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gene therapy

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Biotech Adam Feuerstein

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

Biotech Kate Sheridan

Startup Spotlight: Encoded eyes a gene therapy for Dravet syndrome

Biotech Adam Feuerstein

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

Biotech Damian Garde

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

Biotech Kate Sheridan

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

In the Lab Rebecca Robbins

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?

Pharma Adam Feuerstein

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

All Coverage

Biotech Jonathan Saltzman — Boston Globe

Affinia Therapeutics raises $110 million for gene therapy treatments

Biotech Adam Feuerstein

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

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Biotech Adam Feuerstein

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

Biotech Adam Feuerstein

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

Health Elizabeth Cooney

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

Biotech Matthew Herper

Pfizer says efficacy of its Duchenne gene therapy outweighs side effects

Pharmalot Ed Silverman

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

Biotech Jonathan Saltzman — Boston Globe

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

Biotech Adam Feuerstein

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

Biotech Damian Garde

Europe approves first gene therapy to treat rare blood disease

Biotech Adam Feuerstein and Matthew Herper

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

Biotech Jonathan Saltzman — Boston Globe

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

Biotech Adam Feuerstein

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

Biotech Adam Feuerstein

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

Biotech Adam Feuerstein

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

Biotech Matthew Herper

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

Biotech Adam Feuerstein

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

Adam's Take Adam Feuerstein

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

Biotech Adam Feuerstein

Amicus Therapeutics acquires portfolio of gene therapies targeting neurologic disorders

Adam's Take Adam Feuerstein

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

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

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