CRISPR Therapy Signifies the Dominant Era of Clinical Medicine

CRISPR (Clustered regularly interspaced short palindromic repeat) therapies are regarded as one of the most potentially powerful and transformative technologies in human history with disruptive potential in the cancer therapies in healthcare as per the GlobalData’s (a leading data and analytics company) Pharmaceutical Intelligence Centre.

The current treatment of cancer drugs can only prolong life by a few years at best, whereas CRISPR-based therapies will be able to prolong the life of cancer patients by decades. Thereby they may inevitably transform the lives of patients and the shape of the industry if they are brought to market.

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‘CRISPR (Clustered regularly interspaced short palindromic repeat) therapies are regarded as one of the most potentially powerful and transformative technologies in human history, especially in cancer therapies. Three leading companies with overlapping and distinct target indications are among the race to introduce these CRISPR therapies to the clinic, are – CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine, which may help develop novel therapeutics.’

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Among the race to introduce these CRISPR therapies to the clinic, there are three leading companies – CRISPR Therapeutics, Intellia Therapeutics and Editas Medicine.

Leading the race with the largest market cap of the three, at $10.1bn, and a more advanced clinical development program is CRISPR Therapeutics. Promising data on the use of CRISPR in £]-thalassemia and sickle cell disease have been published by the company in the New England Journal of Medicine.

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The early-stage clinical trials for an immuno-oncology program, based on the development of allogeneic chimeric antigen receptor T (CAR-T) cells targeting well-characterized targets in hematological malignancies such as CD19+ and B cell maturation agent, has also been initiated by the company.

Second in the race is Intellia Therapeutics that is utilizing Intellia’s NTLA-2001 – an in vivo approach to tackle transthyretin amyloidosis, a rare hereditary disorder with only chronic treatment options currently available. A high-profile collaboration of Intellia with companies like Regeneron and Novartis, may boost its ability to navigate a successful route to market.

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Editas Medicine holds the smallest market cap among the three companies with only a single agent in clinical development: EDIT-101, focusing on Leber Congenital Amaurosis – an ocular disease.

Thus the data imply overlapping and distinct target indications for all three companies in hematological disorders and developing an immuno-oncology program, either in the clinical or pre-clinical stages.

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“This suggests potential future competition but also indicates that each company has room to carve out and monopolize distinct markets. CRISPR Therapeutics has a head start in terms of its clinical development and raising capital, and represents a safer choice for investors as a result. However, each of these companies produces little to no current revenue, and current expectations are contingent on favorable results from upcoming clinical trials, which are still in early stages of development”. “Future challenges in this space include how to develop a fair pricing strategy, the logistics of offering broad access to the most needy populations in underserved markets (such as sickle cell patients in Africa), and potential unfamiliar adverse events associated with this novel modality of therapy. However, the potential upside for patients and long-term investors is huge,” says Adam Pearson, a Senior Oncology Analyst at GlobalData.

Source-Medindia