Since the dawn of molecular biology medical science has been captivated by the dream of curing diseases by correcting defective genes. Recent advances have produced a number of gene editing programs in or near clinical development, implying the dream could soon be reality. In this piece we review the science and discuss leading biotech programs. Our top stock picks include BLUE, EDIT, and SGMO.
Gene Editing Refers To A Number Of Technologies Which Have The Potential To Repair Defective Genes.
It is estimated that there are roughly 6,000 diseases caused by genetic mutations, of which perhaps 5% can be addressed with approved therapies today. Gene editing seeks to directly remove or repair the defective DNA. The promise of gene editing is therefore in its ability to correct the defective genes themselves, curing the genetic diseases. As gene editing has the theoretical potential to address most (if not all) diseases with a genetic basis, over the long haul it could revolutionize medicine, and create a multi-billion dollar therapeutic class. In this piece, we discuss the technologies that have been developed to manipulate genes and review our surveys of, and conversations with, experts in the space.
The First Data From Sangamo’s Gene Editing Programs Anticipated In Early 2018
Sangamo Therapeutics (SGMO, Outperform, Baral) is developing therapeutics based on its Zinc Finger Nuclease (ZFN) gene editing platform. The company has initiated Ph1/2 trials of gene editing programs SB-FIX in hemophilia B, SB-318 in MPS I and SB-913 in MPS II, with data from all anticipated in early 2018.
EDIT To File An IND For Its Lead CRISPR Program In A Genetic Form Of Blindness In Mid-2018
Editas Medicine (EDIT, Outperform, Nadeau) is a leader in the discovery and development of therapeutics based on the CRISPR gene editing technology. Editas’ lead program targets Leber Congenital Amaurosis 10 (LCA 10), a genetic form of retinal degeneration and blindness that is caused by mutations in the CEP290 gene. Editas expects to move a CRISPR/Cas9 system into the clinic during 2018 that employs the NHEJ editing mechanism, delivered to the back of the eye by the AAV vector.
Gene Editing Programs In Development At A Number Of Other Companies.
Other companies with notable gene editing programs include bluebird (BLUE, Outperform, Schmidt), Caribou (private), Cellectis (CLLS), CRISPR (CRSP), Homology Medicine (private), Intellia (NTLA), and Precision Medicines (Private).
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