#117257
Re: Farmas USA
An Epic biotech week Notes: Gene Editing
The Genetics editing space has been challenging of late. Ever since SGMO has failed in-vivo editing, the move has been toward ex-vivo editing of T cells and Stem cells. That is all good since there are some huge opportunities in that space, but now you have a dozen companies all chasing the same indications. Some of the newer technologies like Homolgous Recombination and Homing Endonucleases are unproven. It could be a decade before we know they are truly safe. My biggest fear in in-vivo gene editing is another disaster where the patients all develop cancer years after being treated.
SGMO – They have gone from the hopeful front runner in the in-vivo race to the underdog. I still think SGMO has a huge platform of technology that it can develop, and its all at an extremely cheap value. They have the new gene therapy platform they are developing to fill the gap until their in-vivo editing issues get worked out. They have a fantastic ex-vivo editing program that is very successful for both T cells and Stem cells. I will admit I am not sold on much beyond that yet. I do think the cell therapies alone more then justify this value. A lot of shots on goal here with multiple platforms. It would be foolish if I thought everything would be a success with these programs.
CRSP – This is my top dog in gene editing. It still owns all of its T cell editing programs, and it is in some very awesome programs like its regenerative medicine around iPSC. There is no doubt that this company is the fan favorite and the valuation is really up there. That hasn't stopped me from embracing it on the fundamentals and making a ton of profits off trading it. I think they have a huge potential future that could make them the Celgene of gene editing someday.
EDIT – Not my favorite company for CRISPR, but its just so cheap. I think they don't have their act together wit their science. They do have a very cheap valuation, and they are in some good indications. The eye has a limited immune system. There are not antibodies inside the eye. The retinal injections for eye disorders do not provoke immune responses. We see this in RGNX's data as they are doing people with pre-existing antibodies with no problems. They also have their T cell editing program already partnered which gives them lower potential. The rest of their pipeline is around muscle and lung diseases and we see issues in this space as the current vectors to not penetrate these tissues well. I think is a solid back up company in the CRISPR space if my CRSP got bought out.
NTLA – I am not a fan of this CRISPR company at all. They are highly partnered with Regeneron for their lead drug. They are going right into the in-vivo editing. I think that only sets them up to be the company that makes all the mistakes with developing in-vivo editing. I think this one is only setting itself up to disappoint its investors over and over again as it pioneers in-vivo editing with CRISPR technology. God help us if they really mess it up. They really have no other programs that are all that interesting. Cheap is sometimes cheap for a reason.
FIXX – They have a very early stage science platform around Homologous Recombination. The science sounds great, but I am concerned how it translates in-vivo without off target editing. This science platform has not been proven to edit in humans yet. I am just watching from the sidelines.
DTIL – They have a platform called ARCUS which uses custom built homing endonucleases. These are built for each and every indication. I wonder how much time and costs really goes into designing these things. They did have some early data for ex-vivo editing of T cells that was very good. That goes a long way to show this works at least in the ex-vivo setting. All the same concerns of off target editing with the in-vivo applications still exist.
