Gene Therapy
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Permission granted by Editas Medicine
Gene editingTrailing rivals, Editas shares first study data for sickle cell treatment
Editas said its gene editing therapy appeared safe and was well tolerated in two patients. The data comes five months after U.S. regulators lifted a partial hold on the trial.
By Kristin Jensen • Dec. 7, 2022 -
Danielle Ternes/BioPharma Dive
Gene therapy approvals bring validation as field closes year on high
It’s been a busy fall in gene therapy, with FDA nods for new treatments from CSL and Bluebird as well as a few deals. Catch up on our best stories here.
By Ned Pagliarulo • Dec. 6, 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 -
Seth Babin/BioPharma Dive
Gene therapy safetyVerve shares slide after company reveals details on FDA trial hold
In a letter to the biotech, the regulator asked for more information on its gene editing medicine for heart disease as well as on its potential risks.
By Delilah Alvarado • Dec. 6, 2022 -
JuSun via Getty Images
Gene editingFDA lifts pause on Beam plans to test base editing cancer therapy
The FDA’s decision is a step forward for a type of drugmaking known as “multiplex editing,” which involves editing several genes simultaneously.
By Ben Fidler • Dec. 2, 2022 -
iStock / Getty Images Plus via Getty Images
Gene editingGene editing startup iECURE returns to investors for fresh funding
The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data.
By Gwendolyn Wu • Nov. 30, 2022 -
Meletios Verras via Getty Images
AstraZeneca reveals its cell therapy ambitions with deal for startup
The British drugmaker will pay as much as $320 million to acquire Neogene Therapeutics, a developer of cell-based treatments for solid tumors — its first significant investment in the field.
By Ben Fidler • Nov. 29, 2022 -
Courtesy of Sarepta
FDA grants speedy review to Sarepta’s Duchenne gene therapy
The agency will decide on an accelerated approval of Sarepta’s treatment by May 29, months before results are expected from a potentially confirmatory Phase 3 trial.
By Jonathan Gardner • Nov. 28, 2022 -
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Sponsored by Cardinal HealthNavigate the complexities of cell and gene therapies
See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product.
Nov. 28, 2022 -
Permission granted by ICON
Sponsored by ICONMainstreaming cell and gene therapy – Realizing its potential
The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.
Nov. 28, 2022 -
ismagilov via Getty Images
FDA approves first gene therapy for hemophilia B
The one-time treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million per infusion.
By Ned Pagliarulo • Updated Nov. 23, 2022 -
Permission granted by Editas Medicine
Gene editing
Editas to seek partner for CRISPR medicine after lackluster study results
While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish.
By Ned Pagliarulo • Nov. 17, 2022 -
Permission granted by Ionis Pharmaceuticals and Metagenomi
Gene editingIonis teams with Metagenomi and dives into gene editing
The RNA drug developer is paying the richly funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.
By Ben Fidler • Nov. 14, 2022 -
Sarah Silbiger via Getty Images
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 -
Seth Babin/BioPharma Dive
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 -
Jonathan Gardner / BioPharma Dive
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 -
Getty / Edited by BioPharma Dive
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 -
Spencer Platt via Getty Images
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 -
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 -
Leonid Sorokin via Getty Images
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 -
JuSun via Getty Images
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 -
Permission granted by Carl June
Emerging biotechCAR-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
