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  2. FDA approves cure for sickle cell disease, the first ... - AOL

    www.aol.com/news/fda-approves-cure-sickle-cell...

    December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...

  3. FDA approves two gene therapies for sickle cell, bringing ...

    www.aol.com/news/fda-expected-approve-first...

    The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...

  4. FDA considers first CRISPR gene editing treatment that may ...

    www.aol.com/fda-considers-first-crispr-gene...

    CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function ...

  5. CRISPR gene editing - Wikipedia

    en.wikipedia.org/wiki/CRISPR_gene_editing

    The CRISPR treatment for LCA10 (the most common variant of Leber congenital amaurosis which is the leading cause of inherited childhood blindness) modifies the patient's defective photoreceptor gene. In March 2020, the first patient volunteer in this US-based study, sponsored by Editas Medicine , was given a low-dose of the treatment to test ...

  6. Exagamglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Exagamglogene_autotemcel

    DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

  7. Off-target genome editing - Wikipedia

    en.wikipedia.org/wiki/Off-target_genome_editing

    The safety of gene therapy treatment is of utmost concern, especially during clinical trials when off-target modifications can block the further development of a candidate product. [55] Perhaps the most well-known example of modern gene therapy is CAR-T therapy, which is used for the treatment of B-cell lymphoma .

  8. CRISPR treatment has been greenlit in UK in global first ...

    www.aol.com/uk-regulators-approve-medical...

    The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool. CRISPR treatment has been greenlit in UK in ...

  9. CRISPR Therapeutics - Wikipedia

    en.wikipedia.org/wiki/CRISPR_Therapeutics

    CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. [2][3] The company has approximately 500 employees and has offices in Zug ...