Gene Therapy: Page 2

Intellia cleared to start key CRISPR drug study in UK

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

By Ned Pagliarulo • Oct. 19, 2020

Roche, aiming to make better gene therapies, turns to an emerging startup

The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology.

By Ben Fidler • Oct. 14, 2020

Explore the Trendline➔

Trendline

Gene 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

FDA puts Voyager study on hold in latest gene therapy speed bump

The biotech won't be able to start human testing of an experimental Huntington's disease treatment until regulators see more manufacturing data.

By Jonathan Gardner • Oct. 13, 2020

Pfizer and Sangamo, after rival's setback, kick off late-stage gene therapy trial

The FDA's recent rejection of a hemophilia gene therapy from BioMarin sets a high standard for approval. Will Pfizer fare any better?

By Jonathan Gardner • Oct. 07, 2020

Solid gets all-clear from FDA to restart gene therapy trial

Worrisome immune responses had led the FDA to halt Solid's study. Now, the agency will permit Solid to continue after the biotech made adjustments to its manufacturing and patient enrollment procedures.

By Jonathan Gardner • Oct. 01, 2020

Sponsored by Biomere

Preclinical done right: The importance of using relevant animal models in gene therapy studies

Gene therapy is no longer an approach for the future. It's a technique used now.

Sponsored by Emergent Biosolutions

Gene therapy solution: The value of a CDMO as your end-to-end partner

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment.

New data bolsters Sarepta gene therapies for two deadly muscle diseases

The results disclosed at a medical meeting on Monday show that all patients tested have seen their motor skills stabilize or improve when history suggests they wouldn't.

By Ben Fidler • Sept. 28, 2020

Gene therapy company Taysha completes sprint from first funding to IPO

Led by alumni of Zolgensma developer AveXis, the university spinout could have four experimental therapies in the clinic by the end of 2021.

By Jonathan Gardner • UPDATED: Sept. 24, 2020 at 4:23 p.m.

Takeda adds to cell therapy push with new manufacturing site

The Japanese drugmaker has made cell therapy a focus, advancing three experimental cancer treatments into clinical testing with plans for two more.

By Ned Pagliarulo • Sept. 15, 2020

FDA wants more details on Sarepta gene therapy, potentially delaying final test

A new request from the regulator could tighten the race between Sarepta and Pfizer to prove out a gene therapy for Duchenne muscular dystrophy. 

By Ben Fidler • Sept. 10, 2020

With latest data, a gene therapy for a deadly brain disease inches closer to market

The results keep Bluebird Bio on track to seek approval of the experimental treatment, known as eli-cel, in Europe later this year and in the U.S. in 2021. 

By Jacob Bell • Aug. 29, 2020

BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds

A draft report from the drug pricing watchdog concluded that, under the right conditions, Roctavian may be cost-effective at a price of $2.5 million.

By Jacob Bell • Aug. 28, 2020

Deep Dive

A cure for hemophilia seemed closer than ever. For many patients, it's now further out of reach

The surprise rejection of BioMarin's hemophilia A gene therapy delayed a decades-long mission to fix the rare bleeding disorder.

By Jacob Bell • UPDATED: Aug. 19, 2020 at 12:05 p.m.

Editas, AbbVie rework gene editing deal as pioneering CRISPR trial resumes

Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership.

By Ben Fidler • Aug. 07, 2020

Sponsored by Parexel Biotech

Cell and gene therapies: For biotechs, collaboration is key to successful innovation

With the right partner, biotech companies can fully embrace cell and gene therapy opportunities.

By Matthew M. Cooney & Roberta L. Steere • July 30, 2020

Biogen to study use of key SMA drug after Novartis gene therapy

A planned trial is meant to test whether Spinraza can further help infants and young children who have already received Zolgensma, which, in theory, is meant to be curative if given very early on.

By Ned Pagliarulo • July 21, 2020

New data from MeiraGTx help bolster J&J's gene therapy bet

The results are the first from a slate of gene therapies J&J licensed from Meira last year, a deal that remains the pharma's largest investment in the field.

By Ben Fidler • July 17, 2020

Spark, chasing BioMarin, plans 2021 start for key hemophilia gene therapy study

The Roche subsidiary said it will begin Phase 3 dosing its hemophilia A gene therapy next year, potentially putting it well behind a competing treatment from BioMarin.

By Jonathan Gardner • July 13, 2020

Third patient dies in halted study of Audentes gene therapy

The three patients, treated for a rare neuromuscular disease, were given a high dose of Audentes' gene therapy.

By Ben Fidler • UPDATED: Aug. 21, 2020 at 10:47 a.m.

In a surprise, UniQure sells its hemophilia gene therapy for $450M

The licensing deal with CSL Behring is one of the largest for a gene therapy, but the partnership could make a buyout of the Dutch firm less likely. 

By Ben Fidler • June 25, 2020

UniQure gets out the gate first in race for Huntington's gene therapy

The biotech has dosed patients in the first trial of a gene-targeted treatment for the degenerative disease, but competitor Voyager could be chasing soon.

By Jonathan Gardner • June 19, 2020

New CRISPR, gene therapy results strengthen potential for treatment of blood diseases

CRISPR Therapeutics and Vertex can more convincingly say their CRISPR-based medicine is helping three people with beta thalassemia or sickle cell disease, while Bluebird takes another step forward.

By Ned Pagliarulo • June 12, 2020

Regeneron expands Intellia gene editing deal as its post-Sanofi future emerges

Regeneron and Intellia will grow their alliance and work on hemophilia, an already competitive space that includes several experimental gene therapies. 

By Jonathan Gardner • June 01, 2020

With $11B stock sale, Sanofi loosens ties with Regeneron

The French pharma will get a cash windfall by selling most of its 20% stake in Regeneron, spurring speculation as to how it'll spend the money. 

By Jonathan Gardner • UPDATED: May 27, 2020 at 7 p.m.