Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=26519140
&cmd=llinks): Failed to open stream: HTTP request failed! HTTP/1.1 429 Too Many Requests
in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 215
Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\26519140
.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Hum+Mol+Genet
2016 ; 25
(R1
): R42-52
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
Twit Text #
English Wikipedia
Progress and challenges in viral vector manufacturing
#MMPMID26519140
van der Loo JC
; Wright JF
Hum Mol Genet
2016[Apr]; 25
(R1
): R42-52
PMID26519140
show ga
Promising results in several clinical studies have emphasized the potential of
gene therapy to address important medical needs and initiated a surge of
investments in drug development and commercialization. This enthusiasm is driven
by positive data in clinical trials including gene replacement for Hemophilia B,
X-linked Severe Combined Immunodeficiency, Leber's Congenital Amaurosis Type 2
and in cancer immunotherapy trials for hematological malignancies using chimeric
antigen receptor T cells. These results build on the recent licensure of the
European gene therapy product Glybera for the treatment of lipoprotein lipase
deficiency. The progress from clinical development towards product licensure of
several programs presents challenges to gene therapy product manufacturing. These
include challenges in viral vector-manufacturing capacity, where an estimated 1-2
orders of magnitude increase will likely be needed to support eventual commercial
supply requirements for many of the promising disease indications. In addition,
the expanding potential commercial product pipeline and the continuously
advancing development of recombinant viral vectors for gene therapy require that
products are well characterized and consistently manufactured to rigorous
tolerances of purity, potency and safety. Finally, there is an increase in
regulatory scrutiny that affects manufacturers of investigational drugs for
early-phase clinical trials engaged in industry partnerships. Along with the
recent increase in biopharmaceutical funding in gene therapy, industry partners
are requiring their academic counterparts to meet higher levels of GMP compliance
at earlier stages of clinical development. This chapter provides a brief overview
of current progress in the field and discusses challenges in vector
manufacturing.
free
free
free
