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Handb Exp Pharmacol. 2016;232:59-70. doi: 10.1007/164_2015_17.

In Vivo Target Validation Using Biological Molecules in Drug Development.

Author information

1
Bayer HealthCare, 455 Mission Bay Blvd. South, Suite 493, San Francisco, CA, 94158, USA. derek.sim@bayer.com.
2
Bayer HealthCare, 455 Mission Bay Blvd. South, Suite 493, San Francisco, CA, 94158, USA.

Abstract

Drug development is a resource-intensive process requiring significant financial and time investment. Preclinical target validation studies and in vivo testing of the therapeutic molecules in clinically relevant disease models can accelerate and significantly de-risk later stage clinical development. In this chapter, we will focus on (1) in vivo animal models and (2) pharmacological tools for target validation.

KEYWORDS:

Bleeding disorders; Gene therapy; Gene-editing; Hemophilia; Monoclonal antibodies; Mouse models; Non-mammalian models; Recombinant proteins; Target validation

PMID:
26552401
DOI:
10.1007/164_2015_17
[Indexed for MEDLINE]

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