Gene Therapy
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Dr_Microbe via Getty Images
Using twin viruses, startup AAVantgarde aims to extend gene therapy’s reach
The biotech has raised about $65 million to test two ways to deliver larger genes into the body, which could help gene therapy treat more diseases.
By Ben Fidler • June 6, 2023 -
fpm via Getty Images
Sponsored by Bio-RadOvercoming common pitfalls in lentiviral cell and gene therapies
See how ddPCR technology can take your lentiviral cell and gene therapies to the next level.
By Marwan Alsarraj • May 30, 2023 -
Explore the Trendline➔
Mario Tama 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 -
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PTC lays off staff, fires CFO as neuromuscular drug study fails
The biotech is giving up on early-stage gene therapy research as part of a restructuring meant to cut costs and redirect resources toward therapies most likely to succeed.
By Kristin Jensen • May 24, 2023 -
Courtesy of Sarepta
FDA delays decision on Duchenne gene therapy, considers narrower approval
The agency will extend its review of Sarepta’s treatment by one month as it weighs limiting an initial OK to children with Duchenne aged 4 to 5 years old.
By Ben Fidler • Updated May 24, 2023 -
Sarah Silbiger/Getty Images via Getty Images
FDA approves Krystal gene therapy for rare wound disorder
Cleared to treat dystrophic epidermolysis bullosa, Vyjuvek is the sixth gene therapy for an inherited disease to gain clearance in the U.S.
By Delilah Alvarado • May 22, 2023 -
Novartis
Novartis buys rare disease gene therapy from Avrobio
The pharma will pay nearly $90 million to acquire Avrobio’s treatment for cystinosis, an inherited condition caused by the toxic buildup of an amino acid.
By Ned Pagliarulo • May 22, 2023 -
Maximusnd via Getty Images
Mustang pares research, sells manufacturing plant to save money
The biotech joins a score of other cell and gene therapy developers that, on the heels of a historic market downturn, have cut costs.
By Jacob Bell • May 18, 2023 -
Dr_Microbe via Getty Images
Emerging biotech
Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”
By Ned Pagliarulo • May 17, 2023 -
Lydia Polimeni, National Institutes of Health
Gene therapy consortium targets eight rare diseases for clinical trials
Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.
By Christopher Newman • Updated May 16, 2023 -
Courtesy of UniQure
UniQure sells royalty rights to hemophilia gene therapy
The Dutch biotech sold a portion of royalties owed by CSL Behring on sales of Hemgenix, adding to a cash pile it has built since out-licensing the treatment.
By Christopher Newman • May 15, 2023 -
Courtesy of Sarepta
FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy
Shares in Sarepta rose by 25% Monday following a close advisory committee vote supporting accelerated approval of the biotech’s treatment, despite uncertainty around its benefit.
By Ned Pagliarulo , Ben Fidler • Updated May 12, 2023 -
Courtesy of Sarepta
FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show
Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.
By Ben Fidler • Updated May 10, 2023 -
Permission granted by Susan and Chris Finazzo
Deep Dive‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA
FDA advisers voted 8-6 in support of the agency approving a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease.
By Ben Fidler , Shaun Lucas • May 7, 2023 -
Permission granted by Bluebird bio
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 -
Ilya/Stock.adobe.com
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 -
libre de droit via Getty Images
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 -
Copyright © 2022 Miltenyi Biotec B.V. & Co. KG. All rights reserved.
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 -
Dr_Microbe via Getty Images
Emerging biotechA 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 -
Warren Little via Getty Images
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 -
Courtesy of Ginkgo Bioworks
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 -
Dr Microbe via Getty Images
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.
Gene editingVertex, 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 -
BillionPhotos via Adobe Stock
Sponsored by Advanced ClinicalChanging 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 -
Ilya/Stock.adobe.com
Emerging biotechGene 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
