Next Generation Delivery System for Proteins and Genes of Therapeutic Purpose: Why and How?

Table 1

Next generation therapeutic proteins or genes and their delivery system which are in the market or in clinical trial.

Brand name of therapeutic protein/gene	Therapeutic protein/gene	Indication	Remark	References
Oncaspar	Therapeutic protein (pegylated formulation of L-asparaginase; Pegaspargase)	Leukaemia	USFDA-approved in 1994	[50]
PEG-INTRON	Therapeutic protein (pegylated formulation of IFN-a2B; Peginterferon alfa-2b)	Hepatitis C as well as malignancies	USFDA-approved in January 2000	[51]
PEGASYS	Therapeutic protein (pegylated formulation of IFN-a2A; Peginterferon alfa-2A)	Hepatitis C	USFDA-approved in January 2001	[52]
Neulasta	Therapeutic protein (pegylated formulation of Granulocyte-colony stimulating factor (GCSF) and monomethoxypolyethylene glycol; Pegfilgrastim)	Neutropenia	USFDA-approved in January 2002	[53]
Mircera	Therapeutic protein (pegylated formulation of Erythropoietin (EPO); Epoetin beta-methoxy polyethylene glycol)	Anemia associated with kidney disease	USFDA-approved in January 2007	[54]
— (No brand name available)	Therapeutic gene (RNAi therapeutics delivery of ALN-PCS02 using SNALP liposome)	Hypercholesterolemia	Clinical trial	[55]
Glybera	Therapeutic gene (alipogene tiparvovec used adeno-associated virus serotype 1 (AAV1) viral vector delivery)	Familial lipoprotein lipase deficiency (LPLD, synonym: type I hyperlipidaemia).	First gene-therapy medicine and approved by all 27 European Union member states	[56]