Editas Medicine

Editas Medicine is a leading genomic medicine company that is focused on translating the power and potential of gene editing technologies into a new class of precision medicines. The company's mission is to develop transformative, durable, precision genomic medicines for a broad class of diseases.

History

Editas Medicine was founded in 2013 by a group of world-renowned scientists who are pioneers in genome editing. The company is headquartered in Cambridge, Massachusetts, a hub for biotechnology innovation. Since its inception, Editas Medicine has been at the forefront of developing and advancing CRISPR gene editing technologies.

Technology

Editas Medicine's technology platform is based on CRISPR-Cas9, a revolutionary gene editing system that allows for precise, directed changes to genomic DNA. CRISPR-Cas9 has been adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to "remember" the viruses. If the viruses attack again, the bacteria produce RNA segments from the CRISPR arrays to target the viruses' DNA. The bacteria then use Cas9 or a similar enzyme to cut the DNA apart, which disables the virus.

Clinical Development

Editas Medicine is developing a robust pipeline of therapies for the treatment of serious diseases with significant unmet medical need. The company's lead program, EDIT-101, is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis (LCA), a rare genetic disorder that leads to blindness early in life. EDIT-101 is the first in vivo (inside the body) CRISPR medicine to be administered to patients.

Future Directions

Editas Medicine continues to leverage its leading genome editing platform to develop a broad pipeline of therapies for the treatment of serious diseases with significant unmet medical need. The company is committed to discovering and developing novel medicines that can transform the lives of patients with genetically defined diseases.

See Also

• Genomic Medicine
• Gene Editing
• CRISPR
• Leber congenital amaurosis

References

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