FDA approves first gene-editing treatment for human illness
Dec 8, 2023 · It's a blessing that they approved this therapy," said Victoria Gray, the first person in the U.S. to undergo CRISPR gene-editing for sickle cell, of the Food and Drug Administration's …
FDA Approves First Gene Therapies to Treat Patients with ...
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first …
Gene Therapy News - ScienceDaily
6 days ago · Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing. ... Gene …
Gene therapy - Latest research and news - Nature
Oct 31, 2024 · RSS Feed. Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated …
Muscular dystrophy patients get first gene therapy - NPR
Jun 22, 2023 · In a first, FDA approves Sarepta gene therapy for Duchenne muscular dystrophy : Shots - Health News The Food and Drug Administration approved the first gene therapy for …
FDA Approves First Gene Therapy for Adults with Severe ...
June 29, 2023. Español. Today, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe …
First Patient Begins Newly Approved Sickle Cell Gene Therapy
May 6, 2024 · Kendric Cromer, 12, the first commercial patient for Bluebird Bio’s gene therapy to cure his sickle cell disease, in the hospital as his bone marrow stem cells were being removed …
FDA approves cure for sickle cell disease, the first ...
Dec 8, 2023 · The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene …
The world’s first CRISPR therapy is approved: who will ...
Nov 21, 2023 · The go-ahead for Vertex’s gene editing therapy in sickle cell disease and β-thalassemia is a historic milestone, but this one-time treatment is costly. ... An essential round …
Fresh from the biotech pipeline: record-breaking FDA approvals
Feb 26, 2024 · Nature Biotechnology 42, 355–361 (2024) Cite this article. New drug approvals reached an all-time high in 2023, with five gene therapies, the first CRISPR–Cas9-edited …
FDA expands approval of first gene therapy for rare form of ...
Jun 20, 2024 · 03:27 - Source: CNN. CNN —. The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be …
One-time gene therapy reverses liver disease MASH in mouse …
1 day ago · The UAB licensed this gene therapy program with AAV-FGF21 to the company Tramontane Therapeutics Inc., now part of the biopharmaceutical company Kriya, which will …
FDA Approves First Gene Therapy for Treatment of Certain ...
Jun 23, 2023 · FDA Approves Elevidys, a gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in …
Experimental gene therapy restores some vision in patients ...
May 6, 2024 · The trial, which started in 2019, enrolled 12 adults, ages 17 to 63, and two children, ages 9 and 14, with inherited retinal degeneration caused by mutations in the CEP290 gene. …
Most of today’s gene therapies rely on viruses - Science News
Oct 20, 2023 · Since gene therapy was first proposed to treat such genetic diseases in the 1970s, it has had thrilling highs — including the first successful gene therapy, in 1990, in a 4-year-old …
Gene therapy for pancreatic cancer: Experimental approach ...
Jun 1, 2022 · A new gene therapy shrank the tumors of a woman with pancreatic cancer. Researchers think it has the potential to benefit millions more people. An experimental …
FDA approves $3.2 million gene therapy for rare muscular ...
Jun 22, 2023 · Sarepta said the therapy, called Elevidys, will cost $3.2 million per patient. It’s designed to be taken just once. The group for whom the drug was approved, children ages 4 …
Stanford Medicine offers gene therapy for a devastating ...
Jun 20, 2024 · Stanford Medicine Children’s Health has become one of only four locations nationally to offer gene therapy for a devastating neurologic disease called X-linked …
Gene therapy: 'Now I can see my own face again' - BBC
Gene therapy: 'Now I can see my own face again'. "Last year, for a lot of people was a dark and miserable year, but for me it was probably easily the best year of my life." Jake Ternent has …
Neurogene Reports Positive Interim Efficacy Data from First ...
3 days ago · Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome Business …