Gene Therapy: Page 24
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Novartis gene therapy Zolgensma cleared for use in Europe
The Swiss pharma estimates 550 to 600 infants are born in Europe each year with spinal muscular atrophy, the hereditary disease Zolgensma treats.
By Kristin Jensen • May 20, 2020 -
Thermo Fisher to invest $180M in new gene therapy plant
The planned facility, to be built in Massachusetts, will rival in size a gene therapy plant in Texas owned by CDMO competitor Lonza.
By Kristin Jensen • May 13, 2020 -
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 -
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Dyno spins out of Harvard's laboratories with an aim to make gene therapy better
The startup announced collaborations with Novartis and Sarepta, both leading gene therapy makers, that could be worth $2 billion in total.
By Jonathan Gardner • May 11, 2020 -
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Orchard lays off a quarter of its staff amid major shift
The gene therapy developer has given up on plans to build a new manufacturing facility and is adjusting its clinical resources to focus on more common diseases.
By Kristin Jensen • Updated May 8, 2020 -
Texas university spins out Taysha, a gene therapy startup with 15 programs
The university uses a gene therapy delivery tool that Taysha says can be scaled for both small and large manufacturing needs.
By Jonathan Gardner • April 29, 2020 -
Permission granted by Merck KGaA
Merck KGaA to spend $110M on new gene therapy facility in California
The investment continues a spate of build-outs by Merck KGaA, which, like its contracting peers, sees an opportunity in the fast-growing gene therapy field.
By Kristin Jensen • April 23, 2020 -
Ultragenyx's gene therapy technology finds another interested party
Daiichi Sankyo has agreed to pay $225 million to access a manufacturing platform that Ultragenyx acquired through its buy of Dimension Therapeutics.
By Jacob Bell • April 1, 2020 -
Permission granted by Novartis
No sanctions for Novartis as FDA ends review of gene therapy violations
Quietly completing an inspection review, the FDA indicated it will not penalize Novartis for submitting altered data in its approval application for Zolgensma.
By Ned Pagliarulo • March 30, 2020 -
Permission granted by University of Pennsylvania
Deep DiveBig pharma shied away from gene therapy for years. Academia picked up the slack
The byproduct was richer licensing fees for universities and a throng of startups, but also questions on fair prices and research priorities.
By Jonathan Gardner • March 17, 2020 -
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Sangamo sees vindication in Biogen gene-editing deal
Delivering products nearly ready for clinical testing has sparked greater dealmaking interest from big pharma, Sangamo's CEO told BioPharma Dive.
By Jonathan Gardner • Feb. 28, 2020 -
BioMarin inches closer to a gene therapy first
A decision by the FDA on whether to approve valrox, potentially the first gene therapy for hemophilia, should come by late August.
By Jacob Bell • Feb. 21, 2020 -
With supply tight, Novartis readies gene therapy plant for production
Bringing the facility in Durham, North Carolina, online is a critical step for the Swiss pharma's plans to expand the market for Zolgensma, its gene therapy for spinal muscular atrophy.
By Ned Pagliarulo • Feb. 21, 2020 -
Audentes to spend $110M on new gene therapy plant in North Carolina
The biotech, which was bought last year by Astellas, joins Pfizer, Bluebird bio and AveXis in choosing to locate gene therapy manufacturing in the state.
By Ned Pagliarulo • Feb. 18, 2020 -
Decibel CEO steps down as company pares workforce
The Third Rock-backed company put an acting executive in place as it repositions for development in gene therapy and regenerative medicine.
By Jonathan Gardner • Jan. 29, 2020 -
Zolgensma sales grow, signaling gene therapy momentum
Another 100 infants received Novartis' gene therapy in the fourth quarter, supported by broad insurance coverage and more newborn screening.
By Ned Pagliarulo • Jan. 29, 2020 -
Pfizer lays out gene therapy aspirations
With more Phase 3 trials on the near horizon, the big pharma plans to add one new gene therapy project per year to its pipeline.
By Jonathan Gardner • Jan. 28, 2020 -
FDA, expecting a gene therapy boom, firms up policies
In seven guidance documents, the agency gave drugmakers guidelines on manufacturing, testing and long-term follow-up for the one-time treatments.
By Ned Pagliarulo • Jan. 28, 2020 -
Danielle Ternes/BioPharma Dive
Orchard isn't for the picking, Biohaven's Allergan headache, and who's on Vascepa?
Amarin CEO John Thero had one question for investors eager to hear about his company's plans for its heart pill, while Orchard made a case for independence.
By Jacob Bell • Jan. 16, 2020 -
Permission granted by Novartis
Q&A
Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public
While Zolgensma's launch has gone well, the pharma still faces challenges in expanding the gene therapy's market and addressing criticism over its price.
By Andrew Dunn • Jan. 15, 2020 -
Nationwide Children's, a gene therapy leader, launches manufacturing spinout
Andelyn Biosciences, named after two children treated with gene therapy at Nationwide, will operate as a for-profit subsidiary of the Ohio-based hospital.
By Ned Pagliarulo • Updated Jan. 15, 2020 -
Danielle Ternes/BioPharma Dive
Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones
On the first day of the JPM conference, Alexion mapped out ambitious plans in neuroscience, while Rubius did its best to explain delays in testing its first drug.
By Jacob Bell , Andrew Dunn • Jan. 14, 2020 -
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BioMarin sets gene therapy sights beyond hemophilia
At the JPM conference, the biotech announced a manufacturing lift and a new trial start that affirm its ambitions in gene therapy go beyond its closely watched hemophilia A treatment.
By Jacob Bell • Jan. 13, 2020 -
Ultragenyx boosted by trial data for 2nd gene therapy
One analyst called the results, which sparked a 30% jump in Ultragenyx's stock price, better than expected. The biotech is now preparing a Phase 3 trial.
By Jonathan Gardner • Jan. 10, 2020 -
Getty / Edited by BioPharma Dive
Solid cuts one-third of staff, goes all in on delayed gene therapy
Re-starting a halted muscular dystrophy trial is Solid's priority as it puts a second drug and an assistive device on the backburner.
By Jonathan Gardner • Jan. 9, 2020 -
Permission granted by Roche
Roche hands $1B to Sarepta in major return to gene therapy deals
The Swiss pharma acquired a muscular dystrophy treatment for markets outside the U.S. in a pact that could reach a value of $10 billion.
By Jonathan Gardner • Dec. 23, 2019
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