Gene Therapy
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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 01, 2020 -
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 -
Getty / Edited by BioPharma Dive
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 -
Novartis
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 -
Getty Images
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 -
Novartis
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 -
Jacob Bell
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 -
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 -
Courtesy of Nationwide Children's Hospital
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 at 10:10 a.m. -
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 -
Getty / Edited by BioPharma Dive
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. 09, 2020 -
Courtesy of 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 -
Getty / Edited by BioPharma Dive
Muscle data doesn't help Solid build faith in its gene therapy
A regulatory delay due to an adverse event, meanwhile, has put Solid further behind Sarepta in the pursuit of a Duchenne muscular dystrophy gene therapy.
By Jonathan Gardner • Dec. 18, 2019 -
Getty / Edited by BioPharma Dive
Homology unveils first data for PKU gene therapy, marking early milestone
Data presented Tuesday are from just three patients, but set the pace in a field that includes BioMarin and, most recently, Sangamo Therapeutics.
By Andrew Dunn • Dec. 17, 2019 -
Roche
Roche's $4.8B deal for Spark cleared by FTC after long delay
An unconditional OK for the deal, which was unexpectedly the target of regulatory scrutiny, should ease sector-wide concerns about acquisitions of gene therapy biotechs.
By Ned Pagliarulo • UPDATED: Dec. 17, 2019 at 7:23 p.m. -
Getty / Edited by BioPharma Dive
Value-based pricing for gene therapy? Maybe not ready for hemophilia
A small patient population plus large potential savings could make blood-clotting gene therapies an easier sell to payers, biotech executives say.
By Jonathan Gardner • Dec. 11, 2019 -
UniQure, Pfizer updates hint at gene therapy potential in hemophilia B
The two companies are advancing toward market one-time treatments for the blood disorder, with Takeda about to begin clinical testing of its own therapy.
By Jonathan Gardner • Dec. 10, 2019 -
Novartis
Dive Awards
Drug Launch of the Year: Zolgensma
While clouded by controversy, Novartis' launch of Zolgensma shows gene therapy can be successfully commercialized, even as questions of affordability remain paramount.
By Ned Pagliarulo • Dec. 09, 2019
