Gene Therapy
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Fujifilm to invest $120M in gene therapy, build center in Texas
The Japan-based company plans to spend $55 million on the center while also outfitting its joint venture with new clean rooms and bioreactors.
By Kristin Jensen • Nov. 21, 2019 -
The hemophilia gene therapy race faces a critical year in 2020
While BioMarin looks poised to reach market in hemophilia A first, Sangamo and Spark are advancing rival gene therapies to tackle the blood disorder.
By Andrew Dunn • Nov. 20, 2019 -
Takeda sees cell, gene therapy in its future. Is it too late?
Nearly a year on from completing its $62 billion Shire deal, Takeda is pitching investors on its plans to stay one of the industry's leading rare disease drug developers.
By Ned Pagliarulo • Nov. 20, 2019 -
SponsoredThe evolving demands of the temperature-controlled supply chain for cell and gene therapy
Cell and gene therapies are heightening the need for advanced temperature control solutions.
By Gulam Jaffer, President, Yourway • Nov. 20, 2019 -
First look at CRISPR, Vertex gene-editing therapy hints at treatment potential
Results from two patients, one with sickle cell disease and the other with beta-thalassemia, offer an initial glimpse at the disease-altering potential of CRISPR gene-editing.
By Ned Pagliarulo • UPDATED: Nov. 19, 2019 at 8:38 a.m. -
Sarepta brings in more gene therapies with StrideBio deal
The deal adds four more neuromuscular candidates to the Exondys maker's already-rich pipeline.
By Jonathan Gardner • Nov. 14, 2019 -
ICER draws new gene therapy pricing framework
One-time and short-term curative therapies require a different approach to evaluating benefit, the cost watchdog group said.
By Jonathan Gardner • Nov. 13, 2019 -
Solid gene therapy trial halted again by FDA
Shares in Solid fell to a new low as the biotech's muscular dystrophy treatment dropped further behind the leader Sarepta Therapeutics.
By Jonathan Gardner • Nov. 12, 2019 -
Regenxbio, stung by trial hold on gene therapy, sues FDA
The biotech seeks to overturn clinical holds placed by the FDA on two gene therapy programs, arguing the agency action was "arbitrary and capricious."
By Ned Pagliarulo • Nov. 11, 2019 -
Lonza taps Cryoport to bolster cell and gene therapy delivery
The Swiss manufacturer inked a deal with the cold chain specialist, which works with sector leaders like Gilead, Novartis and Bluebird bio.
By Kristin Jensen • Nov. 07, 2019 -
Regenxbio hit with FDA clinical hold, pushes back gene therapy timelines
It's the second trial hold for the gene therapy field in recent weeks, after the FDA partially suspended a Novartis study testing the SMA treatment Zolgensma.
By Andrew Dunn • Nov. 06, 2019 -
Novartis gene therapy ambitions dealt another blow by FDA hold on Zolgensma
A partial trial suspension threatens to delay the gene therapy's expansion into older children, as the FDA works through safety findings from an animal study.
By Jonathan Gardner • Oct. 30, 2019 -
Gilead CAR-T sales slow, renewing growth doubts
Yescarta, the cell therapy Gilead acquired in its $12 billion deal for Kite, appeared to hit a snag, with sales from July to September declining versus the second quarter.
By Ned Pagliarulo • Oct. 25, 2019 -
Bluebird gets European green light for gene therapy production
Although Bluebird won EU approval for Zynteglo in June, the biotech delayed launching the one-time treatment to finalize manufacturing specifications.
By Kristin Jensen • Oct. 24, 2019 -
Novartis' Zolgensma starts strong as about 100 infants are treated with gene therapy
Sales totaled $160 million in the gene therapy's first full quarter of commercial availability, well above Wall Street's expectations.
By Andrew Dunn • Oct. 22, 2019 -
Gene therapy biotechs caught in manufacturing crunch
Rising demand for contract manufacturing services could push companies to invest more in their own capabilities despite the high cost.
By Ned Pagliarulo • UPDATED: Oct. 11, 2019 at 10:49 a.m. -
ProQR eye-disease data lend confidence that pivotal trial will succeed
The positive data also helps to keep the Netherlands drugmaker ahead of gene-editing specialists Editas and its partner, Allergan.
By Jonathan Gardner • Oct. 10, 2019 -
Feeling pressure in hemophilia, Novo signs gene editing deal with Bluebird
The deal may come too late, though, as the launch of Hemlibra and approach of gene therapies dampens demand for Novo's factor replacement therapies.
By Jonathan Gardner • Oct. 09, 2019 -
Sarepta, in shadow of FDA setback, marks gene therapy progress
The biotech released the first functional data from a trial of an experimental gene therapy treating a different type of muscular dystrophy than Exondys 51.
By Jonathan Gardner • Oct. 04, 2019 -
Gene therapy stocks have taken a beating. Their recovery may be slow
Share prices of at least 26 gene therapy biotechs are down from last October, a sign that investors may want more clarity on how the drugs will be sold.
By Jacob Bell • Oct. 03, 2019 -
Novartis to speed AveXis integration in wake of data manipulation
AveXis will be folded into Novartis' quality organization as a result of a scandal involving altered testing data for the company's gene therapy Zolgensma.
By Ned Pagliarulo • Sept. 24, 2019 -
Novartis, shadowed by data scandal, renews case for Zolgensma
Presymptomatic infants treated with the gene therapy reached motor milestones consistent with normal development, updated study results showed.
By Ned Pagliarulo • Sept. 19, 2019 -
Adverum sinks on first gene therapy data for eye disease
A decline in eyesight post-treatment reflects poorly on the therapy's potential, lengthening the company's odds of catching rival Regenxbio.
By Jonathan Gardner • Sept. 12, 2019 -
BioMarin talks down worries on gene therapy data differences
BioMarin's R&D head said timing of steroid treatment impacted interim Phase 3 data, which had spurred questions on valrox's durability.
By Andrew Dunn • Sept. 10, 2019 -
After scandal, Novartis pledges faster action on data integrity
CEO Vas Narasimhan committed to informing the FDA within five business days of any credible data integrity issues tied to a pending drug application.
By Ned Pagliarulo • Sept. 09, 2019
