Gene Therapy: Page 13
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Permission granted by Kevin Trimmer/Ascidian Therapeutics
Ascidian starts up with $50M and a twist on RNA editing
The startup, run by Translate Bio cofounder Romesh Subramanian, believes its RNA “exon editing” approach could have long-lasting effects without the risks that come with editing DNA.
By Gwendolyn Wu • Oct. 12, 2022 -
Peddalanka Ramesh Babu via Getty Images
Allogene starts first pivotal trials of an ‘off-the-shelf’ cell therapy for cancer
The biotech believes the two Phase 2 trials initiated Thursday can support approval applications for what could be the first allogeneic cancer cell therapy.
By Ben Fidler • Oct. 7, 2022 -
Explore the Trendline➔
iStock via Getty Images
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 -
ismagilov via Getty Images
BioMarin resubmits its hemophilia gene therapy to the FDA
The resubmission has been long awaited after BioMarin’s original application was unexpectedly rejected by the FDA two years ago. The company expects a decision around the middle of next year.
By Delilah Alvarado • Sept. 30, 2022 -
Courtesy of Sarepta
Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy
The application comes a year earlier than previously had been expected, as company says drug reviewers are open to accelerated review based on data from early-stage trials.
By Jonathan Gardner • Sept. 29, 2022 -
Courtesy of Vertex Pharmaceuticals
Vertex given green light to seek US approval of CRISPR-based therapy
The company and its development partner, CRISPR Therapeutics, will begin submitting a rolling application in November. The blood disease treatment is the first of its kind to near an FDA review.
By Jacob Bell • Sept. 27, 2022 -
ismagilov via Getty Images
Pfizer, Sangamo set to resume gene therapy study after safety delay
Concerns over blood clotting risk had derailed testing of the hemophilia treatment last year, leading the companies to adjust their trial protocol.
By Delilah Alvarado • Sept. 23, 2022 -
Permission granted by Bluebird bio
Back-to-back gene therapy approvals give Bluebird shot at survival
The FDA’s clearances of Zynteglo and Skysona are a boost to Bluebird, and could help lift the research field after a series of setbacks. But selling the high-priced therapies will be a challenge.
By Ned Pagliarulo • Sept. 19, 2022 -
Courtesy of Bluebird bio
Bluebird wins FDA approval of gene therapy for rare brain disorder
The therapy, called Skysona and cleared to treat cerebral adrenoleukodystrophy, is the product of more than a decade of work by Bluebird. It will cost $3 million per patient.
By Ned Pagliarulo • Updated Sept. 17, 2022 -
Danielle Ternes/BioPharma Dive
BioMarin reports cancer case in hemophilia gene therapy trial
The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S. Drug regulators have not ordered a trial hold, however.
By Jonathan Gardner • Sept. 13, 2022 -
Permission granted by Stig Albansson / Pretzel Therapeutics
Mitochondrial drugs, with a twist: Pretzel Therapeutics launches with $72.5M in funding
Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.
By Gwendolyn Wu • Sept. 12, 2022 -
Christoph Burgstedt via Getty Images
Beam-linked startup launches with an eye on the next generation of RNA drugs
Orbital co-founder and former Alnylam CEO John Maraganore said the startup aims to “overcome some of the shortcomings that have been there with the first generation of RNA companies.”
By Gwendolyn Wu • Sept. 7, 2022 -
Dr Microbe via Getty Images
Sangamo presses ahead with Fabry disease gene therapy
Preliminary results from a Phase 1 study show Sangamo’s treatment to be safe and suggest it is working as intended, leading the biotech to move into the trial’s next phase.
By Ned Pagliarulo • Sept. 1, 2022 -
Sarah Silbiger via Getty Images
Beam details reasons for FDA hold on base editing cancer therapy
The regulator has asked Beam for a range of technical data before it can start testing the blood cancer treatment in humans.
By Kristin Jensen • Aug. 31, 2022 -
Courtesy of BioMarin Pharmaceutical
With European approval secured, BioMarin puts roughly $1.5M price tag on hemophilia gene therapy
On a conference call, BioMarin executives revealed the anticipated price for Roctavian and the company's initial launch plans, which include pay-for-performance deals customized to different markets.
By Jacob Bell • Aug. 25, 2022 -
Dr_Microbe via Getty Images
ElevateBio finds a new manufacturing home and long-term gene therapy partner in Pittsburgh
The biotech signed a 30-year deal with the University of Pittsburgh to establish a gene and cell therapy manufacturing hub that’s being built with a $100 million grant.
By Kristin Jensen • Aug. 25, 2022 -
iLexx via Getty Images
Ovid turns to gene therapy startup to restock drug pipeline
The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.
By Ben Fidler • Aug. 24, 2022 -
Permission granted by Bluebird bio
With $2.8M gene therapy, Bluebird sets new bar for US drug pricing
Approved for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments. Its market launch is likely to be watched carefully by other gene therapy developers.
By Ned Pagliarulo • Aug. 18, 2022 -
Sarah Silbiger via Getty Images
Bluebird gene therapy approved by FDA for rare blood disease
The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a cost of $2.8 million in the U.S.
By Ned Pagliarulo • Updated Aug. 17, 2022 -
Gene therapy safety
Novartis reports deaths of two patients treated with Zolgensma gene therapy
The deaths were due to acute liver injury, a known risk of Zolgensma and a concern for gene therapies like it.
By Ned Pagliarulo • Aug. 11, 2022 -
Permission granted by Bristol-Myers Squibb
GentiBio partners with Bristol Myers as cell therapy for immune disease gains momentum
The deal, worth as much as $2 billion, is the latest sign of industry interest in treatments that harness regulatory T cells, an approach behind several recent biotech startups.
By Ben Fidler • Aug. 10, 2022 -
Meletios Verras via Getty Images
Roche digs into off-the-shelf cell therapy with Poseida deal
The pharma has now signed two alliances with allogeneic drug developers since September, signalling its interest in CAR-T treatment alternatives.
By Ben Fidler • Aug. 3, 2022 -
JuSun via Getty Images
FDA halts testing of Beam’s base editing cancer therapy
The regulator has put on hold human testing of a blood cancer treatment called BEAM-201, one of two the biotech aims to bring into clinical trials this year.
By Ben Fidler • Aug. 1, 2022 -
Courtesy of Sarepta
Sarepta to ask FDA for accelerated approval of Duchenne gene therapy
After discussions with the FDA, the biotech aims to submit an application this fall — sooner than expected and ahead of a Phase 3 study that’s now ongoing.
By Jonathan Gardner • July 29, 2022 -
Permission granted by Editas Medicine
Editas treats first patient in sickle cell trial as FDA lifts partial hold
The sickle cell therapy being studied is the second CRISPR-based medicine that Editas has advanced into clinical testing. Initial trial results could come as soon as the end of this year.
By Ned Pagliarulo • July 28, 2022 -
Courtesy of Bristol Myers Squibb
Bristol Myers CAR-T sales rise despite production problems
Demand has increased faster than Bristol Myers can make Abecma, although the company said supply is improving. A manufacturing issue for Breyanzi, meanwhile, impacted sales.
By Ned Pagliarulo • July 27, 2022
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