The World’s First Crispr Drug Gets a Slow Start
2 weeks, 4 days ago

The World’s First Crispr Drug Gets a Slow Start

Wired  

Deshawn “DJ” Chow waited a year to receive a treatment that could change his life. It’s the first approved medicine to use the Nobel Prize–winning technology known as Crispr, a type of gene editing. Another genetic treatment for sickle cell, Lyfgenia, won approval last December, and the first patient was treated in September. Chow is City of Hope’s first sickle cell patient, while a beta thalassemia patient has been treated at Children’s National. “The process of getting this drug is very different from just taking a pill,” says Leo Wang, Chow’s hematologist-oncologist at City of Hope.

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History of this topic

The First Crispr Treatment Is Making Its Way to Patients
1 month, 3 weeks ago

The First Crispr Treatment Is Making Its Way to Patients

Wired  

Nearly a year after its approval, the first medical treatment that uses the Nobel Prize–winning technology Crispr is now being given to patients. Called Casgevy, the gene-editing treatment is for people with sickle cell disease and a related blood disorder called beta thalassemia. “Cagevy has been enthusiastically received by patients, physicians, and policymakers, and the launch is gathering momentum across …

Gene Editing Needs to Be for Everyone
1 year ago

Gene Editing Needs to Be for Everyone

Wired  

At the end of 2023, we witnessed an important moment in the history of medicine: For the first time, the US Food and Drug Administration approved a therapy that uses Crispr gene editing. This new therapy was developed by Crispr Therapeutics and Vertex Pharmaceuticals to treat sickle cell disease, an ailment caused by a single-letter mutation in the genetic code …

The Age of Crispr Medicine Is Here
55 years ago

The Age of Crispr Medicine Is Here

Wired  

Jimi Olaghere used to end up in the emergency room so often that the hospital reserved a bed for him. “It was a circus, bouncing from specialist to specialist and constantly desecrating my body with endless amounts of prescription pills, all in the hopes of finding a sliver of what it feels like to be alive,” Olaghere told an advisory …

Game-changer: The Hindu Editorial on approval for gene therapy to treat sickle cell disease and beta thalassemia
1 year ago

Game-changer: The Hindu Editorial on approval for gene therapy to treat sickle cell disease and beta thalassemia

The Hindu  

Less than a month after the U.K. drug regulator approved Casgevy, the gene therapy to treat people above 12 with sickle cell disease and beta thalassemia, the U.S. FDA has approved two gene therapies — Casgevy and Lyfgenia — to treat sickle cell disease in patients over 12. By disabling the BCL11A gene, foetal haemoglobin that is produced, which does …

The First Crispr Medicine Is Now Approved in the US
55 years ago

The First Crispr Medicine Is Now Approved in the US

Wired  

“This is a terrible disease,” says Samarth Kulkarni, president and CEO of Crispr Therapeutics. Casgevy uses the Nobel Prize–winning technology Crispr to modify patients’ cells so that they produce healthy hemoglobin instead. Of the 31 patients followed for two years, 29 have been free of pain crises for at least a year after receiving a single dose of their own …

FDA approves 2 gene therapies for sickle cell. One is the first to use the editing tool CRISPR
1 year, 1 month ago

FDA approves 2 gene therapies for sickle cell. One is the first to use the editing tool CRISPR

Associated Press  

Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” the FDA’s Dr. Nicole Verdun said in a statement announcing the approvals. “We are excited to …

Gene therapy offers new hope for sickle cell disease cure
1 year, 1 month ago

Gene therapy offers new hope for sickle cell disease cure

LA Times  

Jalen Matthews of Louisville, Ky., who was diagnosed with sickle cell disease at birth, said she plans to ask her doctor about the new gene therapy options. Regulators on Friday approved two new gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. “Sickle cell disease …

UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia
1 year, 1 month ago

UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia

The Independent  

For free real time breaking news alerts sent straight to your inbox sign up to our breaking news emails Sign up to our free breaking news emails Sign up to our free breaking news emails SIGN UP I would like to be emailed about offers, events and updates from The Independent. Read our privacy policy Britain's medicines regulator has authorized …

A new cure for sickle cell disease may be coming. Health advisers will review it next week
1 year, 2 months ago

A new cure for sickle cell disease may be coming. Health advisers will review it next week

Associated Press  

The only cure for painful sickle cell disease today is a bone marrow transplant. The agency is expected to decide on the treatment in early December, before taking up a different sickle cell gene therapy later that month. Dr. Allison King, who cares for children and young adults with sickle cell disease, said she’s enthusiastic about the possibility of new …

A Gene Therapy Cure for Sickle Cell Is on the Horizon
1 year, 9 months ago

A Gene Therapy Cure for Sickle Cell Is on the Horizon

Wired  

Evie Junior’s life has been defined by pain. He was born with sickle cell disease, which causes red blood cells to be sticky and C-shaped, not smooth and round. People with sickle cell are often fatigued because their red blood cells die fast, cutting off oxygen to the body. Junior, who's now 30 years old, is one of dozens of …

Gene-editing treatment shows promise for sickle cell disease
4 years, 1 month ago

Gene-editing treatment shows promise for sickle cell disease

Associated Press  

Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide, especially Black people. Doctors hope the one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, may treat and possibly cure sickle cell disease and beta thalassemia. In sickle cell, defective hemoglobin leads …

Doctors In The U.S. Use CRISPR Technique To Treat A Genetic Disorder For The 1st Time
5 years, 5 months ago

Doctors In The U.S. Use CRISPR Technique To Treat A Genetic Disorder For The 1st Time

NPR  

Doctors In The U.S. Use CRISPR Technique To Treat A Genetic Disorder For The 1st Time For the first time, doctors have used the gene-editing technique CRISPR to treat a genetic disorder in the U.S. STEIN: Yeah, so Victoria has sickle cell disease, and it's a terrible genetic disease that primarily affects African Americans in the United States. And instead …

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder
5 years, 5 months ago

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

NPR  

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder Enlarge this image toggle caption Meredith Rizzo/NPR Meredith Rizzo/NPR Updated at 4:05 p.m. Enlarge this image toggle caption Meredith Rizzo/NPR Meredith Rizzo/NPR "Sometimes it feels like lightning strikes in my chest and real sharp pains all over," Gray says. "It's a gene that tells the …

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