sicklecellanemianews6d
SCD gene-editing therapy Casgevy now being offered at CHLA
With the addition of Casgevy, Children’s Hospital Los Angeles now offers two gene therapies for sickle cell, the first being Lyfgenia.
sicklecellanemianews1d
SCD patients can access Casgevy via England’s public health system
Vertex has entered a reimbursement agreement with NHS England enabling eligible SCD patients to access Casgevy in the public ...
STAT6d
The CRISPR companies are not OK
Gene editing promised a revolution, but biotech layoffs, stock slumps, and industry struggles reveal a stark reality: CRISPR is hard to do profitably.
pharmaphorum4d
FDA gives go-ahead for CRISPR-based sickle cell disease trial
Development of a stem cell therapy for sickle cell disease from Vertex and development partner CRISPR therapeutics can go ahead, after the FDA lifted a hold on a review. The companies had applied ...
Sickle cell disease is a genetic disorder that causes lifelong suffering – here’s what you need to know
Accumulation of clots causes some of the most severe symptoms of sickle cell disease, including strokes, kidney failure, ...
The Punch Newspapers5d
Understanding gallbladder issues in sickle cell patients
Gallbladder problems are a common complication in individuals with sickle cell disease. These issues often arise due to the breakdown of red blood cells and the subsequent formation of gallstones ...
The EMBO Journal2d
A CRISPR-Cas9 screen reveals genetic determinants of the cellular response to decitabine
A temporally resolved CRISPR-Cas9 screen reveals DAC-induced DNA damage drives trans-cell cycle cytotoxicity that depends on ...
pharmaphorum1d
Pfizer pulls sickle cell med Oxbryta off market after deaths
Pfizer is voluntarily withdrawing its sickle cell disease (SCD) therapy Oxbryta from all world markets after finding an "imbalance" in deaths in its clinical trials programme. It's a blow to the ...