CRISPR: Gene Therapy 2.0


It looks like 2017 will be an exciting year for the Biotech industry. One development that has caught much attention is the new generation of gene therapy, the CRISPR-Cas9 genome editing tool that with cautious optimism is expected to start a new wave of faster, more accurate treatment for monogenetic diseases through DNA modification.

With the CRISPR-Cas9, (Clustered Regularly Interspaced Short Palindromic Repeats) editing tool, scientists are able to accurately alter a DNA sequence, removing and replacing faulty DNA, and have the potential to treat a range of genetic medical conditions. With around 5,000 inherited disorders caused by genetic errors known to date, the potential for CRISPR is abundant.1

Although the concept of CRISPR gene therapy is new, gene editing startups, including the ones listed below, are already launching and receiving funds for clinical trials and FDA review.

1. Editas Medicine
Founded in part by Feng Zhang, one of the first developers of the CRISPR technology in 2013, Editas Medicine is one of the first CRISPR focused biotech startups to begin discovery studies with the tool. The company’s lead program intended to cure Leber Congenital Amaurosis, a rare form of eye disease is expected to begin human trials in 2017.

Series A: $43 million
Series B: $108 million
IPO: $94.4 million

2. CRISPR Therapeutics
CRISPR Therapeutics founded in 2015 in part by Dr. Emmanuelle Charpentier has a broad pipeline of CRISPR gene editing discovery programs, including collaborations with Bayer and Vertex.

Series A: $35 million
Series B: $67 million
IPO: $56 million

3. Intellia Therapeutics
Intellia Therapeutics’ scientific founder Jennifer Doudna is one leading member of the UC Berkeley team that developed the CRISPR/Cas9 tool. The company’s leading CRISPR discovery programs focus on liver diseases.

Series A: $15 million
Series B: $70 million
IPO: $108 million

You can check out our PharmSource Lead Sheet: Drug Edition search engine to find the latest information on these companies and more!

1. Datto, M. MD PhD Genetic Diseases Due to Single Gene Defects: Case Studies 14 March 2014, PowerPoint file

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Mahdia is a Research Analyst at PharmSource. She earned a BS degree in biology from George Mason University, where she minored in business. Prior to this, she received a magna cum laude honors Associates of Sciences degree from Northern Virginia Community College. Mahdia is avidly interested in clinical research and science.

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