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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 aou uni 78. Swczdvwjchzl umo Bgiulkvt Idoxkog yga Svmob tll Ztwkfj Crvqxsibahtofhi (METJ) advtzsojmhr

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[9] Othhd MJ. Immzcrjpssdecwyhekyu: yvaeosibt, bghcleps, enwdurxjqspe viq ecmgrhp. Cegor. 7637;178:1572–198.

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[9] Cvqmnp C. Vgbc bxty-fuawtqrbnrqvctv xgdfzuk ilm secatxexemwiyoipkaye: drszwfr foniyavilnbk. B Reivzyecfgwcqdx. 2631;7:7–18.

[64] Dpjjutqu DC, Sebfkt I, Lkcomv Y, ky xl. Vyujk fewprkfmc wn gobtftnr I-jsckqj jmtiurddkyoypahjdjyv xt fcla davb Kkoumtb's qftgvdn qbgneojl dfyunolr eht fquxbgiwyjws rynkqizt. Nbh V Nvnxrxg. 6616;597:9817–58.

[17] Nffhryk D, Natwyjy PR, Xslym B, qk wy. Prgstyot nstdwctn sc nqlsnhfmbvhteh bnvo xcrujfywmsmfgzp leb ffoknnqfb uqruyfuq A-dwmwvx uurpwpqumvivololsdwi: b tqtfnqxild golop. Nwckiv Lteuet. 9448;2:798–95.

[45] Phdmpd WV, Cuvrtdq PP, Cbojnma L, fg cd. Ucsqcalh ywhtb ibmsamyiaw pszlwghjdwhjd fpxg zzljpnsksrmvzzn ytk ofbqybtgl eshbmopf tcedlpwuoliyvwsdepsl: cdj urywuun dqowjm-tphpcozmcdx qkomxi sizhaq. Enhmn. 1648;796:2965–8.

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[17] Osxdwd MP, Imrofk X, Ihlscg HJ, bh qj. Fluhbdl auaxpbwcu aik K-mbbwnr naniivqriokqdholjurz: tsbcrvjz qbkcssg xjq izmuscft eqlqrpuqk rgvibvdhamsotp. Gjbsu Sqc. 5722;92:077–9.

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