Consequently, Broad received the first issued US patent to the use of CRISPR-Cas9 technology in gene editing in eukaryotic cells in April 2014. UCB’s patent application remained in the examination queue. In essence, despite UCB being the first to file its patent applications, the Broad patent was preferentially issued.

Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Given CRISPR Therapeutics already has data on its key gene therapy candidate and has more potential market reach with its oncology pipeline, it is without a doubt a much safer choice than Editas. In addition, CRISPR Therapeutics is in a slightly better financial position than Editas.

Researchers at U.S.-based Editas Medicine and Ireland-based Allergan have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness.

Top CRISPR Startup Companies Changing the Future of Biotech and Medicine

• Mammoth Biosciences: Using CRISPR to Advance Clinical Diagnostics.
• Inscripta Therapeutics: Increasing CRISPR’s Reach.
• eGenesis: Using CRISPR to Improve Organ Transplants.
• Synthetic Genomics: Harnessing CRISPR to Create Sustainable Energy.

Using the CRISPR system, researchers can precisely edit any target DNA locus – a feat that was not achievable using other gene editing tools. The possibility to edit a disease mutation to correct genetic errors creates opportunities for treating conditions that have long eluded the medical research community.

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Overall, given the vast institutional interest, its stable financial position, and the success of similar gene therapeutics in the industry, Editas is a risky but high-reward stock to buy.

These biotech start-ups use a handful of powerful new tools to repair genetic mistakes.

CRISPR has the financial flexibility to fund its research programs for many more years to come. So it can prove to be an attractive investment for healthcare investors in the next five years.

Gene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA.

Gene therapy , or somatic gene editing, changes the DNA in cells of an adult or child to treat disease, or even to try to enhance that person in some way. The changes made in these somatic (or body) cells would be permanent but would only affect the person treated.