StemCell Therapy

The gene-editing revolution is already here, and these stocks will help you capitalize on the movement.

Gene-editing therapies allow you to remove cells from the body, modify them and reintroduce them. With this technique, theres hope for a cure for cancer, blood disorders, blindness, AIDS, cystic fibrosis, muscular dystrophy, Huntingtons disease and a host of other diseases.

For example, gene-editing has already been used to eliminate HIV in mice.

According to the National Institute on Drug Abuse, HIV-1 could be eliminated in mice using a combination of two antiviral technologies long-acting viral reservoir-targeted antiretroviral therapy and CRISPR/Cas-9 gene editing.

Doctors are even attempting to cure blindness after hacking a patients genes.

A patient recently had the procedure done at the Casey Eye Institute at Oregon Health & Science University for an inherited form of blindness.

We literally have the potential to take people who are essentially blind and make them see, said Charles Albright, chief scientific officer at Editas Medicine. We think it could open up a whole new set of medicines to go in and change your DNA.

If the procedure is found to be a success, doctors plan on testing it on more children and adults. Success for one company could also create a sizable opportunity in the sector for related stocks as well.

Source: Catalin Rusnac/ShutterStock.com

CRISPR Therapeutics (NASDAQ:CRSP) is one of the top names in the gene-editing market with nine drug candidates.

One candidate is CTX001, a drug that targets sickle cell and transfusion-dependent beta-thalassemia (TBT). With both, patients have poorly formed red blood cells that just cant delivery oxygen throughout the body well. In Nov. 2019, CTX001 successfully helped to eliminate symptoms in a patient with TBT, and another with sickle cell.

Overall, while still very early, the results provide the first suggestion of curative potential for this cutting-edge technology in such genetic diseases, and with potential for further safety refinement of Crispr/Cas9 administration, could suggest broad long-term potential of the many early-stage gene editing therapeutic tools VRTX has accumulated, RBC Capital Markets Brian Abrahams wrote.

Other candidates (CTX110, CTX120, CTX130) are also candidates for a cancer treatment known as chimeric antigen receptor T cell (CAR-T) therapy.

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Editas Medicine (NASDAQ:EDIT) along with Allergan (NYSE:AGN) just treated a blind patient with EDIT-101 as part of a Brilliance phase clinical trial for the treatment of Leber congenital amaurosis (LCA). With this study, its the first time a patients genes are being modified in the body itself, which is known as in vivo treatment.

Editas is also working on a sickle-cell disease and transfusion-dependent beta-thalassemia (TDT) drug, EDIT-301. EDiT-102 is being developed for Usher Syndrome 2a, a genetic condition characterized by hearing loss and vision loss that begins in adolescence or adulthood.

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Intellia Therapeutics (NASDAQ:NTLA) is also working on a sickle-cell disease drug. Its also working on NTLA-5001, a drug that could help treat acute myeloid leukemia (AML). This is a rare type of cancer found in the bone marrow, which leads to the production of abnormal red and white blood cells.

In addition, the company is working on NTLA-2001 for transthyretin amyloidosis, a rare condition characterized by buildups of protein deposits called amyloids throughout the body, which can lead to the loss of sensation in extremities, and in internal organs.

Better, according to the company, In 2019, we advanced our full-spectrum strategy, guiding both ourin vivoandex vivo lead programs toward the clinic. We also continued to build on our genome editing and delivery capabilities to enable a rapid succession of candidates, said Intellia President and Chief Executive Officer, John Leonard, M.D., adding:

We are off to a productive start in 2020. We announced the nomination of NTLA-5001, a WT1-directed TCR-T cell therapy for the treatment of AML, and plan to select our third development candidate in the first half of this year, which will be for the treatment of HAE. In addition, in the second half of the year, we expect to begin dosing ATTR patients with NTLA-2001, a potential single-course treatment for ATTR patients. This is anticipated to be the first-ever systemically delivered CRISPR/Cas9-based therapy to enter the clinic, representing an important milestone in our mission to deliver potentially curative therapies from our proprietary modular platform.

Ian Cooper, an InvestorPlace.com contributor, has been analyzing stocks and options for web-based advisories since 1999. As of this writing, Ian Cooper did not hold a position in any of the aforementioned securities.

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The Top 3 Gene-Editing Stocks to Own in 2020 - Investorplace.com

This entry was posted on March 12, 2020 at 8:47 am and is filed under Genetic medicine. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |