Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products
Abstract Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases. In 2016, the European Commission granted market approval to GlaxoSmithKline (GSK) for ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of...
Saved in:
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Springer Nature
2017-04-01
|
| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.15252/emmm.201707573 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849342135230267392 |
|---|---|
| author | Alessandro Aiuti Maria Grazia Roncarolo Luigi Naldini |
| author_facet | Alessandro Aiuti Maria Grazia Roncarolo Luigi Naldini |
| author_sort | Alessandro Aiuti |
| collection | DOAJ |
| description | Abstract Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases. In 2016, the European Commission granted market approval to GlaxoSmithKline (GSK) for ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of adenosine deaminase (ADA)‐deficient severe combined immunodeficiency (SCID), a very rare congenital disorder of the immune system. The new medicine, named Strimvelis™, is an advanced therapy medicinal product (ATMP) (Salmikangas et al, 2015) originally developed by the San Raffaele Telethon Institute for Gene Therapy (SR‐Tiget), a joint venture between Telethon Foundation and San Raffaele Scientific Institute. This ATMP is the first ex vivo stem cell gene therapy to receive regulatory approval anywhere in the world. Strimvelis™ consists of a single infusion of autologous gene‐corrected HSC and is prepared from the patient's own bone marrow (BM) HSCs, which are genetically modified using a gamma‐retroviral vector to insert a functional copy of the ADA gene. |
| format | Article |
| id | doaj-art-6700be1243f84893a0f01eee5df21658 |
| institution | Kabale University |
| issn | 1757-4676 1757-4684 |
| language | English |
| publishDate | 2017-04-01 |
| publisher | Springer Nature |
| record_format | Article |
| series | EMBO Molecular Medicine |
| spelling | doaj-art-6700be1243f84893a0f01eee5df216582025-08-20T03:43:29ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842017-04-019673774010.15252/emmm.201707573Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal productsAlessandro Aiuti0Maria Grazia Roncarolo1Luigi Naldini2San Raffaele Telethon Institute for Gene Therapy (SR‐Tiget), IRCCS San Raffaele Scientific InstituteSan Raffaele Telethon Institute for Gene Therapy (SR‐Tiget), IRCCS San Raffaele Scientific InstituteSan Raffaele Telethon Institute for Gene Therapy (SR‐Tiget), IRCCS San Raffaele Scientific InstituteAbstract Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases. In 2016, the European Commission granted market approval to GlaxoSmithKline (GSK) for ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of adenosine deaminase (ADA)‐deficient severe combined immunodeficiency (SCID), a very rare congenital disorder of the immune system. The new medicine, named Strimvelis™, is an advanced therapy medicinal product (ATMP) (Salmikangas et al, 2015) originally developed by the San Raffaele Telethon Institute for Gene Therapy (SR‐Tiget), a joint venture between Telethon Foundation and San Raffaele Scientific Institute. This ATMP is the first ex vivo stem cell gene therapy to receive regulatory approval anywhere in the world. Strimvelis™ consists of a single infusion of autologous gene‐corrected HSC and is prepared from the patient's own bone marrow (BM) HSCs, which are genetically modified using a gamma‐retroviral vector to insert a functional copy of the ADA gene.https://doi.org/10.15252/emmm.201707573 |
| spellingShingle | Alessandro Aiuti Maria Grazia Roncarolo Luigi Naldini Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products EMBO Molecular Medicine |
| title | Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products |
| title_full | Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products |
| title_fullStr | Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products |
| title_full_unstemmed | Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products |
| title_short | Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products |
| title_sort | gene therapy for ada scid the first marketing approval of an ex vivo gene therapy in europe paving the road for the next generation of advanced therapy medicinal products |
| url | https://doi.org/10.15252/emmm.201707573 |
| work_keys_str_mv | AT alessandroaiuti genetherapyforadascidthefirstmarketingapprovalofanexvivogenetherapyineuropepavingtheroadforthenextgenerationofadvancedtherapymedicinalproducts AT mariagraziaroncarolo genetherapyforadascidthefirstmarketingapprovalofanexvivogenetherapyineuropepavingtheroadforthenextgenerationofadvancedtherapymedicinalproducts AT luiginaldini genetherapyforadascidthefirstmarketingapprovalofanexvivogenetherapyineuropepavingtheroadforthenextgenerationofadvancedtherapymedicinalproducts |