Module 9 2021

A little bit of history ...

2014

2018

1990

1992

1999

2002

2003

2008

2009

2011

2012

2013

2016

2017

1 st FDA GTP-AAV approved (Luxturna)

Safety and efficacy in AMLD, WAS (Science) And CAR + T (NEJM)

SME Academia and big pharma collaboratio n

FIM ex- vivo transdu ced HSC with LV

EMA First cell based product approv al

Safety and

1 st EMA ex-vivo transduced approval (Strimvelis & Zalmoxis)

1 st FDA CAR + T

GT approval in US for ADA-SCID

FIM ex- vivo transduced HSC with RV

AVV5 treated patient death

FIM in EU for ADA- and X-SCID and Blood tumors

Leukem ia-like disease

1 st EU GTP

Safety and efficacy of

approved pALL and DLBCL (Kymriah & Yescarta)

efficacy in ADA- SCID (NEJM)

Approval (Glybera)

CAR+T (NEJM)

in X- SCID

Novartis Pharma AG- Business use only- not for further distribution

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