Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...
Interesting Engineering on MSN
First-ever experimental gene therapy seeks to restore vision by rejuvenating eye neurons
Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.
A Johnson & Johnson gene therapy in development for an inherited vision-loss disorder has failed a Phase 3 study, the latest clinical research setback for a rare eye disease that so far has no ...
Adeno-associated virus (AAV) has emerged as a reliable vehicle for delivering therapeutic DNA into patient cells, with eight AAV-based gene therapies approved worldwide as of mid-2025. 1 Yet questions ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this rare form of SMA.
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