Research

Focus on the pharmaceutical and biotech industry
Gene Therapy for Hemophilia A

Gene Therapy for Hemophilia A

Sangamo is a genomic medicine company that is starting to develop gene therapies for a number of conditions. Sandy Macrae, PhD, Chief Executive Officer of Sangamo talks about the work they are currently doing to develop a gene therapy for hemophilia A. While great...

Drug Price vs Drug Value

Drug Price vs Drug Value

Nick Kenny, PhD, Chief Scientific Officer at Syntheos Health discusses his company’s efforts to streamline data management for clinical trials, including the need to determine the value that a new drug will have on the health care system.

Angelman Syndrome Clinical Study Showing Promise

Angelman Syndrome Clinical Study Showing Promise

Angelman syndrome is a rare neurodevelopmental disorder caused by a genetic mutation – usually a mutation in the ubiquitin protein ligase E3a (UBE3A) gene – that leads to a reduction in UBE3A protein.

An Oral Patch to Treat Lennox-Gastaut Disease

An Oral Patch to Treat Lennox-Gastaut Disease

Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc).

The Genetics of Huntington’s Disease Explained

The Genetics of Huntington’s Disease Explained

Spark Therapeutics is developing a gene therapy to treat one of the most devastating genetic disease known – Huntington’s disease. A disease that robs people of their motor skills and psychological stability in the prime of their life.

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