Sorrento Therapeutics Inc (SRNE)

[dia76ca]

Very little attention has been paid to SmartPharms work on a whole host of Orphan enzyme replacement diseases. This a very important future market...with undeniable blockbuster potential.
"Enzyme replacement therapy is the standard therapy for a number of inherited diseases but is mainly used in treating lysosomal storage diseases. Lysosomes are cellular organelles responsible for the metabolism of many different macromolecules and proteins within the cell. The lysosomes utilize enzymes to break down these macromolecules, which are then recycled or disposed of. If genetic mutations prevent the production of certain enzymes used in the lysosomes, this often leads to a buildup of the substrate within the body. The accumulation of substrate can result in a variety of symptoms, many of which are severe and can affect the skeleton, brain, skin, heart, and the central nervous system. Increasing the concentration of missing enzyme in the blood provides cells the ability to correctly process these substrates. There are more than 50 lysosomal storage diseases, each of which is considered to be a rare/orphan disease with a prevalence of less than 1 out of 50,000 people. Lysosomal storage diseases for which enzyme replacement therapy is already approved or in late clinical development include:

Alpha Mannosidosis
Fabry Disease
Gaucher Disease
Mucopolysaccharidosis Type 1 (MPS 1)
Mucopolysaccharidosis Type II (MPS II or Hunter syndrome)
Mucopolysaccharidosis Type 4A (MPS 4A)
Mucopolysaccharidosis Type 6 (MPS 6)
Mucopolysaccharidosis Type 7 (MPS 7)
Neuronal Ceroid Lipofuscinosis Type 2 (NCL2)
Niemann-Pick Type B
Pompe Disease
For more information go to https://smartpharmtx.com