gene therapy

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

Biotech Adam Feuerstein

STAT Plus: Amicus Therapeutics acquires portfolio of gene therapies targeting neurologic disorders

Adam's Take Adam Feuerstein

STAT Plus: A gene therapy for overactive bladder? A waste of time and money that trivializes a lifesaving technology

First Opinion Fred D. Ledley

30 years is too long to wait for new medicines. There are ways to speed up drug development

Biotech Ike Swetlitz

STAT Plus: FDA plans to speed path to approval for some gene therapies, starting with hemophilia

First Opinion Maria Kefalas

How much would you pay for the miracle of gene therapy?

Biotech Eric Boodman

‘That’s $425,000 right there’ — the anxious launch of a gene therapy with a record sticker price

Pharmalot Ed Silverman

STAT Plus: Pharmalittle: Insulin study causes a big flap; Glaxo gets a reprieve thanks to a Novartis setback

Biotech Adam Feuerstein

STAT Plus: New paper raises safety concerns about neuro gene therapy, stocks slide

Health Associated Press

In a milestone year, gene therapy is finding a place in medicine