Clinical Research

‘Supercharged’ T-cells show promise against Hodgkin’s lymphoma

Cancer has been winning the arms race against the immune system for too long, but scientists are developing plenty of ...
woman cancer patient

Rural-urban divide: Groundbreaking gene therapies could exacerbate inequality in cancer care

Two new cancer treatments have shown miraculous cures, but if you happen to live in Arkansas or Montana, or a ...
gene therapy

Report: Price for blindness gene therapy Luxturna is ‘four times too high’ at $850,000

The $850,000 list price for a new medicine that treats a genetic form of childhood blindness is about four times too high ...
Screen Shot at AM

Can gene therapy reduce terminal cancers to minor chronic diseases that are ‘no different than high blood pressure’?

On Oct. 15 at 8 a.m., Andy Lindsay stood atop 21,247-foot Mera Peak in Nepal, a wildly improbable place for ...
gene therapy

CAR-T gene therapy treatments may fall short of corrective goals, requiring surgical gene editing

For decades, researchers, some physicians, and a few patients have had visions of treatments that would go in and fix ...
red ribbon

Could we fight HIV with new Car-T therapy?

The same kind of DNA tinkering that produced the first FDA-approved gene therapy for cancer has shown hints of suppressing and even ...
eye x

Luxturna gene therapy approved for vision loss

The first two novel gene therapies for cancer treatment passed through FDA approval earlier this year, first B-cell acute lymphoblastic leukemia (ALL) followed quickly ...
gene therapy

Should patients—not the FDA— have final say on risky gene therapy treatments?

Working with mice, researchers have used gene therapy to restore sight to the blind, reprogram the body’s own T cells ...
breakthrough

Looking back at 2017’s genetics breakthroughs

It was a big year for the building blocks of life. Here were the most significant breakthroughs in genetics research ...
gene editing

Should it matter if the public is wary of gene editing and human enhancement?

A survey of people in the US and 10 European nations found opposition to some forms of gene editing for ...
GEN

FDA approves first gene therapy for a genetic disease—onetime cure for rare blindness

The first gene therapy to restore sight to individuals who suffer from a rare inherited genetic blindness was approved by ...
marsili family pain

Italian family’s inability to feel pain spurs research on relief for chronic sufferers

The six members of the family barely notice broken bones or severe burns. Researchers have identified the mutation behind their ...
hemophilia

Hemophilia A gene therapy trial results: ‘This is huge’

British doctors say they have achieved "mind-blowing" results in an attempt to rid people of haemophilia A. Patients are born ...
obesity kids

Obesity epidemic: Could gene therapy be an answer?

Scientists are working with treatments that have shown success in amping up the metabolism of mice, helping them lose weight ...
M Cancer chemotherapy SPL

Cancer treatment without chemotherapy? CAR T-cells could become the new standard

Although rare, it is not unheard-of for new treatments to achieve substantial early successes in one or two patients only ...
Screen Shot at PM

2017: The year gene therapy became a ‘clinical reality’

This year, gene therapy finally became a clinical reality. The U.S. Food and Drug Administration approved two personalized treatments that ...
odin crispr bacterial kit

Scientists sound alarm over DIY gene therapy, calling it ‘unsafe’

In just the past few months we’ve seen not one, but two examples of people pursuing unregulated gene therapy. … [T]he largest organization of ...
sickle cell

Gene therapy, CRISPR could provide treatments for sickle-cell, but cures far off

More than 50 years after the cause of sickle-cell disease was discovered, a dozen treatments for the painful and life-shortening inherited ...
stem

Transplanting stem cells directly into brain could alter treatment of nervous system disorders

A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach ...
gene therapy

Gene therapy challenge: How much should it cost and how do we pay for it?

When evaluating the high prices of one-time gene therapy treatments, should we look to the costs of organ transplants when ...
allergies

Fighting infectious diseases with immunotherapy on ‘cusp of commercialization’

Immunotherapy, which involves adapting immune cells to destroy specific cellular targets, has made a name for itself treating cancer. But ...
Hemophilia A

Gene therapy could be ‘ideal cure’ for hemophilia B

[L]ast year, [Jay] Konduros enrolled in a clinical trial, receiving an experimental gene therapy at Children's Hospital of Philadelphia [to ...
HE AA GENES J

DIY gene therapy? FDA is not a fan

A few biohackers (fans of do-it-yourself (DIY) science experiments) recently designed a gene therapy for HIV. Not only that, they posted a video ...
healthcare costs x

Exorbitant costs of gene therapy raise payment concerns for patients and government

[Editor's note: Executives from several companies developing gene therapies gathered for a panel discussion at the recent Forbes Healthcare Summit, ...
O colors

Genetic engineering, synthetic biology poised to boost photosynthesis and carbon capture

In a feature article published in the open access journal eLife, an international team of experts led by Dr Bonnie Wintle ...
A F A F A D E C F

Can we solve the patent issues that threaten CRISPR research?

[Editor's note: Lawrence Horn is President and CEO of MPEG LA, which provides licenses for standards and other technology platforms.] Although recent ...
MEDEX TBVietnam CROP original original

Insecticide resistance threatens to derail malaria control in Africa

The largest genetic study of mosquitoes has found their ability to resist insecticides is evolving rapidly and spreading across Africa, ...
dl belgium

Why life-saving gene therapy isn’t available yet to children who need it most

[The first patient to permanently edit his DNA,] Brian Madeux, 44, of Arizona, is part of a clinical trial testing ...