Contributing Author: Gina Hagler Gene therapy companies are powering a revolution in rare disease cures by offering treatments that target the genetic cause of the disease. Rather than try different combinations of chemicals to create a small-molecule treatment as is the case with traditional pharmaceuticals, gene therapy uses genetic material in drug development to repair or replace the genetic cause…. [Read More]
Contributing Author: Gina Hagler Pharmaceutical companies have been developing therapies to treat or prevent disease since the end of the 19th century. Chemical compounds are used to create these drugs and medications. The resulting small-molecule drugs – think aspirin – are ingested, metabolized and circulate through the blood. Because it’s not possible to target these drugs, they have a systemic… [Read More]
Contributing Author: Gina Hagler These five biotechnology companies in Maryland may make history in 2021 because they have multiple therapeutic programs in trials for a variety of genetic diseases. REGENXBIO is a publicly-traded company that develops products for one-and-done, single-dose solutions addressing genetic defects. Their products use adeno associated virus (AAV) viral vectors that make up their proprietary NAV Technology… [Read More]
Biotechnology is a rapidly expanding sector where game-changing innovation is a daily occurrence. Gene and cell therapies are of particular interest because of the steep technology curve driving the technology platform. The gene and cell therapy industry is characterized by numerous small, competitive, agile businesses; each with a unique toolset. The decreasing cost trend of these genetic technologies continually lowers… [Read More]
Contributing Author John Vandermosten, Senior Biotechnology Analyst Gene therapy is a diverse technology that can address a broad variety of diseases, conditions and disorders. About a year ago, the FDA approved its latest gene therapy product, Zolgensma, for spinal muscular atrophy (SMA), a neuromuscular disorder caused by a mutation in the SMN1 gene. Gene therapy is a natural fit for solving… [Read More]
Written by Jeff Galvin, CEO of American Gene Technologies The birth of high-value therapeutics and cures Does the development of effective drugs have to be incredibly expensive? Maybe not anymore. There is a new type of drug development emerging: Gene Therapy. The hallmark of gene therapy is the ability to edit or modify the drivers of disease at the most fundamental… [Read More]
Contributing Author: Norman Rogers, Business Development Lead at AGT Gene therapy is a tool to modify the composition and expression of human DNA. Our DNA is the software that codes for every mechanism in the human body. Many diseases are caused by a single error in this code. Until recently, there was no way to directly correct DNA so diseases… [Read More]
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