Up to 89% of published research in the Life Sciences can not be reproduced according to Bayer and Amgen studies and is a part of the growing reproducibility crisis -- which is mainly due to lack of standardization, varying reagents, and inconsistent conditions. Another fact worth mentioning is that in the era of robotics and automation almost 90% of biologists are still doing pipetting by hand with monotonous, routine operations being accountable for a bigger part of a project timeline. The cloud-based approach to the chemistry and biology field will provide the necessary degree of repeatability and greatly reduce the costs while speeding up the whole process. In the near future, we could see a massive shift which lies in outsourcing the daily operations to a robotized remote lab while freeing up the researcher's time for creativity and generating new ideas. 

As of now, only 5% of so-called rare diseases (orphan diseases) have an FDA-approved treatment available to patients. Rare diseases are defined as those affecting fewer than 1 in 200000 people with almost 80% of all rare diseases being of genetic nature. while only  

With recent progress in gene-editing technologies, including breakthroughs like CRISPR/Cas9 and new therapy delivery technologies, there is a wave of innovative biotech companies entering the field of rare diseases equipped with new ways to address the problem. 

In this article, I would like to outline a list of notable companies targeting rare diseases with cutting-edge gene therapies.