Gene Therapy: Page 13
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String of gene therapy deals spurs cautious optimism on Wall Street
To some analysts, the recent acquisitions of Akouos and AGTC, as well as Astellas’ equity investment in Taysha, are a positive signal for an area of drug research that’s been “in investors' penalty box for much of 2022.”
By Jacob Bell • Nov. 1, 2022 -
Jacob Bell/BioPharma Dive
BioMarin to face FDA advisers before approval decision on hemophilia gene therapy
Though the panel represents a new regulatory hurdle for Roctavian, BioMarin’s research head said the company has been preparing for such a meeting “over the last few months.”
By Jacob Bell • Oct. 27, 2022 -
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 -
Courtesy of GSK
GSK, dialing back cell therapy work, ends deals with Lyell and Adaptimmune
The British drugmaker terminated the two alliances following disappointing results this year for a once highly touted cell-based treatment for solid tumors.
By Ben Fidler • Updated Oct. 26, 2022 -
Courtesy of Astellas
Astellas takes stake in gene therapy developer Taysha
Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.
By Delilah Alvarado • Oct. 24, 2022 -
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AGTC yields to biotech downturn with gene therapy buyout deal
The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.
By Ben Fidler • Oct. 24, 2022 -
Permission granted by Aldevron
Sponsored by AldevronSimplify IP for your gene therapy with OTS backbones, plasmids and enzymes
Read how IP-simplified products can transform your speed to market without needing to reassess operations midway through to commercialization.
Oct. 24, 2022 -
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Prime Medicine raises $175M in one of the year’s largest biotech IPOs
Shares in the gene editing startup, one of only 19 biotechs to debut on Wall Street this year, have fallen 10% since trading began last Thursday.
By Gwendolyn Wu • Oct. 20, 2022 -
Courtesy of Eli Lilly
Lilly bets more than $600M on a gene therapy developer targeting hearing loss
Like with Prevail Therapeutics, Lilly’s first gene therapy acquisition, the pharma’s planned buyout of Akouos includes a contingent value right that could hike the deal’s overall cost.
By Jacob Bell • Oct. 18, 2022 -
Permission granted by Carl June
CAR-T pioneer Carl June on founding startups and cell therapy’s next act
The University of Pennsylvania immunologist and inventor of Kymriah spoke with BioPharma Dive about working with pharma, starting companies and the future of the cell therapy field.
By Ben Fidler • Oct. 18, 2022 -
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State of Play‘In vivo’ cell therapy: expanding beyond CAR-T
At least five startups have emerged with new ways to genetically modify immune cells within the body, an approach that, if successful, could widen the field of CAR-T treatment.
By Ben Fidler • Oct. 18, 2022 -
Ermath, Michael. (2020). "Individualized Therapies Workshop" [Photograph]. Retrieved from Flickr.
‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy
Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference.
By Ned Pagliarulo • Oct. 13, 2022 -
Jacob Bell/BioPharma Dive
BioMarin gets FDA decision date for hemophilia gene therapy, but questions on review remain
After issuing a surprise rejection of the therapy in 2020, the FDA has now agreed to again review BioMarin’s Roctavian therapy. An approval decision is expected by March 31.
By Kristin Jensen • Oct. 13, 2022 -
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 -
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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 -
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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 -
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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 -
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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
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