Biosecurity Assessments for Emerging Transdisciplinary Biotechnologies: Revisiting Biodefense in an Age of Synthetic Biology.

Introduction: Rapid advances in biotechnologies and transdisciplinary research are enhancing the ability to perform full-scale engineering of biology, contributing to worldwide efforts to create bioengineered plants, medicines, and commodities, which promise sustainability and innovative properties.

Objective: This rapidly evolving biotechnology landscape is prompting focused scrutiny on biosecurity frameworks in place to mitigate harmful exploitation of biotechnology by state and non-state actors. Concerns about biosafety and biosecurity of engineering biology research have existed for decades as views about how advances in this and associated fields might provide new capabilities to malicious actors.

Open-label, single-center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I.

Lysosomal enzyme deficiency in mucopolysaccharidosis (MPS) I results in glycosaminoglycan (GAG) accumulation leading to pain and limited physical function. Disease-modifying treatments for MPS I, enzyme replacement, and hematopoietic stem cell therapy (HSCT), do not completely resolve MPS I symptoms, particularly skeletal manifestations. The GAG reduction, anti-inflammatory, analgesic, and tissue remodeling properties of pentosan polysulfate sodium (PPS) may provide disease-modifying treatment for musculoskeletal symptoms and joint inflammation in MPS I following ERT and/or HSCT.