Gene Therapy: Page 5
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First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer
Adaptimmune’s Tecelra is the first TCR cell therapy to reach market and, at $727,000, is also the priciest cellular medicine for cancer in the U.S.
By Ben Fidler • Aug. 2, 2024 -
Courtesy of UniQure
UniQure restructuring to claim 300 jobs
The job cuts affect 65% of UniQure’s workforce and include the gene therapy developer’s recent sale of a manufacturing plant to Genezen.
By Delilah Alvarado • Aug. 1, 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 University of Pennsylvania
Jim Wilson, prominent gene therapy researcher, to depart UPenn
Wilson, who founded UPenn’s gene therapy program three decades ago, will step down to start two new spinouts, Gemma Bio and Franklin Biolabs.
By Ned Pagliarulo • Aug. 1, 2024 -
Permission granted by AIRNA
Biotech startup Airna raises $60M for RNA editing medicines
Launched last September, the company is working on a treatment for alpha-1 antitrypsin deficiency, which it said could enter the clinic in 2025.
By Gwendolyn Wu • July 31, 2024 -
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Pfizer quits Duchenne gene therapy, lays off staff following study setback
The company is letting go of 150 staffers alongside a decision to officially terminate the high-profile program, which was acquired in 2016.
By Ben Fidler • July 30, 2024 -
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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 -
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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 -
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 -
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Immune resetArtiva 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
Vertex 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 -
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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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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 -
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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 -
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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 -
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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
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