Wednesday, October 18, 2017

Seeing "Cus" Gene

This week something different and yet, an amazing, miracle like news. For some kids that have a disease that affects sight also known as Leber congenital amaurosis, seeing Cousin Gene is a problem. The good news there is a light at the end of the tunnel.
An FDA panel has unanimously voted to recommend a gene therapy by Sparks pharmaceuticals which makes the treatment. If you are thinking of buying the stock, it is already up 70% this year. I suggest that you wait for a pullback.
No pills
No injections just one treatment. Sparks uses a virus to deliver a healthy copy of a gene to make up for the one that causes the harm or disease. There are others in this field, but Sparks stands to be the first to bring this style of medicine to the US market. At the moment, the recommendations of the panel along with the trials will be decided by the FDA to allow the treatment to be used in the market. The therapy is designed to treat a rare, inherited form of blindness. There is no approved treatment for the disease and people with the condition will go blind.
There has been some incredible success stories associated with Sparks' therapy. The FDA should make their decision by mid-January 2018.
By the way, this treatment and style of medicine is getting a lot of buzz by Big Pharma. The concept has many applications. Pfizer is already partnering with Sparks. Voyager Therapeutics is using genes to work with problems in the brain.(Ma, your prayers for me are answered). Other companies in this field are: Bluebird Bio and Regenxbio.
This is so cool and congrats to the people at Sparks.
Future World
In a related aspect, TransEnterix(TRXC) has received approval for its robotic surgery system, Senhance. In the near future it is possible that a doctor using Senhance robotic system could apply the Sparks treatment to cure the Leber disease. Wow!