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In a decision, the FDA greenlit two new drugs for the treatment of sickle cell disease in patients 12 and older, one of which —Vertex’s drug Casgevy — is the first approved use of genome editing technology CRISPR in the US. Bluebird Bio’s Lyfgenia also is a cell-based gene therapy, however, it uses a different gene modification technique to deliver tweaked stem cells to the patient.Both approvals cultivate new pathways for the treatment of , which is an inherited blood disorder that is characterized by red blood cells…

Casgevy uses the Nobel Prize-winning technology Crispr to modify patients’ cells so that they produce healthy hemoglobin instead. The Crispr system has two parts: a protein that cuts genetic material and a guide molecule that tells it where in the genome to make the cut.To do this, a patient’s stem cells are taken out of their bone marrow and edited in a laboratory. Scientists make a single cut in a different gene, called BCL11A, to turn on the production of a fetal form of hemoglobin that typically shuts off shortly…

Blood sample tube with blood for Sickle Cell Screen -Test. Sickle cell blood test, abnormal hemoglobin test.Md Babul Hosen | Istock | Getty ImagesThe U.S. Food and Drug Administration on Friday approved the country's first gene-editing treatment, Casgevy, for use in patients with sickle cell disease.The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the…

Last week, in a global milestone for medicine, U.K. regulators approved the first-ever CRISPR gene-editing drug. The treatment, from Vertex and CRISPR Therapeutics, will be licensed under the brand name Casgevy in the U.K. for patients with sickle cell disease (SCD) and beta thalassemia. The regulatory green light abroad isn’t surprising given the therapy’s established record of clinical trial success in treating these painful, and sometimes fatal, inherited blood disorders. But it marks a historic turning point for…

Curated By: Shahrukh ShahLast Updated: November 22, 2023, 18:19 ISTOdysse Electric Motorbike Vader. (Photo: Odysse Electric)Thw brand has confirmed that Vader has received ICAT certification, and will hit the Indian road in December 2023.The fastest-growing electric two-wheeler maker Odyssey has finally launched the much-awaited electric bike Vader. The vehicle had already made its official appearance earlier this year. However, due to some certification issue, it never came out in the market.Now, the brand has confirmed…

The UK has become the first country in the world to approve CRISPR gene-editing therapy. The landmark biotech decision involves the treatment of two specific blood dieseases, but also opens the door for the use of the technology in treating many other genetic disorders.  Regulators approved the use of CRISPR for the treatment of inherited diseases sickle-cell anaemia and β-thalassaemia on Thursday. The former affects the shape of red blood cells of 20 million people worldwide and can cause…

In sickle cell disease, abnormal hemoglobin makes a person’s blood cells hard and crescent-shaped. These misshapen cells clump together and block blood flow to organs, causing bouts of extreme pain. The cells then die off early, leaving a lack of healthy red blood cells, or anemia.Beta thalassemia also causes anemia because the body makes less hemoglobin than normal.People with life-threatening beta thalassemia need blood transfusions every three to five weeks and other medications throughout their lifetime.“Both sickle…

Last Updated: July 19, 2023, 15:39 ISTChandigarh Metro (Photo: IANS)Punjab, Haryana, and Chandigarh Collaborate to Expand Tricity Metro Network to 77 Kilometers.In a significant development, the governments of Punjab and Haryana, in conjunction with representatives from the Chandigarh administration, came together on Tuesday for a meeting that resulted in the revision and approval of an expanded Metro network in the Tricity.The initial plan of 66 kilometers was revised to a more extensive stretch of approximately 77…

Early clinical trials of norgestrel, the active ingredient in Opill, have shown that the pills can be up to 98 percent effective at preventing pregnancy, but only when the drug is taken exactly as indicated and used along with a back-up form of birth control. In real life, effectiveness is usually lower.Doctors in the US more commonly prescribe birth control pills that contain both progestin and a synthetic form of estrogen. These combination pills thicken the mucus of the cervix while also preventing the ovaries from…

On July 13, 2023, the U.S. Food and Drug Administration approved a drugmaker's application for the first daily over-the-counter birth control pill for people seeking to prevent pregnancy.The pill, called Opill – the brand name for the tablet formulation of norgestrel – is an oral contraceptive containing only progestin hormone, which helps prevent pregnancy by thickening cervical mucus, preventing ovulation or both.Opill was initially approved by the FDA for prescription use in 1973. Its approval for nonprescription use…