Cell/Gene Tx channel feed – Endpoints News

Cell/Gene Tx channel feed

Sarepta leads off Duchenne MD gene therapy study with a ‘home run’ on efficacy — but can they deliver in 2 years?

7 years ago

Determined to build the leading biotech player in a new R&D space, Steven Holtzman lines up $55M for the next stage at Decibel

7 years ago

Solid Bio is back in business after the FDA lifts clinical hold on Duchenne MD gene therapy

7 years ago

Nightstar's drug for rare vision loss admitted to FDA's gene therapy speedway

7 years ago

CRI report tracks explosive growth of cell therapies in the global pipeline, as China researchers muscle toward the front

8 years ago

FDA chief Gottlieb is building a regulatory speedway to accelerate gene therapy development

8 years ago

Is biopharma’s first $1.5M therapy now in Phase III?

8 years ago

Bluebird’s gene therapy for beta-thalassemia falls short of a cure, but still wows in 22 patients

8 years ago

Full FDA letter in hand, Solid Bio says it's working to reverse clinical hold on its DMD gene therapy

8 years ago

Novartis CEO Vas Narasimhan cuts loose an old anchor and sets sail with $8.7B AveXis buyout

8 years ago

A gene therapy mystery? GenSight blasted as 1st PhIII eye study ends in an intriguing failure

8 years ago

Hauling in a $100M mega-round, gene therapy startup Generation Bio builds alternative to potentially risky viral vectors

8 years ago

Bellicum outlines a few quick steps the FDA wants before lifting its hold on lead cell therapy, shares rise

8 years ago

Bioverativ inks $100M gene therapy deal with Oxford BioMedica

8 years ago

With a potential cure for lysosomal storage disorders in hand, Avrobio raises $60M to back gene therapy pipeline

8 years ago

Brain damage cases force an FDA hold on Bellicum's lead cell therapy -- shares tank

8 years ago

Gene therapy pioneer James Wilson sounds an alarm on high-dose AAV studies following toxic reactions in monkeys

8 years ago

Bluebird hopes for 3 drug approvals by 2019, including CAR-T and gene therapies

8 years ago

J&J does Alzheimer's gene therapy deal, along with 14 other new collaborations

8 years ago

Atlas builds its dream team and hands over $25M to launch a new approach to gene therapy

8 years ago

Pfizer doubles down on Sangamo’s zinc finger tech, adding ALS to a mix of gene therapy alliances

8 years ago

The gene therapy pricing debate gets real as Spark sets $850,000 charge for its pioneering drug

8 years ago

In gene therapy's second big win this week, Orchard nabs $110M Series B

8 years ago

FDA rolls out an early and historic OK for Spark's pioneering gene therapy -- now let's talk price

8 years ago

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