FDA tightens Duchenne gene therapy after deaths of two teens

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...
WYFF News 4 on MSN

Greer, South Carolina, family hosts fundraiser for gene therapy to treat children with rare ...

A Greer couple is hosting a fundraiser to support a costly gene editing procedure for their two children, who suffer from a ...

RNA therapy silences KRAS gene and boosts immune attack on cancer cells

Two complementary studies led by researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS ...
腾讯网

复旦大学舒易来团队Trends in Molecular Medicine综述丨范式变革：临床基因 ...

先天性耳聋患者约2600万，常伴有言语障碍，甚至影响智力发育，一直以来没有临床治疗药物。2022年，复旦大学附属眼耳鼻喉科医院完成全球首例OTOF遗传性耳聋患者基因治疗，成为先天性耳聋首次基因治疗临床实践，成功恢复了患者听力。此后又有7项基因治疗临床 ...
腾讯网

Cell Press头条发布遗传性耳聋基因治疗国际专家共识

医学Medicine2025年10月23日，由复旦大学领衔的全球首个遗传性耳聋基因治疗国际专家共识在Cell Press细胞出版社旗下医学旗舰刊Med上正式发布[1]，并登上Cell Press官网头条。论文题为“International ...
Fred Hutchinson Cancer Center

Scientists see foam as starting point of a path to bedside gene therapy

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

下一代细胞与基因疗法（CGT）：攻克实体瘤与实现体内递送的创新 ...

细胞与基因疗法 (Cell and Gene Therapy，CGT)正以前所未有的速度重塑现代医学的边界。自2017年首款 CART 疗法获批以来，CGT ...

YolTech Therapeutics Receives FDA IND Clearance to Initiate Global Pivotal Trial of In Vivo ...

YolTech Therapeutics, a clinical-stage biotechnology company developing next-generation in vivo gene editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the ...
The Daily Pennsylvanian

Penn Medicine, CHOP to begin testing specialized gene therapy after working on new FDA protocol

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.
STAT

FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk

Risk of “acute, serious, and life-threatening” heart inflammation is now on the label of Sarepta's gene therapy for Duchenne ...

Thermo Fisher Scientific Opens East Coast Collaboration Center at BioLabs for Advanced ...

New state-of-the-art facility in Philadelphia provides integrated lab and collaboration space along with tailored guidance to help move advanced therapies from early research to clinical readiness ...

Cell and gene therapy training

Hyderabad: BRIC-Translational Health Science and Technology Institute (BRIC-THSTI), in collaboration with Miltenyi Biotec and ...