Gene Therapy: Page 2
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BioMarin signals lengthier FDA review for hemophilia gene therapy
The agency wants to see forthcoming study results for the biotech's treatment, which could potentially push out an approval decision by three months.
By Ned Pagliarulo • Nov. 8, 2022 -
Gene editing
FDA halts Verve plans to test gene editing therapy for heart disease in US
Verve didn’t say what led to the FDA’s decision, but claimed it hasn’t observed any safety issues in an ongoing trial in New Zealand and the U.K, where enrollment will continue.
By Ben Fidler • Nov. 7, 2022 -
Explore the Trendline➔
Spencer Platt via Getty ImagesTrendlineThe BioPharma Dive Outlook on 2023
Against a stormy backdrop, 2022 did bring bright spots in Alzheimer’s, obesity, breast cancer and rare disease. Dig in to BioPharma Dive’s 2023 outlook for what’s in store this year.
By BioPharma Dive staff -
Bispecific cancer drugs and gene therapy advances: What to watch at this year’s ASH meeting
Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test.
By Ben Fidler , Ned Pagliarulo • Nov. 4, 2022 -
Abeona to submit cell therapy for approval after positive results for skin disorder treatment
The company said its therapy, which could become the first drug for a type of epidermolysis bullosa, helped heal wounds and reduce pain in a clinical trial.
By Jacob Bell • Nov. 3, 2022 -
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 -
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 -
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 -
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 -
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 -
Sponsored by Aldevron
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Oct. 24, 2022 -
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 -
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 -
Emerging biotech
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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