The First Crispr Medicine Is Now Approved in the US
54 years, 11 months 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 edited cells. “But we don’t want to waste any time—and patients don’t want to waste any time, because they’ve been waiting for this for a while.” Today, the FDA also approved a second type of gene treatment for sickle cell, called Lyfgenia. Made by Bluebird Bio of Somerville, Massachusetts, it also involves modifying patients’ cells outside the body.

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History of this topic

The First Crispr Treatment Is Making Its Way to Patients
1 month, 1 week 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 therapy trials treat inherited blindness and deafness
7 months, 2 weeks ago

Gene therapy trials treat inherited blindness and deafness

Hindustan Times  

Scientists have restored vision and hearing in people born with congenital blindness or deafness in two separate gene therapy trials A new CRISPR gene editing called EDIT-101 has successfully restored vision in some patients with congenital blindness A British toddler born deaf has become the first person to have their hearing restored via gene therapy. CRISPR gene therapy restores vision …

With CRISPR poised to revolutionise therapy, a pause to consider ethical issues
10 months, 2 weeks ago

With CRISPR poised to revolutionise therapy, a pause to consider ethical issues

The Hindu  

“We will each write a ghost story”, said Lord Byron; and his proposition was acceded to. With the first gene editing technology securing approval for the treatment of sickle cell anemia and beta-thalassemia, we transcend into a new revolutionary phase. Casgevy and Lyfgenia, the two cell-based gene therapies approved by the Food and Drug Administration for sickle cell anemia treatment …

Gene Editing Needs to Be for Everyone
11 months, 2 weeks 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 …

How can gene editing help cure diseases? | In Focus podcast
1 year ago

How can gene editing help cure diseases? | In Focus podcast

The Hindu  

This month, there’s been a lot of excitement in the medical world over the approval given to treatments for diseases that based on genome editing. In 2020, the Nobel Prize for Chemistry was given to two women scientists, Emmanuelle Charpentier and Jennifer Doudna for their discovery of what is essentially a genetic scissors, a tool that allows scientists to cut …

The Age of Crispr Medicine Is Here
54 years, 11 months 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 …

Scientifically Speaking | Nobel Prize-winning gene editing tech offers a ray of hope for blood disorders
1 year ago

Scientifically Speaking | Nobel Prize-winning gene editing tech offers a ray of hope for blood disorders

Hindustan Times  

The United Kingdom and the United States have made a groundbreaking decision to approve gene editing technology known as CRISPR for the treatment of sickle-cell disease and beta-thalassaemia. Sickle-cell disease and beta-thalassaemia are both caused by genetic errors that affect haemoglobin, the molecule in red blood cells responsible for transporting oxygen. In India, according to a review published in the …

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 …

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

Jennifer Doudna Believes Crispr Is for Everyone
54 years, 11 months ago

Jennifer Doudna Believes Crispr Is for Everyone

Wired  

It’s been a monumental year for Crispr, the molecular tool scientists use to edit genetic material. This November, the United Kingdom authorized the first medical treatment using Crispr gene editing, giving people with sickle cell disease new opportunities to receive a one-time therapy to prevent episodes of terrible pain. “It’s expensive,” Jennifer Doudna, the pioneering biochemist who won a Nobel …

The First Crispr Medicine Just Got Approved
54 years, 11 months ago

The First Crispr Medicine Just Got Approved

Wired  

The first medical treatment that uses Crispr gene editing was authorized Thursday by the United Kingdom. The one-time therapy, which will be sold under the brand name Casgevy, is for patients with sickle cell disease and a related blood disorder called beta thalassemia, both of which are inherited. Developed by Vertex Pharmaceuticals of Boston and Crispr Therapeutics of Switzerland, Casgevy …

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 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 …

Can Genetic Modification Technology CRISPR be Applied to Covid-19 diagnostics?
3 years, 4 months ago

Can Genetic Modification Technology CRISPR be Applied to Covid-19 diagnostics?

News 18  

US scientists have repurposed genetic modification technology CRISPR to identify antibodies in patient blood samples in a move that could inspire a new class of medical diagnostics, particularly in the treatment of Covid-19. The technology involves customisable collections of proteins which are attached to a variant of Cas9, the protein at the heart of CRISPR, that will bind to DNA …

Gene-editing treatment shows promise for sickle cell disease
4 years 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 …

Crispr: What is genome editing? All you need to know about Nobel-winning controversial technology
4 years, 2 months ago

Crispr: What is genome editing? All you need to know about Nobel-winning controversial technology

India Today  

Two women -- Emmanuelle Charpentier and Jennifer Doudna -- were awarded the 2020 Nobel Prize for chemistry by the Nobel Committee on Wednesday. The duo received the Nobel Prize in chemistry award for developing a method of genome editing, or gene editing, likened to "molecular scissors" that offers the promise of one day curing inherited diseases and even cancer. A …

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