General Stock Ideas

[gfp927z]

Ombow, The SGMO presentation from March 12 is available on their website (link below), and I see they have another presentation tomorrow -


https://investor.sangamo.com/events/detail/1438/cowen-38th-annual-health-care-conference


Gene therapy has always been interesting, and a lot of the earlier safety concerns have apparently been solved, so looks like the sector is here to stay. One investment approach to consider would be to own some shares of the top players like Sangamo, Bluebird, and Crispr for the long haul. I put Bluebird on my watchlist several years ago and I see their market cap is up 10 fold since then. Too bad I didn't own any lol.

Here's an interesting panel discussion with some top players in gene therapy, including Sangamo's CEO -

I see there's some research going on for a gene therapy approach for treating Wet Macular Degeneration, which currently lacks good treatment options, other than the Lucentis approach (MAB/monoclonal antibody) which has only limited benefit. My mom lost most of her vision due to that condition, and my dad has the dry form. They're also working on gene therapies that can cross the blood brain barrier and thus be able to treat CNS conditions.

Some of the coolest science anywhere is found in this sector, which unfortunately makes it dangerous to us investors who get all gaga and succumb to the 'blinded me with science' syndrome, ala Cortex -


https://en.wikipedia.org/wiki/Gene_therapy


>>> Gene Therapy - Approaches

Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered – replacing or disrupting defective genes.[27] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[26]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[28] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[29][30] Naked DNA approaches have also been explored, especially in the context of vaccine development.[31]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014 these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[32]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[33] viral diseases,[34] and cancer.[35] As of 2016 these approaches were still years from being medicine.[36][37] <<<