Category: Gene Therapy

The Future of Gene Therapy

By AGT | Oct 24, 2022

Economies of scale can apply to gene therapy too Gene therapy’s advantage is its ability to modify the genetic cause of disease. The approach has the potential to correct genetic errors, cure genetic disease and fight serious infections using the body’s own machinery. Its capabilities seem almost limitless, as does the cost of recently approved therapies. However, gene therapy’s mechanistic… [Read More]

FDA Gene Therapy Approvals

By AGT | Aug 25, 2021

Despite the impact of the pandemic and the seemingly solitary focus of the health care machine on addressing SARS-CoV-2, the gene therapy space continued to progress over the last 18 months.  Several new approvals were granted and promising candidates were advanced in clinical trials and submitted to regulatory authorities.  CAR T has dominated recent gene therapy approvals with three receiving… [Read More]

What Is Gene Editing? How It Works In 2-Minutes

By AGT | Apr 17, 2021

  Big Picture    Gene therapy, stem cell therapy, CAR T, cell therapy, and gene editing are all forms of genomic medicine1 – an approach to cure and treat human diseases that uses human biology rather than chemical compounds made in the lab. All of these tools are unlocking techniques and therapies with the power to cure formerly incurable diseases…. [Read More]

Technology Transfer, A Catalyst For Gene Therapy Startups | The Spark Therapeutics Example

By AGT | Apr 5, 2021

Contributing Author: Gina Hagler   The ability to consistently bring treatments and cures to market depends upon a company’s capacity to create and sustain a successful product development pipeline. Traditionally, pharmaceutical companies have developed small molecule medicines such as Zantac or Lipitor to treat conditions such as heartburn or high cholesterol through a trial and error process that begins without… [Read More]

5 Ways Gene Therapy Companies are Transforming Rare Disease Cures

By AGT | Feb 12, 2021

Contributing Author: Gina Hagler Historically, developing rare disease cures and treatments has been challenging. The small, globally dispersed patient population made it difficult and expensive to bring together enough individuals for a clinical-stage trial. The small patient population of fewer than 1% of the population also made it impossible to recoup the investment in research and development costs required for… [Read More]

3 Gene Therapy Companies Powering a Revolution in Rare Disease Cures

By AGT | Jan 20, 2021

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]

5 Reasons Gene Therapy Companies are Vital to the Future of Biopharma

By AGT | Jan 20, 2021

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]

5 Biotech Companies in Maryland That May Make History in 2021

By AGT | Jan 20, 2021

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]

List of 88 Gene Therapies

By AGT | Oct 20, 2020

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]

Gene Therapy: More than Monogenic Disease

By AGT | Aug 26, 2020

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]

The Death of the Regulatory Barrier to Better Medicine

By AGT | Jul 7, 2020

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]


By AGT | Mar 2, 2020

Contributing Author:  Norman Rogers, Former 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… [Read More]

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