20205 results
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Orphan designation: autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human palmitoyl-protein thioesterase 1 gene for: Treatment of neuronal ceroid lipofuscinosis
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021that is responsible for the production of an enzyme called PPT1 … lipofuscins in the body. The medicine consists of blood stem cells … patient and treated with a modified virus containing the gene … -
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Orphan designation: Autologous CD34+ haematopoietic stem and progenitor cells genetically modified with a lentiviral vector encoding for the N-acetylgalactosamine 6-sulfatase cDNA for: Treatment of mucopolysaccharidosis type IV A (Morquio A syndrome)
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis … from EMA to advance their medicine to the stage where they can … designation does not mean the medicine is available or authorised … -
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Orphan designation: Autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human iduronate 2-sulfatase gene for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 20/08/2021, Positive, Last updated: 02/03/2022stem and progenitor cells genetically modified with the lentiviral vector … 781 6000 © European Medicines Agency, 2021. Reproduction … EMA/COMP/424709/2021 Human Medicines Division Committee for … -
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Opinion/decision on a Paediatric investigation plan (PIP): Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor (CAR) for B-cell maturation antigen (BCMA) (JNJ-68284528)
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Oncology
PIP number: EMEA-002585-PIP01-19, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Dispersion for infusion
Decision date: 04/10/2019, Last updated: 25/03/2020, Compliance check: XAutologous human T cells genetically modified ex-vivo with a lentiviral … 2019 on the granting of a product specific waiver for autologous … autologous human T cells genetically … -
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Opinion/decision on a Paediatric investigation plan (PIP): Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Other
PIP number: EMEA-003001-PIP01-21, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Dispersion for infusion
Decision date: 13/04/2022, Last updated: 17/04/2023, Compliance check: Xprogenitor cells (HSPCs) genetically modified with the lentiviral vector … progenitor cells (HSPCs) genetically modified....(EMEA-003001-PIP01-21) PDF icon application/pdf … EMA/145564/2022 European Medicines Agency decision P/0105/2022 … -
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Opinion/decision on a Paediatric investigation plan (PIP): Ex vivo expanded autologous human keratinocytes containing epidermal stem cells genetically modified with a gamma-retroviral (RV) vector expressing the full-length LAMB3 cDNA (Hologene 5) (new)
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Dermatology
PIP number: EMEA-003137-PIP01-21, Route(s) of administration: Implant use, Pharmaceutical form(s): Living tissue equivalent
Decision date: 09/09/2022, Last updated: 28/09/2023, Compliance check: Xcontaining epidermal stem cells genetically modified with a gamma-retroviral (RV … containing epidermal stem cells genetically modified with a gamma-retroviral (RV … EMA/706927/2022 European Medicines Agency decision P/0375/2022 … -
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Orphan designation: Genetically modified allogenic (human) tumor cells for the expression of IL-7, GM-CSF, CD80 and CD154, in fixed combination with a DNA-based double stem loop immunomodulator (dSLIM) for: Treatment of renal-cell carcinoma
Date of designation: 23/10/2006, Positive, Last updated: 24/04/2009MOLOGEN AG, Germany, for genetically modified allogenic (human) tumour … the blood, taking out waste products, and making urine. Renal cell … the Committee for Orphan Medicinal … -
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Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Scientific guideline
Last updated: 17/12/2020non-clinical and clinical aspects of medicinal products containing genetically modified cells - Scientific guideline … non-clinical and clinical aspects of medicinal products containing genetically modified cells - Scientific guideline …
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Environmental risk assessments for medicinal products containing, or consisting of, genetically modified organisms (GMOs) - Scientific guideline
Last updated: 11/12/2006nmental risk assessments for medicinal products containing, or consisting … containing, or consisting of, genetically modified organisms (GMOs) - Scientific … nmental risk assessments for medicinal products containing, or consisting …
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Guideline on core SmPC, labelling and package leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells - Scientific guideline
Last updated: 26/10/2022leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells - Scientific guideline … leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells - Scientific guideline …
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Human medicine European public assessment report (EPAR): Imvanex
modified vaccinia Ankara - Bavarian Nordic (MVA-BN) virus, Smallpox Vaccine; Monkeypox virus
Date of authorisation: 31/07/2013,
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, Revision: 25, Authorised, Last updated: 29/03/2023
Complex Mixtures Biological Products Vaccines Viral Vaccines Monkeypox Smallpox … and monkeypox vaccine (Live Modified Vaccinia Virus Ankara) Overview … vaccinia virus called ‘modified vaccinia virus Ankara’ … -
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Human medicine European public assessment report (EPAR): Hepacare
hepatitis B surface antigens recombinant (S, pre-S1, pre-S2) adsorbed on aluminium hydroxide [produced on genetically engineered murine (C127I) cells], Hepatitis B; Immunization
Date of authorisation: 04/08/2000, Withdrawn, Last updated: 17/12/2002Product details Name Hepacare Agency product number EMEA/H/C/000261 Active … aluminium hydroxide [produced on genetically engineered murine (C127I … aluminium hydroxide [produced on genetically engineered murine (C127I … -
