Gene Therapy
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Pfizer says hemophilia gene therapy meets late-stage study goal
While the study results were positive, questions remain about the longer-term potential of hemophilia treatments like Pfizer’s.
By Kristin Jensen • July 24, 2024 -
Permission granted by Bluebird bio
US government declines to clear Bluebird fertility support for Zynteglo patients
The Health and Human Services' inspector general issued a "negative opinion" on Bluebird's request one week after Vertex sued the federal government over access to fertility preservation services.
By Ned Pagliarulo • July 22, 2024 -
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 -
Permission granted by InterVenn Biosciences
Sponsored by InterVenn BiosciencesBreakthroughs in immunotherapy: glycoproteins as predictive biomarkers for therapeutic response
Serum-based glycoproteins demonstrate success in predicting immune checkpoint inhibitor therapy outcomes.
July 22, 2024 -
Marcin Klapczynski via Getty Images
Emerging biotechArtiva prices $167M IPO, riding optimism for autoimmune cell therapy
The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.
By Ben Fidler • July 18, 2024 -
Courtesy of Vertex Pharmaceuticals
Gene editingVertex taps Orum to hunt for better ‘preconditioning’ drugs
The deal gives Vertex rights to use Orum’s ADC-like technology to discover gentler preparatory regimens for patients receiving intensive treatments like Casgevy.
By Gwendolyn Wu • July 16, 2024 -
Courtesy of Vertex Pharmaceuticals
Vertex sues US over limits on providing fertility services to Casgevy patients
In the suit, Vertex argues HHS’ stance forces a “Hobson’s choice” on people with sickle cell seeking to undergo the potentially curative treatment.
By Ned Pagliarulo • Updated July 16, 2024 -
iStock via Getty Images
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 -
Popartic via Getty Images
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 -
Meletios Verras via Getty Images
Cartesian 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 -
luismmolina via Getty Images
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 -
Olena_T via Getty Images
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 -
Maximusnd via Getty Images
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 -
Getty Images via Getty Images
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 -
libre de droit via Getty Images
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 -
David Dee Delgado via Getty Images
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
