Gene Therapy: Page 3
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Deep Dive
‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA
The FDA in June approved a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease, but hasn’t yet proven that it works.
By Ben Fidler , Shaun Lucas • May 7, 2023 -
After delay, Bluebird submits sickle cell gene therapy for FDA approval
Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.
By Christopher Newman • April 24, 2023 -
Explore the Trendline➔
Mario Tama via Getty ImagesTrendlineGene 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 -
French biotech pulls EMA application for eye disease gene therapy
With cash running low and a search for a buyer underway, GenSight wants advice from regulators on the type of data that would support an approval.
By Jonathan Gardner • April 21, 2023 -
Function Oncology raises $28M to use CRISPR to tailor cancer diagnosis and treatment
The startup says its genomics platform can improve on conventional sequencing by uncovering drug target vulnerabilities directly in tumor samples.
By Nick Paul Taylor • April 13, 2023 -
Century Therapeutics changes leadership as CEO steps down
The resignation of top executive Lalo Flores comes three months after a restructuring, marking the latest change of direction for the once high-flying cell therapy startup.
By Delilah Alvarado • April 12, 2023 -
A new biotech wants to ease a bottleneck in cell and gene therapy production
Founded by University of Pennsylvania researchers, VintaBio will manufacture the viral vectors that are essential in cell and gene therapy production, but which have been in shorter supply as more companies entered the space.
By Jacob Bell • April 11, 2023 -
GSK outlines deal to send cell therapies back to Adaptimmune
The pharma, which recently retreated from cell therapy research, will pay Adaptimmune about $37 million as part of an agreement to return rights to two cancer treatments.
By Ben Fidler • April 11, 2023 -
Ginkgo grows its gene therapy offerings with StrideBio deal
The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.
By Jacob Bell • April 5, 2023 -
Freeline cuts more jobs and halts Fabry gene therapy work
The U.K. biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.
By Christopher Newman • April 4, 2023 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
Vertex, CRISPR finish US filing for gene editing drug approval
The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio.
By Christopher Newman • April 3, 2023 -
Sponsored by Advanced Clinical
Changing cell & gene therapy landscape and implications on clinical trials
Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.
By Elizabeth Dugan, Director, Business Development • April 3, 2023 -
Gene therapy startup Vedere to close two years after launch
The startup, a successor to a biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.
By Ben Fidler • Updated April 2, 2023 -
NIAID. (2019). "T Regulatory Cells" [Microscope image]. Retrieved from Flickr.
Regeneron bets on ‘Treg’ cell therapy with Sonoma deal
The biotech will work with Sonoma, a well-funded startup run by prominent immunologist Jeffrey Bluestone, to develop cell-based medicines for ulcerative colitis and Crohn’s disease.
By Ben Fidler • March 28, 2023 -
Gene editing
Vertex pays CRISPR to use its gene editing tech for diabetes drugs
Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.
By Christopher Newman • March 27, 2023 -
Moderna inks second genetic medicine deal in a month
The biotech plans to use Generation Bio’s nonviral delivery technology to get nucleic acid-based therapies to immune cells and into the liver.
By Christopher Newman • March 23, 2023 -
Gilead’s Yescarta extends survival in lymphoma study
The results, the first for a CAR-T therapy, are further evidence supporting the use of the complex therapies earlier in a patient’s disease course.
By Ben Fidler • March 21, 2023 -
Sponsored by MilliporeSigma
Addressing manufacturing challenges in gene therapy development
As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.
By Kate Silver • March 20, 2023 -
Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’
The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.
By Gwendolyn Wu • March 14, 2023 -
BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy
The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.
By Kristin Jensen • March 7, 2023 -
Voyager licenses gene therapy tools to Novartis
The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.
By Ben Fidler • March 6, 2023 -
Gene editing
FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug
The announcement, which follows regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.
By Ben Fidler • March 2, 2023 -
Amid industry turbulence, Chroma raises fresh funding to edit the epigenome
As many drug startups struggle to gain cash, Chroma has raised another $135 million to back its research, a new twist on genetic medicine.
By Gwendolyn Wu • Updated March 1, 2023 -
Sickle cell pipeline narrows as gene therapy developers rethink research plans
Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.
By Ned Pagliarulo • Feb. 23, 2023 -
Medicaid, with planned payment pilot, girds for influx of pricey gene therapies
The proposed model could help state Medicaid agencies explore different kinds of outcomes-based payment schemes, but may come too late to prepare for the first sickle cell gene therapy.
By Ned Pagliarulo • Feb. 21, 2023 -
Talaris restructures, ending effort to develop a cell therapy for kidney transplants
Following a patient death and difficulty enrolling study participants, the company is laying off a third of its staff and ending a pair of studies for a two-decade-old program once owned by Novartis.
By Ben Fidler • Feb. 16, 2023