ARPA-H invests $160 million to make custom gene editing routine
ARPA-H, the U.S.’ “moonshot” agency for health research, announced Thursday that it will spend up to $160 million…
How one father built a biotech, Grace Science, to save his daughter
Highlights Matt Wilsey has spent a decade and $70 million trying to develop a cure for his daughter…
Bryan Johnson’s diagnosis shines light on hard-to-detect disease
Longevity entrepreneur Bryan Johnson shared some bad news on social media recently. “I have an autoimmune disease. My…
Louisiana Notches a Sickle Cell First
A 23-year-old from suburban New Orleans may have just opened a new chapter in how Louisiana treats…
Scientists Are Attempting to Reprogram Old Human Cells to Be Young Again
“Hearst Magazines and Yahoo may earn commission or revenue on some items through these links.” Here’s what you’ll…
‘Edited’ human embryos reveal secrets of our development — and fuel ethical debate
A human embryo ‘base edited’ so that it can’t produce a key protein (right), fails to form the…
Gene therapies have high price tags. A financing model could help
A new class of medicines is transforming health care. Gene therapies can now cure diseases like sickle cell…
Gene therapies to fix failing hearts gain steam after years in the doldrums
Heart-muscle cells (in close-up, artificially coloured) do not normally proliferate in adult mammals, but scientists are trying to…
In rare case, scientists link boy’s tumor to gene therapy viruses
When 13-month-old Adam’s stem cell transplant failed, doctors had two options: a second transplant, which they warned carried…
Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
Yasunaga, S. et al. A mutation in OTOF, encoding otoferlin, a FER-1-like protein, causes DFNB9, a nonsyndromic form…
Masked mitochondria slip into cells to treat disease in mice
Mitochondria (artist’s impression) wrapped in red-blood-cell membranes can sneak into cells without being tagged for destruction.Credit: Alfred Pasieka/SPL…
FDA rejects Regenxbio’s MPS II rare-disease gene therapy
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…