US regulators approve two gene therapies for sickle cell disease
Blood disorder that can lead to premature death affects estimated 100,000 people in the United States, most of whom are BlackThe US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients.The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics. Both therapies are made from the patients ’ own blood stem cells and were approved for people aged 12...
Source: Guardian Unlimited Science - December 8, 2023 Category: Science Authors: Edward Helmore and agencies Tags: US news Health World news Society Medicine Gene editing Science Source Type: news
FDA Approves First CRISPR Treatment in U.S.
It was only 11 years ago that scientists Jennifer Doudna and Emmanuelle Charpentier first described a new way to edit genes, called CRISPR, in a scientific paper. The discovery is so game-changing that the pair earned the Nobel Prize in Chemistry in 2020 for how it could transform the way genetic diseases are treated. Now, on Dec. 8, the U.S. Food and Drug Administration (FDA) approved the very first treatment in the country based on the technology.
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In the medical world, that’s lightning speed. “It’s incredible,” says Doudna, professor of chemistry a...
Source: TIME: Health - December 8, 2023 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized healthscienceclimate Source Type: news
FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia
FRIDAY, Dec. 8, 2023 -- The U.S. Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.
Casgevy, developed by Vertex... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - December 8, 2023 Category: General Medicine Source Type: news
FDA Approves Casgevy (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease
– First-ever approval of a CRISPR-based gene-editing therapy in the U.S. – Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 8, 2023 Category: Drugs & Pharmacology Source Type: news
First-ever approval of a CRISPR-based gene-editing therapy in the U.S., Casgevy (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Dec. 8, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 8, 2023 Category: Drugs & Pharmacology Source Type: news
FDA Approves Casgevy (exagamglogene autotemcel) CRISPR/Cas9 Genome-Edited Cell Therapy for the Treatment of Sickle Cell Disease
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Dec. 8, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 8, 2023 Category: Drugs & Pharmacology Source Type: news
FDA approves first gene therapies to treat patients with sickle cell disease
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. (Source: World Pharma News)
Source: World Pharma News - December 8, 2023 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news
FDA Approves First Crispr Treatment That Might Cure Sickle Cell
The Food and Drug Administration approved a new gene editing therapy for sickle cell disease, its first green light for a Crispr treatment. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 8, 2023 Category: Pharmaceuticals Authors: Robert Hart, Forbes Staff Tags: Business /business Innovation /innovation Healthcare /healthcare Science /science Breaking breaking-news topline Source Type: news
FDA approves two sickle cell therapies, including first CRISPR medicine
The therapies offer hope for a long-overlooked genetic illness that can cause excruciating pain and cut decades off people’s lives. (Source: Washington Post: To Your Health)
Source: Washington Post: To Your Health - December 8, 2023 Category: Consumer Health News Authors: Carolyn Y. Johnson Source Type: news
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CNN's Meg Tirrell sits down with Johnny Lubin, one of the first in the world to try a new kind of medicine that uses a gene-editing tool called CRISPR to offer a potential cure for sickle cell disease. (Source: CNN.com - Health)
Source: CNN.com - Health - December 8, 2023 Category: Consumer Health News Source Type: news
Equitable treatment options for pediatric patients with sickle cell disease
Gene therapy, once approved for sickle cell disease, could offer a lifelong cure without the risk associated with allotransplantation. (Source: Yale Science and Health News)
Source: Yale Science and Health News - December 8, 2023 Category: Universities & Medical Training Source Type: news
New sickle cell treatment sparks feelings of hope and some hesitancy among Black patients
The Food and Drug Administration must decide by Friday whether to approve a new gene-editing therapy to treat sickle cell disease, a debilitating blood disorder that affects at least 100,000 Americans, most of whom are Black. The treatment, called exa-cel, would be the first approved medicine in…#crisprtherapeutics #derekrobertson #ceceliacalhoun #smilowcancerhospital #connecticut #kaylasmithowens #chesapeakebeach #maryland #bowiestateuniversity #smithowens (Source: Reuters: Health)
Source: Reuters: Health - December 8, 2023 Category: Consumer Health News Source Type: news
Milestone Gene Therapies for Sickle Cell Disease Greenlit by FDA
(MedPage Today) -- The FDA approved two cell-based gene therapies, exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia), for sickle cell disease (SCD), the agency announced.
Marking the first FDA-approved therapy utilizing... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - December 8, 2023 Category: American Health Source Type: news
U.S. poised to approve first gene-editing treatment in breakthrough for sickle cell patients
At age 19, Joe Tsogbe underwent his first hip replacement. In his 20s, he averaged about nine hospitalizations a year. By his 30s, that rose to more than a dozen. All the result of sickle cell disease, an inherited blood disorder where a genetic mutation causes normally full-moon shaped red blood…#joetsogbe #crisprtherapeutics #nobelprize #crispr #jenniferdoudna #crisprcas9 #linuspauling #africa #markusmapara #vertex (Source: Reuters: Health)
Source: Reuters: Health - December 7, 2023 Category: Consumer Health News Source Type: news
Mayo Clinic Minute: Sickle cell disease explained
The Food and Drug Administration is expected to decide by Dec. 8 on a new therapy to treat sickle cell disease using gene editing technology called CRISPR, which stands for clustered regularly interspaced short palindromic repeats. Approximately 250 million people worldwide carry the gene for sickle cell disease. It affects those with roots in Africa, Spanish-speaking regions in the Western Hemisphere, Saudi Arabia, India and Mediterranean countries. In the U.S., sickle cell disease is most common in… (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - December 7, 2023 Category: Databases & Libraries Source Type: news
