Gene Therapy: Page 27
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Fresh off being bought, Apceth preps to supply Bluebird's gene therapy
Apceth, now a part of Hitachi Chemical, will serve as the commercial manufacturer in Europe for Bluebird's just-approved gene therapy, Zynteglo.
By Jacob Bell • June 6, 2019 -
Getty / Edited by BioPharma Dive
Bluebird gene therapy approved in Europe in first for severe blood disorder
Zynteglo is intended as a one-time, lifelong treatment for patients with transfusion-dependent beta thalassemia.
By Ned Pagliarulo • June 3, 2019 -
Explore the Trendline➔
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TrendlineGene Therapy
Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges.
By BioPharma Dive staff -
Elizabeth Regan/BioPharma Dive
As ASCO begins, Iovance shows progress with cancer cell therapy
The biotech's therapy, which uses tumor-infiltrating lymphocytes, has spurred responses in some patients with advanced melanoma and cervical cancer.
By Andrew Dunn • May 31, 2019 -
Amicus widens UPenn gene therapy collaboration
The Galafold maker is looking beyond protein engineering as it tries to treat genetically driven metabolic disorders.
By Jonathan Gardner • May 29, 2019 -
Kendall Davis/BioPharma Dive
Iovance to invest $75M in cell therapy plant
A new site in Philadelphia, when complete, will be used to produce Iovance's tumor-infiltrating lymphocyte therapies.
By Kristin Jensen • May 29, 2019 -
BioMarin readies hemophilia gene therapy for FDA submission
But the debate over valrox's durability has not dissipated, as factor expression continued to decline in clinical trial patients.
By Jonathan Gardner • May 28, 2019 -
Novartis gene therapy approved, but will come at cost of more than $2M
Zolgensma is designed to be a cure for spinal muscular atrophy, but its steep price will pose difficulties for a system designed for chronic therapy rather than one-time treatments.
By Ned Pagliarulo • Updated May 24, 2019 -
Novartis looking earlier for deals as biotech valuations stay high
The Swiss pharma, which bought AveXis and its gene therapy Zolgensma last year, expects to spend about $10 billion a year on acquisitions.
By Ned Pagliarulo • May 23, 2019 -
Yujin Kim/BioPharma Dive
Could Novartis' gene therapy have more than one price?
Indication-based pricing may be possible for less severe patients, who would receive a smaller dose. Lower manufacturing costs would lessen the blow to Novartis too.
By Jonathan Gardner • May 23, 2019 -
UniQure snags patents on Huntington's gene therapy as it preps for the clinic
The biotech aims to start a Phase 1/2 study in the second half of 2019 for the experimental gene therapy.
By Andrew Dunn • May 22, 2019 -
Bayer pumping $150M more into biologics
A new facility will focus on large-molecule therapies for oncology, cardiology and additional specialty care areas.
By Jacob Bell • May 13, 2019 -
Nearing gene therapy launch, Bluebird cautions a slower start
Zynteglo, Bluebird's experimental therapy for beta-thalassemia, could soon be cleared in Europe. Reworking payment models to adjust will take time.
By Ned Pagliarulo • May 9, 2019 -
Roche risdiplam data heats up SMA rivalry with Novartis
The oral agent looks like it could give gene therapy Zolgensma a run for its money.
By Jonathan Gardner • May 7, 2019 -
Novartis makes final Zolgensma pitch before FDA decision
The most severe spinal muscular atrophy patients showed sustained benefit, the company said in a data update at a neurology meeting.
By Jonathan Gardner • May 6, 2019 -
Gilead's first move under new chief is to untether Kite
Kite is now its own unit within the big biotech, a move that newly minted CEO Daniel O'Day says will improve cell therapy development.
By Jacob Bell • May 3, 2019 -
Yujin Kim/BioPharma Dive
At gene therapy meeting, insurance execs grapple with expected cost
As more gene therapies move closer to market, questions around pricing and reimbursement have taken on greater urgency for regulators, payers and drugmakers.
By Ned Pagliarulo • April 30, 2019 -
Roche's takeout of Spark not working like a Swiss watch
Antitrust review drags on, forcing the two parties to withdraw and refile paperwork and extend the tender offer by another month.
By Jonathan Gardner • April 26, 2019 -
Medicare proposes raising CAR-T pay, but reimbursement solution years away
As new technologies nip at CAR-T's heels, CMS could take three years to gin up a separate reimbursement category for Novartis' Kymriah and Gilead's Yescarta.
By Jonathan Gardner • April 24, 2019 -
Novartis chief defends cell, gene therapy push
CEO Vas Narasimhan has bet heavily on complex medicines, but the pharma's first quarter earnings show that plan is a work in progress.
By Ned Pagliarulo • April 24, 2019 -
'Bubble boy' gene therapy results shoot Mustang Bio's stock up 200%
Following the clinical success, BioPharma Dive spoke with Mustang's CEO on the manufacturing hurdles still standing in the therapy's way.
By Andrew Dunn • April 18, 2019 -
'This is a cure.' St. Jude's gene therapy succeeds in 'bubble boy' disease study
Treatment has rebuilt the immune systems of 10 boys with the life-threatening condition, allowing them to live normal lives so far.
By Andrew Dunn • April 17, 2019 -
Sarepta may have seen off another rival in Wave
Toxicity observed with Wave's DMD drug raises questions over whether the biotech can find a safe and effective dose.
By Jonathan Gardner • April 17, 2019 -
With FDA decision near, Novartis bolsters SMA gene therapy case
Zolgensma could be approved by the FDA within weeks. Fresh clinical data give further evidence of the therapy's benefit, although reports of two deaths invite questions.
By Ned Pagliarulo • April 16, 2019 -
Catalent to buy Paragon for $1.2B as gene therapy demand grows
The deal follows close on the heels of Thermo Fisher's buy of Brammer Bio, a peer of Paragon in making the viral vectors needed for gene therapy.
By Jacob Bell • April 15, 2019 -
Audentes lays down marker in muscular dystrophy
In a challenge to Sarepta, Solid and Pfizer, a new gene therapy approach aims to deliver a more functional muscle-repairing protein to DMD patients.
By Jonathan Gardner • April 8, 2019
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