Gene therapy

Gene therapy is a modern medical technology that can be used against both inherited diseases, such as sickle cell disease, or acquired diseases such as cancer. It entails adding new copies of a gene that may be broken, replacing a gene that’s missing, or replacing a gene that’s defective.

A key term in gene therapy is “vector,” that is, the material used to deliver new DNA. Vectors are often based on viruses that have had the viral genes removed and replaced with therapeutic ones.

In ex vivo gene therapy, cells are removed cells from the patient, new genetic material is inserted via a vector, and then the cells are returned to the patient. With in vivo gene therapy, the vector is directly infused into the bloodstream or injected into an organ.

Stories in this archive include gene therapy research for congenital deafness and Duchenne muscular dystrophy, and an article on why the world’ most expensive drug may not be overpriced.

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STAT Plus: Gene therapy for sickle cell disease remains a tough business model — and may never work

STAT Plus: Bluebird Bio is laying off 25% of its workers to cut costs

STAT Plus: In race to make gene therapy for age-related blindness, 4D Molecular announces positive results

STAT Plus: A rare disease program, portrayed as both lifeline and pharma handout, is set to expire

STAT Plus: ‘Sarepta’s like a curse on me’: FDA commissioner dismisses controversy over Elevidys

STAT Plus: Q&A: Atsena Therapeutics’ gene therapy for inherited blindness shows promise

Breakthrough therapies have given Duchenne muscular dystrophy families like mine hope — and new fears

STAT Plus: Gene therapy pioneer Jim Wilson believes he can commercialize rare-disease drugs in a tough climate

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STAT Plus: With new data, Regenxbio advances plan for Duchenne gene therapy

STAT Plus: Jiankui He, creator of CRISPR-edited children, relocates to a Chinese medical tourism hub

Vertex sues U.S. government over access to fertility support services for Casgevy patients

STAT Plus: Early data from Lexeo’s gene therapy for rare heart disease shows promise

STAT Plus: Duchenne gene therapy setback, Alzheimer’s drug endorsement & why a STAT reporter buys weed (for journalism)

STAT Plus: Pfizer’s gene therapy for Duchenne muscular dystrophy fails in Phase 3 trial

STAT Plus: Ultragenyx gene therapy for rare liver ailment succeeds in late-stage trial

STAT Plus: Gene therapies for deafness dredge up an old question: Do deaf people want a ‘cure’?

STAT Plus: Q&A with Jennifer Adair, researcher on a mission to increase global access to gene therapies

STAT Plus: After gene therapy, two congenitally deaf children hear for the first time

STAT Plus: Young boy dies in trial for Pfizer Duchenne gene therapy

STAT Plus: Moderna ends collaboration with CRISPR company Metagenomi, parts ways with gene editing chief

STAT Plus: Bernie Sanders targets ‘outrageously high price’ of Ozempic

STAT Plus: Use evidence to support early coverage of gene therapy after accelerated approval

STAT Plus: Why the world’s most expensive drug might not be all that overpriced

STAT Plus: How biotech investors spot opportunity when stocks are down — and up

STAT Plus: New gene therapy, to be priced at $4.25 million, has already transformed children’s lives

STAT Plus: FDA approves first gene therapy for fatal neuron disease in children

A dilemma in ALS, the first MASH drug, & why gene therapy is hard

STAT Plus: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?

Meet the fetal surgeon forging CRISPR’s next frontier: curing diseases in the womb