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National Human Genome Research Institute

Division of US Department of Health and Human Services
www.genome.gov

Latest From National Human Genome Research Institute

Deals Shaping The Medical Industry, July/August 2014

Derived from Strategic Transactions, Informa’s premium source for tracking life sciences deal activity, the Dealmaking column is a survey of recent health care transactions listed by relevant industry segment – In Vitro Diagnostics, Medical Devices, and Pharmaceuticals – and then categorized by type – Acquisition, Alliance, or Financing. This month’s column covers deals announced May and June 2014.

BioPharmaceutical Medical Device

Unpacking Lysosomal Storage: New Insights Drive Parkinson’s Drug Development

Investors and dealmakers are using recent discoveries about convergences between Gaucher’s disease and Parkinson’s in the lysosome to generate new therapeutic programs. Start-up Lysosomal Therapeutics thinks that the rare disorder can serve as a model system for developing new drugs to increase lysosomal function as a way to treat Parkinson’s disease and possibly other neurodegenerative disorders.

BioPharmaceutical

Genomics Enters The Neonatal ICU

Tumor profiling is on the upswing, but prenatal genomic screening remains the largest translational application of whole genome sequencing. And now, the use of whole genome sequencing is spreading into the neonatal arena.

Medical Device Clinical Trials

Molecular Diagnostics Ushers In New Era Of Prenatal Testing

The recent launch of several noninvasive gene-based tests for Down syndrome and other fetal chromosomal abnormalities is poised to upend current prenatal screening practices and potentially usher in a new, multibillion-dollar market opportunity in the process. These new tests employ next-generation gene sequencing, along with advanced, risk-based computer algorithms, to detect and characterize fetal genetic abnormalities using the DNA present in a maternal blood sample. And because they are actually able to “interrogate” the DNA, they represent a huge technological advance over the current standard-of-care for prenatal screening.

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