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Antisense Oligonucleotides and colony Stimulating Factors

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Let me move on to Antisense drugs. What is antisense? Or what is antisense technology? Antisense drugs are designed to interfere with messenger RNA or with the function of messenger RNA Now we all remember that protein synthesis involves two major steps, one is the transcription, and the other is translation. Transcription involving coding the genetic information from the DNA to the messenger RNA. And the messenger RNA must remain single-stranded to be function. Now if we have an antisense or Oligonucleotide to couple with the messenger RNA or to block the messenger RNA, And here, the messenger RNA is a sense molecule and the Oligonucleotide is antisense molecule, and that’s where the antisense, the technology, I mean the terminology comes from.
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So when the messenger RNA is blocked, translation could not proceed and therefore, proteins and the cells will not be synthesized. Now, the traditional chemotherapy kills the cells, or the cancer cells, only after they are formed. Now, the antisense drug would prohibit the cells or the cancer cells from being formed. So in principle it has a very great potential for anti-cancer therapy. And this is the first product approved in 2002 for the treatment of CMV, that is the cytomegalovirus infection in AIDS patient. Now, this is the optimistic infection when the AIDS patient has compromised immune function. And it is administered by direct injection of the 300 microgram, a very small dose into the vitreous chamber.
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And as you have noticed the chemical name ends with “Sen”, that’s the antisense. The second approval did not come about ten years later and that’s kynamro. It’s it is an orphan drug approved in 2013. (It) indicated for homozygous familia hypercholesterolemia. It’s a congenital or hereditary disease. It’s given 200 milligram SC weekly. Next, therapeutic class is the colony stimulating factors. colony stimulating factors are glyco proteins. that acts to promote the preparation and the differentiation of specific blood cell types. Primarily the white blood cells. So let’s look at how colony-stimulating factors work.
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Here there are three types: GM-CSF, the G-CSF and the M-CSF, working together in consequence or in sequence. They promote the proliferation and differentiation of stem cells into the neutrophilic vanilla site. And the monocyte macrophages and all these cells form the immune foundation of our defense system. CSF types. GCSF, the granulocyte colony-stimulating factor and there are two products that I wanted mention here. Filgrastim, 12 injections per chemotherapy cycle, but Neulasta which is a peglated Philistine now can be given one those six milligrams per team of therapy cycle. So by pegylation, the half-life is substantially prolonged, and that therefore. it can be injected less frequently for the convenience of the patient. GM-CSF, Sargramostim, another one is Macrophage colony-stimulating factors. Promegapoietin.
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And as you noticed that these products are all indicated to decrease or for the decrease of infection. Given before chemotherapy or during chemotherapy, to reduce the chance or the instant of infection. And these are the packages. For Filgrastim and for Neulasta. And again, pay attention to the dosage strength.

Antisense oligonucleotides are short, single-stranded DNA molecules that interact with messenger RNA to prevent translation of a target gene. In addition to its unlimited potential for therapeutic applications, antisense technology has been widely used in agricultural bioengineering to improve crop trait, known as gene silencing. Colony stimulating factors are glycoproteins that promote production of white blood cells (mainly granulocytes such as neutrophils), in response to infection. Administration of exogenous colony stimulating factors stimulates the stem cells in the bone marrow to produce more of the particular white blood cells.

Antisense oligopeptides (ASO) are named with the suffix of –sen in their nomenclature. Early approval includes Formivirsen (2002) and Mipovirsen (2013). Because of its great therapeutic potential, numerous ASOs are in current development inventory. In 2019, FDA granted accelerated approval to an antisense oligonucleotide (ASO) therapy, Golodirsen for a genetic neuromuscular diseases, Duchenne muscular dystrophy. The best known colony stimulating factors are Amgen’s Neupogen (filgrastim) and Neulasta (peglylated filgrastim), used to reduce chemotherapy-induced infection.

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