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  2. Gene therapy for blood diseases - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy_for_blood...

    In December 2023, the European Medicines Agency recommended approval for a cell based gene therapy that works through the CRISPR/Cas9 system. The therapy known as Casgevy [9] works through editing a dysfunctional protein that interferes with creation of adult hemoglobin. This gene is known as the BCL11A, and when people have Beta thalassemia ...

  3. Elivaldogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Elivaldogene_autotemcel

    Elivaldogene autotemcel was designated an orphan drug by the European Medicines Agency (EMA) in 2012. [23] Elivaldogene autotemcel was granted orphan drug, rare pediatric disease, and breakthrough therapy designations by the US Food and Drug Administration (FDA). [24] In September 2022, elivaldogene autotemcel was granted accelerated approval. [25]

  4. List of Guidances for Statistics in Regulatory Affairs

    en.wikipedia.org/wiki/List_of_Guidances_for...

    EMA/199678/2016: Reflection paper on extrapolation of efficacy and safety in paediatric medicine development. [8] EMA/189724/2018: Reflection paper on the use of extrapolation in the development of medicines for paediatrics. [9] EMA/129698/2012: Concept paper on extrapolation of efficacy and safety in medicine development. [10]

  5. European Medicines Agency - Wikipedia

    en.wikipedia.org/wiki/European_Medicines_Agency

    The Committee for Advanced Therapies (CAT) was established in accordance with Regulation (EC) No 1394/2007 on advanced-therapy medicinal products (ATMPs) such as gene therapy, somatic cell therapy and tissue engineered products. It assesses the quality, safety and efficacy of ATMPs, and follows scientific developments in the field. [24]

  6. Gene therapy - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy

    In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis [238] [239] and the European Commission approved it in June. [240] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene ...

  7. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    "Gene Therapy Arrives". Scientific American This page was last edited on 10 November 2024, at 06:28 (UTC). Text is available under the Creative Commons Attribution ...

  8. ncRNA therapy - Wikipedia

    en.wikipedia.org/wiki/NcRNA_therapy

    One such example is Nusinersen (Spinaraza), it functions as an ASO and targets pre-mRNA before splicing that corresponds to Survival of motor neuron 2 gene (SMN 2). [34] This drug therapy was approved by FDA and EMA in 2016 and 2017 respectively. There are some drugs that have been approved by FDA and not by EMA.

  9. European Directorate for the Quality of Medicines & HealthCare

    en.wikipedia.org/wiki/European_Directorate_for...

    The European Directorate for the Quality of Medicines & HealthCare (EDQM) is a Directorate and partial agreement of the Council of Europe that traces its origins and statutes to the Convention on the Elaboration of a European Pharmacopoeia (an international treaty adopted by the Council of Europe in 1964: ETS 50, [2] Protocol [3]).