‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease
Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS. View on euronews
Sickle cell disease ‘cure’ to be offered to NHS patients
Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem cell transplant
NHS England to offer Casgevy for sickle cell disease patients
Charity organisation Sickle Cell Society has described the recommendation as a “major breakthrough” for sickle cell patients.
Vertex wins reimbursement deal for CRISPR-partnered sickle cell therapy in England
Vertex Pharmaceuticals (VRTX) stock gains as England opts to reimburse its CRSPR (CRSP)-partnered gene therapy Casgevy to treat sickle cell disease. Read more here.
Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY ® (exagamglogene autotemcel).
Vertex, U.K. reach reimbursement deal for CRISPR-based medicine for sickle cell disease
People in England with sickle cell will be able to get the world’s first CRISPR-based medicine under an agreement between the U.K. and Vertex.
After inversion by CRISPR/Cas, the epigenetic state of plant chromosomes remains stable
The epigenetic state of chromatin, gene activity, and chromosomal positions are interrelated. A research team from the IPK ...
MIT Technology Review3d
Mice with two dads have been created using CRISPR
It’s a new way to create “bi-paternal” mice that can survive to adulthood—but human applications are still a long way off.
STAT4h
Pharmalittle: We’re reading about FDA OK for Vertex non-opioid pain drug, vaccine advisory committee, and more
Vertex Pharmaceuticals won approval from the U.S. Food and Drug Administration for a non-opioid pain drug, clearing the way for the rollout of a product that has simultaneously sparked high hopes and ...
News Medical on MSN5h
Researchers create random versions of human genomes to study disease
The most complex engineering of human cell lines ever has been achieved by scientists, revealing that our genomes are more resilient to significant structural changes than was previously thought.
MIT Technology Review2d
The Download: mice with two dads, and Meta’s fact-checking challenges
Once a week, Sun Kai has a video call with his mother, and they discuss his day-to-day life. But Sun’s mother died five years ...
STAT8d
RFK Jr., an investor in a CRISPR biotech, has raised alarms about the technology
RFK Jr.'s comments about the gene-editing technology raise a question about how he would regulate it as head of HHS.
£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...
Crispr Therapeutics price target lowered to $30 from $45 at Morgan Stanley
Morgan Stanley lowered the firm’s price target on Crispr Therapeutics (CRSP) to $30 from $45 and keeps an Underweight rating on the shares. The ...
Complex engineering of human cell lines reveals genome's unexpected resilience to structural changes
The most complex engineering of human cell lines ever has been achieved by scientists, revealing that our genomes are more ...