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European Biopharmaceutical Review

Biopharmaceuticals for the Brain

Biopharmaceuticals have become a mainstay of modern medicine, with seven of the ten top-selling medicines worldwide comprising biological drugs (1). As complex molecules bring a unique set of challenges, biopharmaceuticals offer medicines with high specificity and efficacy, with generally few side effects. With a development success rate of over 11% from Phase 1 clinical development to marketing authorisation, biopharmaceuticals are twice as likely to achieve approval as small molecule drugs (2). Biopharmaceuticals represent a class of drugs which display high molecular diversity, comprising different types of molecules, such as growth factors, cytokines, coagulation factors, lysosomal enzymes, receptor antagonists, antibodies, and antibody fragments. Biopharmaceuticals are generally priced at the high end of the market; with the first biologicals now off-patent, an incipient market for biosimilar drugs (copies of biologicals no longer under patent protection) is starting to bloom as opportunities to produce less expensive versions present themselves.

Many challenges are associated with the production and use of biopharmaceuticals. Their molecular nature requires complex manufacturing methods to assure molecule reproducibility. The need to build and maintain appropriate manufacturing facilities, as well as expensive raw materials and the demand for highly skilled workforces, all contribute to the high cost of biological drug production. Furthermore, an administration burden exists for patients associated with biopharmaceuticals. As oral administration is not yet possible, drugs must be administered via intravenous or subcutaneous injections. These challenges are opening up opportunities for new innovations, as novel routes of administration – as well as targeted delivery – are being investigated (3-4).

The diagnosis of diseases associated with specific organs and tissues of the body, or with specific intracellular organelles, represents an area of unmet medical need which has yet to be successfully addressed. Diseases of the central nervous system, such as Alzheimer’s disease and Parkinson’s disease (generally associated with adults) or some of the mucopolysaccharidoses (more associated with children) have not yet been treated with disease-modifying pharmaceuticals, in particular, biopharmaceuticals. In a similar vein, diseases resulting from cellular stress, leading to dysfunction of the mitochondrion or the endoplasmic reticulum, have not yet received specific targeted medicines. The opportunity that biopharmaceuticals present to address some of these challenges is unique to this class of drugs. Engineering of the proteins which comprise the active ingredients of biopharmaceuticals may help to target them to some of these more intractable conditions, in some cases, exploiting the body’s physiological systems to achieve this.

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Erik Nordling PhD is a Principal Scientist working in the biomedical science and portfolio innovation department at Sobi. Erik has an MSc in chemistry from the Mid Sweden University, Sweden, and holds a PhD in medical biochemistry from Karolinska Institutet, Sweden.

Stefan Svensson Gelius PhD is a Principal Scientist working in the biomedical science and portfolio innovation department at Sobi. Stefan has a long-standing interest in innovation in the field of protein biochemistry. He holds a PhD in medical sciences from Karolinska Institutet, Sweden.

Stephen James PhD is Head of Research and Translational Science at Sobi. He has experience in developing small molecule and biological pharmaceuticals in various disease indications, in particular, in rare inflammatory, haemostasis, and genetic diseases. Prior to moving to industry, Stephen was University of Dundee Research Fellow in biochemistry at the University of Dundee, UK. He obtained his doctoral degree from the University of Leeds, UK.
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Erik Nordling PhD
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Stefan Svensson Gelius PhD
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Stephen James PhD
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