$3 million prize goes to duo whose research led to first sickle cell CRISPR therapy

Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a ...

Woman with three deadly diseases has ‘remarkable’ recovery after cell therapy

Treatment reset wayward immune system of patient with life-threatening conditions, say scientists, in a world first ...

Protective 'switch' in most common blood cancer opens new doors for diagnosis and treatment

A new study published in The Journal of Immunology by researchers at the Josep Carreras Leukaemia Research Institute (IJC) ...

Gene-edited stem cells help five blood disorder patients stop transfusions in clinical trial

Stem cell transplantation could be a rapid and effective way to restore hemoglobin production in individuals with the blood ...
Smithsonian Magazine

In a First, This Personalized Cell Therapy Treated Three Life-Threatening Autoimmune Diseases in One Patient

The patient was bedridden and needed daily blood transfusions, and she had tried nine different therapies that didn't bring ...
New Scientist on MSN

CAR T-cell therapy takes woman from bedridden to 'perfectly fine'

A woman with three different autoimmune conditions had all of them treated simultaneously by genetically modifying her immune ...
WebMD

Kresladi: FDA Approves First One-Time Gene Therapy for Children With Severe Leukocyte Adhesion Deficiency Type I

What Is Kresladi, and Why Does It Matter? Kresladi (marnetegragene autotemcel) is a gene therapy approved by the FDA to treat a rare and serious immune condition called leukocyte adhesion deficiency ...
MPR

Etavopivat Reduces VOCs and Boosts Hemoglobin in Sickle Cell Disease

Topline data for the once-daily PKR activator etavopivat demonstrate a 27% reduction in vaso-occlusive crises and a robust hematologic response in patients with SCD.

FDA opens door to wider use of testosterone therapy for low libido

The FDA may allow broader access to testosterone therapy for men with low libido without known medical cause. Manufacturers ...

Novo’s once-daily sickle cell pill hits milestone. A smaller rival is sinking 24%.

Novo Nordisk says it plans to apply for regulatory approval of its sickle cell disease, etavopivat, in the second half of ...
The Manila Times

Novo Nordisk: Etavopivat is the first in a new class of drugs to meet both co-primary endpoints in the phase 3 HIBISCUS trial, substantially reducing vaso-occlusive crisis ...

Etavopivat showed a 27% reduction in vaso-occlusive crisis events and ~4-month delay to first vaso-occlusive crisis event on ...
Nature

The surprising science behind red-light therapy — and how it really works

In 2021, dermatologist David Ozog was on holiday with his family in the Bahamas, when his 18-year-old son had a massive stroke. The teenager was airlifted to Florida, and then to Chicago for surgery.