Intellia Therapeutics Inc. (NTLA), a biotechnology company working on “editing” defective genes, is developing potential treatments for liver and blood diseases and cancer. It is one of the few biotech companies focused on a technology called Crispr-Cas9 that has raised hopes for future drugs that could home in on specific genes and remove them.
Intellia is in the midst of a high-stakes intellectual property dispute with Jennifer Doudna being one of the founders of the company facing off against cheif rival Editas Medicine (EDIT) and Feng Zhang. Both Doudna and Zhang are locked in a very public battle for discovery credit of the groundbreaking Crispr/Cas9 technology.
Intellia Therapeutics believes that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. The company intends to leverage its leading scientific expertise, clinical development experience and intellectual property position to unlock broad therapeutic applications of CRISPR/Cas9 gene editing and develop a potential new class of therapeutic products.
The CRISPR/Cas9 system offers a revolutionary approach for therapeutic development due to its broad potential to precisely edit genes. This system can be used to make three general types of edits: knockouts, repairs and insertions. Each of these editing strategies takes advantage of naturally occurring biological mechanisms to effect the desired genetic alteration. This approach has the potential to provide curative therapeutic options for patients with chronic diseases by addressing the underlying genetic cause or driver of the disease.
Unlike earlier-generation gene editing technologies, the CRISPR/Cas9 system is simple and involves a single protein, Cas9, that can be directed to precisely cleave a target DNA sequence by using pieces of RNA, called guide RNAs, that specifically recognize the target DNA of interest. Therefore, CRISPR/Cas9-based therapeutics have the potential to be highly efficient, selective and scalable.
Intellia has an extremely long way to go to create value as a drug developer but the company has some big backers with shareholders including Novartis AG and Regeneron Pharmaceuticals Inc.
Intellia Therapeutics went public on May 6, 2016.
Intellia Therapeutics, Inc. is a United States-based genome editing company focused on the development of curative therapeutics using a biological tool, CRISPR/Cas9 system. The company’s CRISPR/Cas9 technology edits diseased genes in the human body through a single treatment course. It focuses on the therapeutic applications of CRISPR/Cas9 genome editing. Its portfolio includes CRISPR/Cas9 platform technology, delivery technologies and nucleic acid modifications for human therapeutic use. Intellia‘s CRISPR/Cas9 platform components include the guide ribonucleic acid (RNA) and the Cas9 nuclease (the genetic scissors). It has access to various delivery modalities specific to various in vivo and ex vivo approaches. Intellia Therapeutics focuses on engineering ex vivo CRISPR/Cas9 cell therapies for chimeric antigen receptor T cell (CAR-T) and hematopoietic stem cell (HSC) applications. The company also focuses on the use of in vivo Lipid Nanoparticle (LNP) delivery technology.
NTLA is heading back towards its original IPO price of $18 after peaking at $30 dollars per share in late May.
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