Gene Therapy
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Novartis
Gene therapy safetyNovartis reports deaths of two patients treated with Zolgensma gene therapy
The deaths were due to acute liver injury, a known risk of Zolgensma and a concern for gene therapies like it.
By Ned Pagliarulo • Aug. 11, 2022 -
Permission granted by Bristol-Myers Squibb
GentiBio partners with Bristol Myers as cell therapy for immune disease gains momentum
The deal, worth as much as $2 billion, is the latest sign of industry interest in treatments that harness regulatory T cells, an approach behind several recent biotech startups.
By Ben Fidler • Aug. 10, 2022 -
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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 -
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Roche digs into off-the-shelf cell therapy with Poseida deal
The pharma has now signed two alliances with allogeneic drug developers since September, signalling its interest in CAR-T treatment alternatives.
By Ben Fidler • Aug. 3, 2022 -
JuSun via Getty Images
CRISPR gene editingFDA halts testing of Beam’s base editing cancer therapy
The regulator has put on hold human testing of a blood cancer treatment called BEAM-201, one of two the biotech aims to bring into clinical trials this year.
By Ben Fidler • Aug. 1, 2022 -
Courtesy of Sarepta
Sarepta to ask FDA for accelerated approval of Duchenne gene therapy
After discussions with the FDA, the biotech aims to submit an application this fall — sooner than expected and ahead of a Phase 3 study that’s now ongoing.
By Jonathan Gardner • July 29, 2022 -
Permission granted by Editas Medicine
Editas treats first patient in sickle cell trial as FDA lifts partial hold
The sickle cell therapy being studied is the second CRISPR-based medicine that Editas has advanced into clinical testing. Initial trial results could come as soon as the end of this year.
By Ned Pagliarulo • July 28, 2022 -
Courtesy of Bristol Myers Squibb and Bluebird bio
Bristol Myers CAR-T sales rise despite production problems
Demand has increased faster than Bristol Myers can make Abecma, although the company said supply is improving. A manufacturing issue for Breyanzi, meanwhile, impacted sales.
By Ned Pagliarulo • July 27, 2022 -
libre de droit via Getty Images
Emerging biotechEpigenetic editing: a tunable CRISPR alternative
Three startups have recently emerged with plans to edit the epigenome rather than DNA directly. Here’s why that matters and what they aim to do.
By Ben Fidler • July 26, 2022 -
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Sponsored by Bio-RadNext-level quality control in cell and gene therapy
Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before.
July 25, 2022 -
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Freeline gives detailed look at hemophilia gene therapy results
The London-based drugmaker is currently seeking a partner to continue development of the hemophilia B treatment, which has shown promise in testing but trails a rival therapy from CSL Behring and UniQure.
By Ned Pagliarulo • July 21, 2022 -
DKosig via Getty Images
Roche digs deeper into gene therapy for the eye
The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments.
By Jonathan Gardner • July 20, 2022 -
Image courtesy of Ultragenyx.
Ultragenyx buys gene therapy partner after new study results
The California biotech is exercising its option to acquire GeneTx BioTherapeutics for $75 million after seeing data from the companies’ study of an Angelman syndrome treatment.
By Ned Pagliarulo • July 19, 2022 -
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Editas names new chief medical officer in latest executive change-up
Baisong Mei, a veteran of Sanofi and Biogen, comes to Editas half a year after the gene editing company fired his predecessor and one month after new CEO Gilmore O'Neill started on the job.
By Christopher Newman • Updated July 18, 2022 -
Seth Babin/BioPharma Dive
CRISPR gene editingVerve starts first human test of gene editing treatment for heart disease
A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.
By Ned Pagliarulo • July 12, 2022 -
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Sponsored by Ferring PharmaceuticalsC. difficile infection: Current treatment options and challenges
Clostridioides difficile infection is a highly contagious disease that affects 500,000 people in the U.S. and results in nearly 30,000 deaths per year.
By Dr. Teena Chopra • July 11, 2022 -
Permission granted by Catalent
Adverum cuts jobs, restructures to give eye gene therapy another shot
A year after reporting side effects “not seen before in ocular gene therapy” in a clinical trial, the biotech is eliminating 38% of its workforce to save cash while it runs a new study.
By Ben Fidler • July 7, 2022 -
Mario Tama via Getty Images
J&J-backed gene therapy for the eye clears early study test
Positive results for the treatment, which was developed by U.K. biotech MeiraGTx, could rebuild expectations after another gene therapy from Biogen failed in the retinal disease.
By Ned Pagliarulo • June 28, 2022 -
DKosig via Getty Images
Gene therapy safety
Another Astellas gene therapy trial paused by FDA after side effect report
The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage. Both of the pharma’s clinical-stage gene therapies are now on hold.
By Ben Fidler • June 27, 2022 -
Kateryna Kon/Shutterstock.com
Sponsored by Ferring PharmaceuticalsC. difficile infection: A close-up on an urgent public health threat
As hospitals have dealt with surging admissions and extended stays with COVID-19, staff and patients alike have still had to contend with another potentially deadly infection from a bacterium called Clostridioides difficile.
By Dr. Dennis Deruelle • June 27, 2022 -
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BioMarin's hemophilia gene therapy recommended for approval in Europe
The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival.
By Jacob Bell • June 24, 2022 -
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UniQure buoyed by early data for Huntington's gene therapy
After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.
By Jonathan Gardner • June 23, 2022 -
Novartis
Novartis hunting for sickle cell cure with Precision deal
The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.
By Ned Pagliarulo • June 22, 2022 -
Getty / Edited by BioPharma Dive
A biotech startup launches with $38M to develop a cystic fibrosis gene therapy
Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.
By Delilah Alvarado • June 21, 2022 -
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Sponsored by Ferring PharmaceuticalsBreaking down the role of the gut microbiome
When it comes to the human body, we are never truly alone. In fact, we share our bodies with a diverse and complex colony of microorganisms, called the microbiome.
By Dr. Carl Crawford (GI) • June 21, 2022 -
Meletios Verras via Getty Images
CRISPR gene editingVertex, CRISPR strengthen case for pioneering gene-editing treatment
Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.
By Jacob Bell • June 11, 2022
