Gene Therapy: Page 5
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Lexeo gene therapy shows signs of heart benefit in small study
The biotech, which went public late last year, argued the results justify exploring an accelerated approval. But shares fell by double digits amid investor skepticism.
By Ben Fidler • July 15, 2024 -
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UniQure stock surges on data for Huntington’s gene therapy
The biotech’s stock rose more than 60% as fresh data appeared to alleviate some investor concerns about the therapy’s effectiveness and reliability.
By Jacob Bell • July 9, 2024 -
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Trendline
Gene 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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Immune resetCartesian data add to progress, questions for CAR-T in autoimmune disease
The company claimed Phase 2 study results prove the therapy’s promise treating myasthenia gravis. But shares lost more than a quarter of their value amid questions about the data.
By Ben Fidler • July 2, 2024 -
Courtesy of UniQure
UniQure, with sale of plant, outsources Hemgenix manufacturing
The gene therapy developer, which cut jobs in October, expects the sale of a Lexington, Massachusetts, facility to Genezen to lower its cash burn.
By Ned Pagliarulo • July 1, 2024 -
Marcin Klapczynski via Getty Images
With revised IPO pitch, Artiva touts ‘natural killer’ cell therapy for autoimmune disease
Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy to ride a wave of investor interest in autoimmune disease research.
By Ben Fidler • July 1, 2024 -
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Sponsored by Bio-RadAre you meeting safety standards for residual host cell DNA?
Ensure the safety of gene therapies with accurate HEK293 DNA measurement using Vericheck ddPCR kits.
July 1, 2024 -
Sarah Silbiger via Getty Images
Rocket gene therapy rejected by FDA over manufacturing
The complete response letter follows other recent manufacturing-related rejections, including one for a cell therapy from Abeona Therapeutics.
By Ned Pagliarulo • June 28, 2024 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
First-of-its-kind Intellia data suggest CRISPR drug could be given more than once
The findings provide early proof that multiple doses of a gene editing medicine can be safely administered with additive effects, overcoming a key constraint of the complex therapies.
By Ben Fidler • June 26, 2024 -
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Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine
The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.
By Gwendolyn Wu • June 25, 2024 -
Permission granted by Accredo
Sponsored by Accredo by EvernorthThe role of a specialty pharmacy in enhancing patient access to rare and gene therapies
Four critical components in which the right specialty pharmacy partner can bring rare and gene therapy manufacturers the scale, expertise and capabilities needed to drive enhanced patient access.
June 24, 2024 -
Courtesy of Sarepta
Sarepta Duchenne gene therapy wins broader use from FDA
The approval makes Elevidys available to most Duchenne patients at least 4 years of age, despite mixed trial results that have led to skepticism about its effectiveness.
By Ben Fidler • Updated June 21, 2024 -
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Syncona melds two gene therapy biotechs for better shot at new nervous system treatments
The new company, Spur Therapeutics, is getting another $50 million from Syncona to support its broadened pipeline, which targets Parkinson’s disease as well as heart conditions.
By Jacob Bell • June 17, 2024 -
Jose Luis Calvo Martin, Jose Enrique Garcia-Maurino Muzquiz via Getty Images
Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision
The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.
By Ben Fidler • June 13, 2024 -
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Can robots break the cell therapy bottleneck?
A partnership between cell therapy delivery specialist Portal Biotechnologies and precision robotics maker Multiply Labs could address manufacturing hurdles, the CEOs said.
By Michael Gibney • May 29, 2024 -
Ermath, Michael. (2020). "Individualized Therapies Workshop" [Photograph]. Retrieved from Flickr.
With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals
But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.
By Ben Fidler • May 24, 2024 -
Courtesy of Millipore Sigma via Business Wire
Merck KGaA to buy gene therapy tools maker for $600M
The German drugmaker is bolstering its MilliporeSigma business with a unit of Gamma Biosciences that specializes in so-called transfection reagents.
By Kristin Jensen • May 23, 2024 -
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Regeneron gene therapy improves hearing in two children
The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.
By Kristin Jensen • Updated May 8, 2024 -
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Patient dies in Pfizer study of Duchenne gene therapy
Pfizer said the patient, a young boy who was treated earlier last year, died suddenly from cardiac arrest. The company is working with trial researchers to investigate further.
By Ben Fidler • Updated May 8, 2024 -
Permission granted by Bluebird bio
Bluebird, Vertex prep for first commercial use of sickle cell gene therapies
One patient has started the treatment process for Bluebird's Lyfgenia, while five others have done the same for Vertex's Casgevy.
By Ned Pagliarulo • Updated May 7, 2024 -
Christopher Furlong via Getty Images
AstraZeneca ups stake in Cellectis in latest cell therapy bet
The British drugmaker, which has made genetic medicine a larger priority of late, added $140 million to a deal to develop up to 10 gene and cell therapies.
By Jonathan Gardner • May 6, 2024 -
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Moderna ends gene editing alliance with Metagenomi
Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in gene editing research in recent years.
By Kristin Jensen • May 2, 2024 -
Permission granted by Astellas
Astellas adds to ‘off-the-shelf’ cell therapy capabilities with Poseida deal
The partnership follows Astellas’ investment in the biotech last year and adds to its portfolio of donor-derived cellular medicines.
By Ben Fidler • May 1, 2024 -
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Prime gets FDA green light to begin first trial test of ‘prime editing’
The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.
By Ned Pagliarulo • April 29, 2024 -
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Pfizer hemophilia gene therapy arrives in US to uncertain future
The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition struggle to gain traction.
By Ben Fidler • April 26, 2024 -
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BioMarin drops drug programs in pipeline cull
The cuts of four pipeline prospects come as BioMarin resets its priorities following the slow launch of its hemophilia gene therapy Roctavian.
By Kristin Jensen • April 25, 2024
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