STAT+: In race to make gene therapy for age-related blindness, 4D Molecular announces positive results
A long-running race to develop a gene therapy for the most common cause of age-related…
Category: gene therapy
STAT+: A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
After his son Michael was diagnosed with a devastating, ultra-rare disease in 2019, Terry Pirovolakis…
STAT+: ‘Sarepta’s like a curse on me’: FDA commissioner dismisses controversy over approval
WELLESLEY, Mass. — At an event with journalists Tuesday outside of Boston, U.S. Food and…
Q&A: Atsena Therapeutics’ gene therapy for inherited blindness shows promise
Seven years after the FDA approved Luxterna, scientists have yet to bring another congenital blindness…
Opinion: Breakthrough therapies have given Duchenne muscular dystrophy families like mine hope — and new fears
Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take)…
STAT+: Gene therapy pioneer Jim Wilson believes he can commercialize rare-disease drugs in a tough climate
If you’d told Jim Wilson three years ago that he’d be stepping down as director…
STAT+: A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases?
Will it work? It was the question everyone around Susannah Rosen kept asking. Since infancy,…
STAT+: The race to build better CRISPR delivery vehicles is heating up
If the first CRISPR decade was marked by a mad dash to evolve, engineer, and…
STAT+: BioMarin to restrict sales of hemophilia gene therapy, seeking to overcome slow sales
Struggling to find new patients, BioMarin Pharmaceuticals on Monday said it would restrict sales of…
STAT+: With new data, RegenxBio advances plan for Duchenne gene therapy
Regenxbio said Thursday that its experimental gene therapy for Duchenne muscular dystrophy was administered to…
STAT+: Jiankui He, creator of CRISPR-edited children, relocates to a Chinese medical tourism hub
Jiankui He, the Chinese scientist who sparked a global uproar in 2018 when he revealed…
STAT+: Pfizer gene therapy reduced hemophilia bleeds, but questions remain
Pfizer said Wednesday that its gene therapy for hemophilia A successfully reduced patients’ bleeds for…
Vertex sues U.S. government over access to fertility support services for Casgevy patients
One of the makers of the Casgevy — the world’s first CRISPR-based medicine — filed a lawsuit…
STAT+: Early data from Lexeo’s gene therapy for rare heart disease shows promise
Lexeo Therapeutics said Monday that a gene therapy designed to prevent fatal cardiac complications of…
Listen: Duchenne gene therapy setback, Alzheimer’s drug endorsement & why a STAT reporter buys weed (for journalism)
On this week’s episode of “The Readout LOUD,” our STAT colleague Nicholas Florko joins us…
STAT+: Pfizer’s gene therapy for Duchenne muscular dystrophy fails in phase 3 trial
Pfizer said Wednesday afternoon that a closely watched gene therapy for Duchenne muscular dystrophy failed…
STAT+: Ultragenyx gene therapy for rare liver ailment succeeds in late-stage trial
Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease…
STAT+: Gene therapies for deafness dredge up an old question: Do deaf people want a ‘cure’?
When Yilai Shu was training to be an otolaryngologist in Shanghai, in the mid 2000s,…
Q&A with Jennifer Adair, researcher on a mission to increase global access to gene therapies
BALTIMORE — Gene and cell therapies have transformed a handful of devastating disorders in the…
STAT+: Gene therapy trial targeting rare form of deafness shows ‘jaw-droppingly good’ results
Two congenitally deaf children can hear for the first time after being treated with gene…
STAT+: Young boy dies in trial for Pfizer Duchenne gene therapy
A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular…
STAT+: Moderna ends collaboration with CRISPR company Metagenomi, parts ways with gene editing chief
Three years ago, Moderna, flush with cash from Covid vaccine sales, announced it would start…
STAT+: Bernie Sanders targets ‘outrageously high price’ of Ozempic
Want to stay on top of the science and politics driving biotech today? Sign up to get…
Opinion: STAT+: Use evidence to support early coverage of gene therapy after accelerated approval
Gene editing and therapy to replace missing or defective genes is one of the most…
STAT+: Why the world’s most expensive drug might not be all that overpriced
The staff of the Institute for Clinical and Economic Review, or ICER, are known as…
STAT+: How biotech investors spot opportunity when stocks are down — and up
Following a blockbuster year of dealmaking and a recent uptick in biotech stocks, top investors…
STAT+: New gene therapy, to be priced at $4.25 million, has already transformed children’s lives
Every morning, Victoria Rasberry gets up before 6 a.m., walks to the bedroom down the…
STAT+: FDA approves first gene therapy for a fatal neuron disease in children
The Food and Drug Administration on Tuesday approved a gene therapy for metachromatic leukodystrophy, a…
Listen: A dilemma in ALS, the first MASH drug, & why gene therapy is hard
What happens when an approved drug doesn’t work? Why don’t patients want gene therapy? And…
STAT+: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?
