In the last six months, I looked at the biotechnology, hoping to find attractive targets for the next investment objectives. Gene therapies, and further reaching into a status of cure, gene editing, are without doubt future of medicine and how genetic diseases will be treated in not so distant future. Each company named below has spent and will continue to spend ample amount of financial resources to deliver the “better” in the science. This index is an attempt to track the market performance of the group and some of the developments in the sector.
I look at two gene treatments in a simple way. A gene therapy is likely to be repeated but it replaces continuing use of existing treatment. Its durability period is limited but could be very long in comparison with conventional treatment. The change in a frequency and the quality of application to eliminate a symptom of the disease is perhaps the closest to the cure versus the current treatment. The outcome could eliminate repetitive doses, injections referred to as enzyme replacement therapies.
On the other hand, editing is an application of the solution just one time, where the cure is obtained via the address of the root cause of the disease by the edit of the gene. The solution may come from a different area of the body to produce the desired effect as a mechanism replacing the normally functioning process in a healthy subject.
The combined worth of companies in the index is about $50B. The edit companies are worth combined $7.5B.
My review uses the formula which will use the market valuation as well as the approach of progress in clinical trials.
The list is arranged based on the market cap as of October 12, 2018. I may have missed companies in gene therapy category simply because of their market cap. The cut off is at $500M. To the readers goes an ask to suggest companies with such a market cap, involved in the gene editing or/and therapy which are missed but meet this criterion.
Sarepta Therapeutics, Inc. (SRPT)
bluebird bio, Inc. (BLUE)
REGENXBIO Inc. (RGNX)
CRISPR Therapeutics AG (CRSP)
Spark Therapeutics, Inc. (ONCE)
Sangamo Therapeutics, Inc. (SGMO)
Editas Medicine, Inc. (EDIT)
Solid Biosciences Inc. (SLDB)
Cellectis S.A. (CLLS)
uniQure N.V. (QURE)
Audentes Therapeutics, Inc. (BOLD)
Intellia Therapeutics, Inc. (NTLA)
Rocket Pharmaceuticals, Inc. (RCKT)
Homology Medicines, Inc. (FIXX)
AVROBIO, Inc. (AVRO)
Voyager Therapeutics, Inc. (VYGR)
Abeona Therapeutics Inc. (ABEO)
The Market Cap and Clinical Programs
The Index list organized by the market cap. Gene edit or therapy programs listed starting with an indicator of phase 2 trial are also reflected in the table. No indication means the level of the program is best at IND or pre-clinical.
Genomic Index Score
Below the Index view since inception on October 12th, 2018 as captured on October 17th, 2018. Arranged by total gain.
Disclosure: I am/we are long SGMO.