The study
analyzed that the therapeutics pipeline comprises approximately 16 drug
candidates in different stages of development. Spinal muscular atrophy is
defined as the inherited genetic disease that is characterized by a failure of
nerve cells called motor neurons. Motor neurons are responsible to accept the
nerve impulses transmitted from the brain to the spinal cord and transmit the
impulses to the muscle with the help of peripheral nerves. The loss of motor
neurons leads to muscle weakness in muscles that are closest to the trunk of
the body such as back, hips and shoulders.
Imago
Pharmaceuticals, Inc. is using nucleic acid therapeutics technology platform
for the treatment of spinal muscular atrophy. Nucleic acid therapeutics
technology includes large and innovative class of drugs that can modulate the
function of target ribonucleic acid, or RNA, to ultimately affect the
production of disease-associated proteins. Nucleic acid therapeutics comprise
of complex mixtures of various chemical entities known as stereoisomers. Some
stereoisomers in these mixtures have therapeutic effects, while others are less
beneficial or contribute to undesirable side effects. Uncontrolled stereoisomer
drug mixtures can lead to suboptimal efficacy and increased risk and safety
concerns.
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The
research also found that there are several companies that use synthetic sources
for the development of drugs for the treatment of spinal muscular atrophy.
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Some of
the other key players developing drugs for the treatment of spinal muscular
atrophy include Cytokinetics, Inc., F. Hoffman La-Roche Ltd, WAVE Life Sciences
Ltd. and others.
Spinal
Muscular Atrophy Therapeutics Pipeline Analysis
By Phase
By Route
of Administration
By
Molecule Type
By Company
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