The MarketWatch News Department was not involved in the creation of this content. EXTON, PA, Aug. 14, 2025 (GLOBE NEWSWIRE) -- As the first wave of gene therapies for transfusion-dependent ...
Clinical Trials Arena on MSNOpinion
Sustaining long-term follow-up in cell and gene therapies
IQVIA’s Diego Correa and Jip Klerks reflect on how engagement in CGT trials can be maintained with flexible, patient-centered ...
Andelyn Biosciences, a leading contract development and manufacturing organization, announced today that Genprex, Inc. has selected Andelyn to support scale-up manufacturing for its diabetes gene ...
Researchers developed a bioinspired lipid nanoparticle that delivered a PCSK9 gene editor to the liver. The approach reduced ...
Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...
The inaugural Genetic Agency Technology Conference (GATC) on November 11th, 2025 will convene technical thought leaders, gene therapy developers, patients, their families, and patient advocates across ...
News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...
Accelerated approval relies on cornstarch-reduction as a surrogate endpoint, with crossover follow-up planned at 96 and 144 ...
Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...
Phase 2 GenePHIT trial successfully randomized and completed enrollment of one of the largest gene therapy trials conducted in heart failure to date, with more than 170 participants randomized across ...
Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...
Key opportunities include automated, closed and digitally monitored production; end-to-end CDMO services; viral vector capacity; scalable autologous and allogeneic platforms; regional manufacturing; ...
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