Gene editing refers to the modification of DNA in the human genome, either by directly delivering gene-editing components into the body or by delivering cells edited in a specialized laboratory to the patient's body. It is often applied in the areas of oncology, hematology, neurology, and genetic disease.
Amongst a variety of gene editing technologies utilized by biotech companies, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9) method has gained traction in recent times due to its simplicity, versatility, and accuracy. Other comparatively old technologies include transcription activator-like effector-based nucleases (TALEN), zinc finger nucleases (ZFN), and meganucleases, which have certain benefits of their own such as low cost (ZFN), higher specificity (TALEN), and low toxicity (meganucleases). At present, gene editing therapies have not been commercialized, but several have already reached Phase I and II clinical trials, with the majority of them based on the CRISPR technology.
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CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.
The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of June 2022, the company had three internal and two collaborative drug candidates in its pipeline, all of which were in the clinical stage. It was also researching other in vivo approaches for four disease indications.
Key customers and partnerships
The company has secured strategic collaborations with a number of companies to co-develop and co-commercialize gene editing medicines that include Vertex Pharmaceuticals (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and ViaCyte (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.
Funding and financials
Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
R&D therapies: CRISPR/Cas9 technology:
R&D therapies: Other technologies:
Diagnostic and delivery systems:
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