Gene Therapy
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Courtesy of Bristol Myers Squibb and Bluebird bio
New CAR-T therapy from Bristol, Bluebird effective but too costly, ICER finds
The influential drug cost watchdog recommended a discount of between 37% and 54% to the $419,500 list price the companies charge for Abecma, a multiple myeloma cell therapy cleared by the FDA in March.
By Ned Pagliarulo • UPDATED: April 6, 2021 at 5:53 p.m. -
Getty / Edited by BioPharma Dive
UniQure says hemophilia gene therapy likely not cause of study volunteer's cancer
The determination, made by UniQure and an independent lab, could ease safety concerns spurred by the patient's diagnosis in December.
By Ned Pagliarulo • March 29, 2021 -
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, reserachers and drugmakers still face major challenges.
By BioPharma Dive staff -
Dollar Photo Club
Drugmakers flock to Vineti's fix for a cell, gene therapy problem
A group of drug companies, including Novartis, Takeda and Roche's Genentech, have agreed to collaborate with Vineti to usher in new identification standards for complex medicines.
By Jacob Bell • March 24, 2021 -
Yujin Kim / BioPharma Dive
With new results, Sarepta's 2nd gene therapy holds steady
Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease.
By Jonathan Gardner • March 19, 2021 -
Getty / Edited by BioPharma Dive
Record funding flowed into cell, gene therapy companies last year
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM.
By Ned Pagliarulo • March 16, 2021 -
Getty / Edited by BioPharma Dive
Solid Bio, eyeing comeback, points to 'totality' of new Duchenne gene therapy results
The data keep Solid in the running in one of gene therapy's most competitive races, but still don't make clear whether the treatment is changing the trajectory of the disease.
By Ben Fidler • March 16, 2021 -
Permission granted by Rocket Pharmaceuticals
How Rocket Pharma quietly became one of gene therapy's high flyers
An unconventional strategy is behind the rise of Rocket, a New Jersey biotech whose executives boldly talk of turning into the "Genentech of gene therapy."
By Sarah de Crescenzo • March 11, 2021 -
Bluebird says gene therapy 'very unlikely' cause of cancer case in trial
The biotech is discussing its findings with U.S. and European regulators in hopes of resuming study of the treatment as well as sales of a related product.
By Ned Pagliarulo • March 10, 2021 -
Permission granted by Biogen
Biogen to spend $200M on gene therapy plant in North Carolina
The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years.
By Kristin Jensen • March 04, 2021 -
Getty / Edited by BioPharma Dive
Doudna-founded biotech Caribou raises fresh funds to fuel gene editing pipeline
The CRISPR biotech raised $115 million in a Series C financing aimed at advancing its engineered cell therapy research.
By Kristin Jensen • March 03, 2021 -
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Bluebird reveals new details on cancer cases in gene therapy trial
Executives shared an update on the biotech's investigation of the two cases at an investor conference, rather than via a statement or regulatory filing.
By Ned Pagliarulo • Feb. 26, 2021 -
Former AveXis executives launch gene therapy startup with uncommon targets
Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.
By Ned Pagliarulo • Feb. 25, 2021 -
Beam, capitalizing on stock surge, buys gene therapy tools developer for $120M
Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body.
By Ned Pagliarulo • Feb. 23, 2021 -
Gates Foundation backs Novartis' search for a sickle cell cure
An initial grant will support a research team at Novartis' R&D labs whose sole focus will be to develop a single-administration gene therapy for sickle cell disease.
By Jacob Bell • Feb. 17, 2021 -
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Bluebird stops studies of sickle cell gene therapy after new cancer cases
The biotech is also suspending sales of its related treatment Zynteglo following a leukemia diagnosis in a clinical trial volunteer and a case of a cancer-like bone marrow disease in another.
By Ben Fidler , Ned Pagliarulo • UPDATED: Feb. 16, 2021 at 10:24 a.m. -
Danielle Ternes/BioPharma Dive
A gene therapy pipeline takes shape for a cluster of rare diseases
Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.
By Ned Pagliarulo • Feb. 12, 2021 -
Getty / Edited by BioPharma Dive
A new startup gets Takeda's backing to take complex genetic medicines 'off the shelf'
Ensoma debuts with a lucrative alliance with the Japanese pharma and an unusual way to deliver genetic medicines into the body.
By Ben Fidler • Feb. 11, 2021 -
Getty / Edited by BioPharma Dive
AbbVie exploring how CRISPR gene editing can improve cell therapies
Using technology from Caribou Biosciences, AbbVie hopes to engineer "off-the-shelf" CAR-T cells that can better withstand attacks from the immune system.
By Jacob Bell • Feb. 10, 2021 -
Courtesy of Editas Medicine
Editas names new CEO in latest executive shakeup
James Mullen, chair of Editas' board and a former Biogen CEO, will replace Cynthia Collins in a critical year for the gene editing biotech.
By Ned Pagliarulo • Feb. 08, 2021 -
Permission granted by Bristol-Myers Squibb
Bristol Myers finally wins FDA approval for cancer cell therapy
Delays tied to factory inspections had pushed an FDA decision on Breyanzi past a key deadline, allowing Bristol Myers to avoid paying billions of dollars in payouts to investors.
By Ned Pagliarulo , Ben Fidler • Feb. 05, 2021 -
Nasdaq
With $588M IPO, Sana leads wave of new biotechs going public
Nine other drug developers are set to price initial public offerings this week, extending a record run of financing activity from last year. Sana's is one of the largest in recent history.
By Ben Fidler • Feb. 03, 2021 -
Neurocine ends partnership with Voyager for Parkinson's gene therapy
The loss of Neurocrine as a collaborator is another blow to Voyager after the FDA placed a clinical hold on the experimental treatment.
By Kristin Jensen • Feb. 03, 2021 -
Danielle Ternes/BioPharma Dive
Gene therapy for hemophilia: So close, yet so far away
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety.
By Ben Fidler • Jan. 28, 2021 -
Bayer AG
How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division
Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers.
By Sarah de Crescenzo • Jan. 21, 2021 -
Courtesy of BioMarin Pharmaceutical
BioMarin tries to get hemophilia gene therapy back on track with positive data
Full one-year results from a Phase 3 study show the therapy, known as Roctavian, sharply reduced bleeds and use of blood-clotting treatments.
By Ned Pagliarulo • Jan. 11, 2021
