New biotherapeutics

The new biotherapeutics may be defined as the molecules being discovered and studied through the disciplines of biotechnology and molecular biology. Research is currently concentrated in two main areas ... 

These new biotherapeutics are discussed briefly below, with particular reference to the problems associated with their successful dmg delivery and targeting. 

In common with the peptide and protein-based new biotherapeutics described above, successful delivery is one of the major practical problems in gene therapy. To be effective, the genetic material must ... 

Limitations of conventional drag delivery systems are particularly acute for the new biotherapeutics, such as peptide and protein drugs and nucleic acid therapies. 

List the drag delivery challenges presented by the new biotherapeutics . 

New technologies are also addressing the pulmonary delivery of the new biotherapeutics , i.e. the products of biotechnology and molecular biology such as peptides, proteins and gene therapies which have been described in detail in Chapter 1 (Section 1.6). Biopharmaceuticals under investigation for potential pulmonary delivery include those for local, and systemic, effects (Table 10.4). 

Ideally, drags used in the treatment of diseases affecting the CNS would be delivered directly to the site of action. However, drags generally do not readily enter the brain from the circulating blood. Access to the brain is particularly difficult for the new biotherapeutics such as peptide, protein and nucleic-acid based biopharmaceuticals. 

In response, the field of advanced drag delivery and targeting has seen an explosion of activity, as researchers address these obstacles and try to facilitate or enhance the action of the new biotherapeutics, as well as conventional drugs. 

Profile A private company, Neugenesis was founded in 1992 by several key individuals who recognized the potential commercial value of Neurospora for biotechnology applications. It has nine employees and occupies a 1600 square foot facility for its headquarters and R D facilities. The company uses proprietary combinatorial biology technology to discover and develop new biotherapeutics, both alone and in partnership with other biopharmaceutical companies. 

Niven RW (1995) Delivery of biotherapeutics by inhalation aerosol. In Bruck SD (ed.) Crit Rev Ther Drug Carrier Syst. Begell House Inc, New York, pp 151-231. 

Akwete, A.L., Gupta, P.K., Eds. Niven, delivery of biotherapeutics by inhalation aerosol. In Inhalation Delivery of Therapeutic Peptides and Proteins, Marcel Dekker, Inc. New York, 1997 151-231. 

A full validation assessing all of the performance characteristics discussed above should be performed for a new method. Unlike for small molecules, for biotherapeutics it is recommended that a full validation be conducted when there is a change in species (e.g., rat to mouse) and a change in matrix within a species (e.g., rat serum to rat urine). In all the situations discussed in detail below, it is necessary to have a validation plan with a priori established acceptance criteria. 

The authors are deeply indebted to their colleagues at GTC Biotherapeutics and Genzyme. The development of a new dmg is truly an enormous endeavor, and entire scientific teams have dedicated years of their professional lives to the filing of ATryn. In addition, the authors extend special thanks to Suzanne Groet, Tom Newberry, and Dick Scotland for their critical reading and constructive suggestions, as well as to Jennifer Williams and Merry Harvey for their generous help with the illustrations. 

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