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gene therapy

Latest Headlines

Latest Headlines

Pfizer buys into gene therapy's renaissance and bets on Spark Therapeutics

Pfizer is the latest pharma heavyweight to get behind the promise of gene therapy, as the New York drugmaker has laid out plans to set up a dedicated R&D operation and signed a deal with startup Spark Therapeutics to kick-start the effort.

Spark snags a 'breakthrough' tag for its Phase III gene therapy

Gene therapy startup Spark Therapeutics has picked up the FDA's coveted breakthrough-therapy designation for its lead candidate, a one-time treatment that promises a permanent solution some rare eye diseases.

Astellas dives into gene therapy with a Harvard expert in tow

Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease.

Gene therapy technique could treat rare lung disease

Using a new type of cell transplantation, researchers at Cincinnati Children's Hospital Medical Center corrected a lung disease in mice that mimics a similar rare disease in humans.

Biogen shoulders into gene therapy amid a second honeymoon for the field

As researchers and investors toast renewed optimism for a new generation of gene therapies, biotech bellwether Biogen Idec is deepening its commitment to the space, another sign of life for the resurgent field.

With $1M gene therapies coming, research pioneer and CVS plot new payment route

New technologies have inspired a wave of biotech startups developing gene therapies. Now, one of the field's leading lights is worried about sticker shock for the treatments working their way down the pipeline.

Think a $150K cancer drug is pricey? Try a $1M-plus gene therapy

Over the last few years, new technologies have inspired a wave of biotech startups developing gene therapies. Now, one of the field's leading lights is worried about sticker shock for the treatments working their way down the pipeline. No wonder: They're likely to cost $1 million-plus. So, how about a new payment model for these therapies?

After a string of gene therapy licensing deals, ReGenX says it's time to shine

ReGenX Biosciences is one of the most intriguing gene therapy biotechs you never heard of. And now the executive team in charge intends to start changing that.

Gene editing method kills cervical cancer cells

Using a gene-editing method, investigators were able to delete certain genes in the human papillomavirus, prompting cancer-causing cells to self-destruct. The antiviral technique could be replicated to target other DNA-based viruses like hepatitis B and herpes simplex, researchers say.

UniQure bets on a gene therapy for heart failure

After making international headlines with the West's first approval for a gene therapy, pioneering biotech uniQure is working to flesh out its pipeline of one-time treatments, buying into an early-stage shot at heart failure that could put its technology on the biggest stage yet.