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Gene therapy product quality aspects in the production of vectors and genetically modified somatic cells - Scientific guideline
Last updated: 31/12/1994Gene therapy product quality aspects in the production of vectors and genetically modified somatic cells - Scientific … Gene therapy product quality aspects in the production of vectors and genetically modified somatic cells - Scientific …
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Orphan designation: Skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector for: Treatment of dystrophic epidermolysis bullosa
Date of designation: 30/04/2009, Positive, Last updated: 07/10/2014for skin equivalent graft genetically corrected with a COL7A1-encoding … epidermolysis bullosa is a genetic disease of the skin, in which … gene is responsible for the production of a protein called 'collagen … -
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Committee for Advanced Therapies (CAT) workshop: scientific and regulatory challenges of genetically modified cell-based cancer immunotherapy products
European Medicines Agency, London, UK, from 15/11/2016 to 16/11/2016, Last updated: 08/11/2016regulatory challenges of genetically modified cell-based cancer immunotherapy … cell-based cancer immunotherapy products … European Medicines Agency, London, UK The CAT … -
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Veterinary medicine European public assessment report (EPAR): Bovela
modified live bovine viral diarrhoea virus type 1, non-cytopathic parent strain KE-9 and modified live bovine viral diarrhoea virus type 2, non-cytopathic parent strain NY-93,
Date of authorisation: 22/12/2014, Revision: 4, Authorised, Last updated: 27/11/2019done by the Committee for Medicinal Products for Veterinary Use (CVMP … that contains two types of a modified live bovine viral diarrhoea … tend never to reach their productive potential, have reduced fertility … -
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News: How to facilitate development of cancer treatment based on genetically modified T-cells
Last updated: 08/11/2016cancer treatment based on genetically modified T-cells … innovative cancer immunotherapy medicines based on genetically modified T-cells (white blood cells … www.ema.europa.eu/contact © European Medicines Agency, 2016. Reproduction … -
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Orphan designation: Modified mRNA encoding the UGT1A1 protein for: Treatment of Crigler-Najjar syndrome
Date of designation: 27/06/2016, Positive, Last updated: 14/09/2022Alexion Europe SAS, France, for modified mRNA encoding the UGT1A1 … body. Bilirubin is a waste product of the breakdown of haemoglobin … the Committee for Orphan Medicinal … -
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Orphan designation: Adeno-associated viral vector containing a modified U7 snRNA gene for: Treatment of Duchenne muscular dystrophy
Date of designation: 27/07/2005, Positive, Last updated: 11/10/2005adeno-associated viral vector containing modified U7 snRNA gene for the treatment … dystrophy is an inherited genetic disease with onset usually … the chromosomes), carry the genetic information that determines … -
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Opinion/decision on a Paediatric investigation plan (PIP): Imvanex, Modified Vaccinia Ankara - Bavarian Nordic virus (smallpox)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Vaccines
PIP number: EMEA-001161-PIP02-11-M01, Route(s) of administration: Subcutaneous use, Pharmaceutical form(s): Suspension for injection
Decision date: 13/07/2022, Last updated: 26/03/2012, Compliance check: XImvanex Active substance Modified Vaccinia Ankara - Bavarian … EMA/639617/2022 European Medicines Agency decision P/0264/2022 … paediatric investigation plan for modified vaccinia ankara - bavarian … -
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Orphan designation: Adeno-associated viral vector containing modified U1 snRNA for: Treatment of Duchenne muscular dystrophy
Date of designation: 08/10/2009, Positive, Last updated: 11/03/2013adeno-associated viral vector containing modified U1 snRNA for the treatment … dystrophy (DMD) is an inherited genetic disease characterised by … that is responsible for the production of a protein called dystrophin … -
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Orphan designation: Chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA for: Treatment of Huntington’s disease
Date of designation: 19/03/2015, Positive, Last updated: 30/05/2018chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted … gene responsible for the production of a protein called huntingtin … the Committee for Orphan Medicinal … -
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Orphan designation: 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA for: Treatment of Angelman syndrome
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022This medicine was designated as an orphan medicine for the Treatment of Angelman … from EMA to advance their medicine to the stage where they can … designation does not mean the medicine is available or authorised … -
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Orphan designation: 2’-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 pre‑mRNA for: Treatment of progressive myoclonic epilepsy type 2 (Lafora disease)
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021for 2’-O-(2-methoxyethyl) modified antisense oligonucleotide … the Committee for Orphan Medicinal Products (COMP). * For the purpose … treating Lafora disease. Medicines to control seizures were … -
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Orphan designation: Chemically modified human recombinant sulfamidase for: Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Date of designation: 14/10/2016, Positive, Last updated: 15/11/2016publ), Sweden, for chemically modified human recombinant sulfamidase … the Committee for Orphan Medicinal Products (COMP) . * Disclaimer: For … behavioural therapy. How is this medicine expected to work? This …