Novel therapeutic uses and formulations of botulinum neurotoxins: a patent review (2012 – 2014)

Abstract

Introduction: Botulinum neurotoxins (BoNTs) are among the most toxic of known biological molecules and function as acetylcholine release inhibitors and neuromuscular blocking agents. Paradoxically, these properties also make them valuable therapeutic agents for the treatment of movement disorders, urological conditions and hypersecretory disorders. Greater understanding of their molecular mechanism of action and advances in protein engineering has led to significant efforts to improve and expand their function with a view towards broadening their therapeutic potential.

Areas covered: Searches of Espacenet and Google Patent have revealed a number of patents related to BoNTs. This review will focus on novel therapeutic uses and formulations disclosed during 2012 – 2014. The seven patents discussed will include nanoformulations of FDA-approved BoNTs, additional BoNT subtypes and novel BoNT variants and chimeras created through protein engineering. Supporting patents and related publications are also briefly discussed.

Expert opinion: The clinical and commercial success of BoNTs has prompted investigation into novel BoNTs or BoNT-mediated chimeras with promising in vitro results. Distinct strategies including the use of nanoformulations and targeted delivery have been implemented to identify new indication and improved functionality. Greater understanding of their systemic exposure, efficacy and safety profiles will be required for further development.

Keywords:

• antinociception
• botulinum toxin
• exocytosis
• hydrogel
• liposome
• motor dysfunction
• new indication
• pain
• spinal cord injury

Declaration of interest

The views, findings, interpretations and recommendations are those of the authors and are not necessarily endorsed by the US Department of Health and Human Services or the US Army. The authors express their gratitude for financial support provided by the Joint Science and Technology Office – Chemical Biological Defense (JSTO-CBD) Defense Threat Reduction Agency (DTRA) under sponsor project number CCAR# CB3675 and National Institutes of Health (5 U01AI082051-05). The authors are indebted to Theresa Smith for providing many helpful discussions and they would also like to thank William F Discher for his assistance in creating the figures for this manuscript. The authors have no other relevant affiliations or financial involvements with the subject matter or materials discussed and indicate no potential conflicts of interest.

Notes

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