Business Strategy & Outlook:
Intellia Therapeutics is a gene editing company focused on the development of CRISPR/Cas9-based therapeutics. Intellia’s technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. CRISPR/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations. CRISPR/Cas9 works by having CRISPR (pieces of DNA sequences) guide Cas9 (an enzyme that can cut and edit DNA) to edit, alter, or repair genes. Intellia is utilizing this gene knockout approach to remove unwanted proteins using its proprietary lipid nanoparticle delivery system. Intellia has leveraged its expertise in CRISPR/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs. It is believed that the company’s proprietary technology has the potential to build blockbusters in rare diseases with limited treatment options available. Intellia currently has no approved drugs and a largely early stage pipeline.
Risk and Uncertainty:
A significant uncertainty related to regulatory approvals for the company’s early-stage pipeline candidates and a range of potential outcomes. Product governance is an ESG risk for Intellia, as failure to adhere to extensive regulations can lead to expensive recalls, increased regulatory scrutiny, and lawsuits from affected customers. Additionally, lawsuits related to patent rights and potential patent infringements are another risk. It is anticipated gene editing companies like Intellia will operate under cross licensing agreements with the Broad Institute as many of the gene editing technology platforms are interrelated. Access to basic services is another ESG risk the company will face if its drugs receive approval since its sales will depend on reimbursements from third-party payers, such as Medicaid or Medicare, private insurers, and national healthcare systems. Attempts by governments to contain healthcare costs could result in pricing pressure and lead to reduced profit margins.
Bulls Say:
- Intellia’s partnerships allow it to receive milestones and economic benefits from drug candidate progression while offsetting some of the clinical development costs.
- Intellia’s CRISPR/Cas9 platform has the potential to develop highly efficacious and curative treatments for rare, genetic diseases with high unmet needs, which will likely lead to pricing power if approved.
- The company’s pipeline is viewed as possessing strengthening intangible assets and assign it a positive moat trend.
Company Description:
Intellia Therapeutics is a gene editing company focused on the development of CRISPR/Cas9-based therapeutics. CRISPR/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), which is a revolutionary technology for precisely altering specific sequences of genomic DNA. Intellia is focused on using this technology to treat genetically defined diseases. It’s evaluating multiple gene editing approaches using in vivo and ex vivo therapies to address diseases with high unmet medical needs, including ATTR amyloidosis, hereditary angioedema, sickle cell disease, and immuno-oncology. Intellia has formed collaborations with several companies to advance its pipeline, including narrow moat Regeneron and wide-moat Novarti
(Source: Morningstar)
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