As a boy growing up with hemophilia A, Noah Frederick reserved the end of his…
Meet the fetal surgeon forging CRISPR’s next frontier: curing diseases in the womb
SAN FRANCISCO — Outside, the August sun wasn’t yet visible through the thick folds of…
STAT+: Ring Therapeutics, a gene therapy startup, lays off 20% of staff
Ring Therapeutics, a gene therapy startup launched by Flagship Pioneering, has laid off just under…
STAT+: A gene therapy designed to heal skin wounds helped preserve a boy’s vision
In the clinical trial, the gene therapy seemed to be helping the boy’s skin wounds…
Texas Medicaid agrees to fully cover gene therapy for Afghan refugees’ infant
After a months-long fight with Texas Medicaid over coverage of a gene therapy, Afghan refugees…
STAT+: Can gene therapies make for a profitable business? Keep your eye on Bluebird Bio
Happy Groundhog Day eve. Some takes and thoughts on Bluebird Bio’s quest for gene therapy…
In partial reversal, Texas Medicaid greenlights first step in gene therapy for Afghan refugees’ infant son
The Pashai family of Dallas on Thursday got some good news, but not exactly the…
STAT+: Doudna institute hatches plan to ‘cure hundreds of diseases’ left behind by CRISPR revolution
Researchers attending gene therapy meetings over the past couple years were liable to bump into…
The Afghan father aided U.S. soldiers. Now Texas Medicaid won’t pay for his son’s gene therapy
On a sweltering day in August 2021, as the engines of a hulking C-17 cargo…
Gene therapy offered this 7-year-old freedom. The price: a grueling year
PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just…
STAT+: Could sickle cell someday be treated not with CRISPR, but with a pill?
Two gene therapies were approved for sickle cell disease last week, but for many researchers…
STAT+: Bone marrow transplant advance could widen use of this less buzzy cure for sickle cell disease
SAN DIEGO — The sickle cell community has for the past few days been buzzing…
Opinion: New sickle cell disease CRISPR treatments can’t make up for the shortage of adult hematologists
After decades of limited progress in the treatment of sickle cell disease (SCD), both patients…
STAT+: Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy
The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell…
Opinion: After living with sickle cell disease for 39 years, I’m both excited and skeptical about the newly approved gene therapies
For 39 years, I have lived with sickle cell disease. As of late, the sickle…
New gene therapies confront many sickle cell patients with an impossible choice: a cure or fertility
As a teenager, Marie Tornyenu was always having to explain herself. If it wasn’t the…
STAT+: Official: FDA needs staff influx to meet gene therapy needs
WASHINGTON — The Food and Drug Administration needs dozens more reviewers if it wants its…
STAT+: How sickle cell became the first disease treated by CRISPR
Nearly a decade ago, consultants delivered to Rodger Novak a kind of Sears catalog of…
Opinion: The FDA is at a crossroads on cell and gene therapies
Cell and gene therapies are the next frontier in medicine and promise long-sought hope for…
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Gene editing has had a big week in the spotlight. Here’s what’s going on
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Opinion: How the government can help lower the price — not just the cost — of cutting-edge gene therapies
At the bustling Chicago O’Hare airport, my luggage and I collided with an elderly couple….