An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working with regulators to ensure stable ongoing supply for the one-time gene therapy ...

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a revolutionary new method to improve compact gene-editing tools known as base ...

Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...