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bluebird bio (Germany) GmbH Josephspitalstr. 15 | Regus 80331 München, Germany http://www.bluebirdbio.com
Contact Ms Claudia Nabaie +41 79 906 58 14
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bluebird bio (Germany) GmbH

bluebird bio präsentiert Langzeitdaten für die Gentherapie mit elivaldogene autotemcel (eli-cel, Lenti-D™)

Zur Behandlung der Zerebralen Adrenoleukodystrophie (CALD)

(PresseBox) (Zug, Schweiz, )
- 90 % der auswertbaren Patienten (27/30) leben und sind frei von schweren funktionellen Behinderungen (major functional disablities, MFDs)* nach zwei Jahren Nachbeobachtung in der Phase-2/3-Studie Starbeam (ALD-102)1

- Patienten der Langzeit-Follow-up-Studie (LTF-304) sind bis zu fast sieben Jahre in der Nachbeobachtung weiterhin am Leben und frei von MFDs, was darauf hindeutet, dass eli-cel das Fortschreiten der Krankheit stabilisiert1

- Bei den 51 Patienten, die bislang in klinischen Studien (ALD-102/LTF-304 and ALD-104) mit eli-cel behandelt wurden, wurden keine Fälle von Transplantatversagen, Transplantatabstoßung, Graft-versus-Host-Disease (GvHD), replikationskompetentem Lentivirus oder Insertionsonkogenese berichtet1

- Diese Daten wurden bei einem Vortrag im Presidential Symposium pum fpt 43. Plzuhkpajewx oyb Rftvqddq Htzolcd dml Stpvp whx Lqgmve Tfrhiglloizmlwn (FJXT) vufgpumobuj

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