Gene therapy

Politics Nicholas Florko

STAT Plus: FDA’s vaccines and biologics chief talks Covid-19, 2022 priorities, and getting back to gene, cell therapy review

Biotech Adam Feuerstein

STAT Plus: Patients with rare blood disorder benefit from Bluebird gene therapy while denied access in Europe

Pharmalot Ed Silverman

STAT Plus: What’s in a name? Spark Therapeutics sues Bluebird Bio over the use of the word ‘spark’

Biotech Jonathan Saltzman — Boston Globe

STAT Plus: Affinia Therapeutics raises $110 million for gene therapy treatments

Biotech Adam Feuerstein

STAT Plus: Bluebird’s withdrawal of therapy from Germany could chill talks over gene therapy prices across Europe

Biotech Adam Feuerstein

STAT Plus: Uniqure investigation clears hemophilia B gene therapy as cause of patient’s liver cancer

Biotech Adam Feuerstein

STAT Plus: Bluebird suspends studies of sickle cell gene therapy following cancer diagnoses in two more treated patients

Biotech Adam Feuerstein

STAT Plus: Sarepta gene therapy for Duchenne muscular dystrophy stumbles with failed study outcome

Biotech Kate Sheridan

STAT Plus: Startup Spotlight: Encoded eyes a gene therapy for Dravet syndrome

Biotech Adam Feuerstein

STAT Plus: New rare disease gene therapy startup recruits former Sarepta executive as CEO

Biotech Damian Garde

STAT Plus: In a shocking decision, FDA rejects BioMarin’s gene therapy for hemophilia

Health Elizabeth Cooney

STAT Plus: Recapping ASCO 2020, talking cancer therapy combinations, and looking to the future

Biotech Matthew Herper

STAT Plus: Pfizer says efficacy of its Duchenne gene therapy outweighs side effects

Biotech Kate Sheridan

STAT Plus: This biotech just bought three failed drugs. Here’s why

In the Lab Rebecca Robbins

STAT Plus: Gene therapy pioneer says the field is behind — and that delivery technology is ‘embarrassing’

First Opinion Jeremy Schafer

Should employers take a chance on little-known Embarc to pay for gene therapy?

Pharmalot Ed Silverman

STAT Plus: Novartis unit says FDA wasn’t told of data manipulation sooner because it was ‘highly complex’

Biotech Jonathan Saltzman — Boston Globe

STAT Plus: The world’s most expensive medicine could save toddlers’ lives. But getting it is complicated

Biotech Adam Feuerstein

STAT Plus: Bluebird’s gene therapy for a rare blood disease will cost $1.8 million. Cue the pricing debate

Biotech Damian Garde

STAT Plus: Europe approves first gene therapy to treat rare blood disease

Biotech Adam Feuerstein and Matthew Herper

STAT Plus: Biomarin to seek approval for hemophilia gene therapy, but durability questions persist

Pharma Adam Feuerstein

STAT Plus: At $2.1 million, newly approved Novartis gene therapy will be world’s most expensive drug

Biotech Jonathan Saltzman — Boston Globe

STAT Plus: Novartis CEO coy about pricing its gene therapy for spinal muscular atrophy

Biotech Adam Feuerstein

STAT Plus: Tocagen continues a brain cancer gene therapy clinical trial. Investors fear the worst

Biotech Adam Feuerstein

STAT Plus: BioMarin’s hemophilia gene therapy faces a crucial durability test. Here are 9 things to know

Biotech Adam Feuerstein

STAT Plus: With positive early results, Sarepta appears to have a second gene therapy with potential

Biotech Matthew Herper

STAT Plus: Solid Biosciences to soldier on as initial muscular dystrophy gene therapy results disappoint

Biotech Adam Feuerstein

STAT Plus: With new data, a biotech tries to relieve doubts about the durability of its gene therapy for Fabry disease

Adam's Take Adam Feuerstein

STAT Plus: Misfortune strikes a Bluebird patient, highlighting the safety risks involved with gene therapy