Beside Crypto, there are also some other highly promising technologies around, that may change the future considerably. One if these technologies is the CRISPR/Cas genome editing technique. For the development of this technique Emmanuelle Charpentier and Jennifer A. Doudna received the Nobel prize for Chemistry this year. Over time it will completely revolutionize bioscience and first use cases will be established in the treatment of devastating mono-genetical diseases. There are currently three small-cap companies that hold patents for the technique and started with clinical programs, i.e. CRISPR Therapeutics AG, Intelia Therapeutics and EDITAS Medicine Inc.
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Yesterday, EDITAS announced that it has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1/2 clinical trial of EDIT-301, an experimental CRISPR/Cas12a gene editing medicine in development for the treatment of sickle cell disease.
Also CRISPR Therapeutics AG and Intelia Therapeutics are targeting sickle cell disease with the new technology and have already started first in men (Phase I) clinical trials. The reason why all three companies competing against each other to develop a treatment on the basis of the new gene editing technology, may be that it is an extremely clever proof of concept use case with a very limited safety risk. Sickle cell disease ist mostly caused by a hereditary mutation in a single gene resulting in a deformation of red blood cells. This single gene will be targeted by the new technique to "repair" it in order to establish normal blood cell formation. The clue is, that the "treatment" could be done outside of the body (ex vivo) by using hematopoietic stem cells that will than be re-transferred to the patient (autologous transplantation). These repaired but stem cells will than give rise to normal blood cells and the patient may be cured from the disease. As the stem cells were harvested directly from the individual patient, a rejection of the transplant is very unlikely (autologous transplantation).
Although the new CRISPR technology could also be used directly within the body of a patient (in vivo), changing the genetic code of millions of cells has a risk to result in errors (off target effects) that could lead to unwanted outcomes. Therefore, using the technique outside of the body, with the possibility to closely monitor the genetic changes that have been introduced, before patients are directly exposed with the modified cells, is a very clever approach. This is the reason why all three companies have chosen exactly this disease to establish proof of concept data for the technique.
My Investment Strategy
I am holding shares of all three companies since late 2017. I am absolutely convinced that the CRISPR technology as well as the companies holding its patents will play a major role in the future. However, seeing the gains that these shares made during the last month, I decided to take some profits and sold 1/3 of my holdings. I will wait for a re-entry that is approximately 30% lower. That may come with an overall correction of the market or with some negative news of delayed clinical trials or whatever. We will see if this is playing out as expected.
In any case do your own research - this is @no-advice
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