Black History Month is poignant for one family after a man got a historic, innovative treatment at Children's Hospital of.

Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol.

Our inbox has been flooded with emails from viewers and readers who want to share their personal stories — people who want to ...

Celebrate International Day of Women and Girls in Science 2026 by discovering some of the most influential women shaping ...

FDA RMAT boosts inhaled gene therapy KB707, delivering IL‑2/IL‑12 to lung tumors; early trial shows responses with manageable ...

Big pharma and biotech take the earnings stage with reports from Eli Lilly and Novo Nordisk leading the lineup. Will they ...

The FDA has cleared the company's investigational new drug application seeking to begin the Phase I/II FUNCTION-DMD trial.

Ever since 6-year-old Emily Whitehead became the first pediatric patient to receive CAR-T cell therapy in 2012, when T cells ...

Dr. Sarah Tabrizi and her team at University College London have been studying Huntington’s treatments for two decades, and led the small but ground-breaking study of Uniqure’s treatment. Results ...

When KJ Muldoon was born in August 2024 with mutations that led to a missing enzyme he needed to process proteins properly, Ahrens-Nicklas knew she had to act fast. Most with his condition need a ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

As many had predicted following news of a clinical hold on the gene therapy last month, Regenxbio Inc. disclosed receipt of a complete response letter (CRL) regarding its BLA for RGX-121 (clemidsogene ...