New Genetic Clues in Retinitis Pigmentosa Bring Hope for Future Treatments
Groundbreaking findings reveal five new genetic causes of retinitis pigmentosa In a major advance for eye health research, scientists analyzing data from Genomics England’s National Genomic Research Library (NGRL) and other sources have identified five new genetic causes of retinitis pigmentosa (RP). RP is a progressive eye condition that damages the retina’s light-sensing cells, often…
-
Five New Genetic Causes of Retinitis Pigmentosa Found, Offering Hope for Sight Loss Patients
New genetic clues deepen understanding of retinitis pigmentosa Scientists analyzing data from Genomics England’s National Genomic Research Library (NGRL) and other sources have identified five new genetic causes of retinitis pigmentosa (RP), a degenerative eye condition that gradually blinds millions worldwide. The discovery adds to a growing body of knowledge about RP, a disease long…
-
From gene therapy breakthroughs to preventable disease outbreaks: Health trends shaping 2026
Overview: A Dynamic Health Landscape in 2026 As we enter 2026, medicine stands at the edge of a transformative era. Researchers are advancing functional cures for once-intractable diseases, novel gene therapies are entering broader clinical use, and global health systems are sharpening their readiness against preventable outbreaks. This convergence of scientific breakthroughs and proactive public…
-
Health Trends 2026: From Gene Therapy Breakthroughs to Preventable Disease Outbreaks
2026 Health Trends: A Leap Toward Functional Cures The world stands at a pivotal moment in medicine. As 2026 unfolds, researchers are accelerating breakthroughs that could redefine what is possible in disease prevention and treatment. From gene therapy breakthroughs aimed at functional cures for once-intractable diseases to targeted public health strategies designed to prevent outbreaks,…