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TOPIC: ANTISENSE OLIGONUCLEOTIDE
PRESENTED BY:
Omkar Kumar Kuwar
Department of Pharmacology
ISF College of Pharmacy, MOGA
omkar.official.955@gmail.com
GUIDED BY:
Dr. Arti Singh
Associate Professor
Department of Pharmacology
ISF College of Pharmacy, MOGA
1
CONTENTS
2/27/2024
ISF college of pharmacy
2
 Introduction
 Definition
 Mechanism of ASO action
 Advantages
 Challenges & Limitations
 Applications
 Refrences
INTRODUCTION to antisense oliGonucleotide
2/27/2024
Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7
3
 Antisense : complementary or negative base sequence to a portion of the
coding or positive sense mRNA.
 Oligo : means scant or few
 Nucleotide : 5 organic molecule that make up the backbone of the DNA &
RNA. ( ATCGU)
 Antisense oligonucleotide therapy is a new way to block protein function is to
prevent the translation of protein by inhibiting translation step.
 Antisense oligonucleotides (ASOs) are synthetic single stranded
oligonucleotides that can bind to precursor mRNA (pre-mRNA) and/or
mRNA through Watson-Crick base pairing.
 ASOs can modulate the function of RNA, there by regulating gene expression.
definition
2/27/2024
Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7
4
 Oligonucleotides are chemically synthesized using phosphoramides.
 The oligonucleotide chain proceeds in direction of 3’ to 5’ terminus.
 Antisense RNA prevent protein translation of certain mRNA strands by
binding to them.
 Antisense DNA can be used to target a specific cRNA.
Mechanismsof ASOs Action
2/27/2024
Refrence; www.researchgate.net
5
Mechanismsof ASOs Action
2/27/2024
Refrence; www.researchgate.net
6
Fomivirsensodium
2/27/2024
Refrence ; Drug bank
7
 Fomivirsen is a 21-base phosphorothioate oligonucleotide.
 Fomivirsen is marketed under the trade name Vitravene for
intravitreal injection and was the first antisense drug to be
approved by the Food and Drug Administration (FDA).
 Indication
Indicated for the local treatment of cytomegalovirus (CMV)
retinitis in patients with acquired immunodeficiency syndrome
(AIDS),
• injected into the eye (intravitreal) 330 µg ( 0.05 mL/eye )
 Half-life about 55-78 hours.
CMV is a viral infection of the retina of the eye resulting if the
eye inflammation.
Mipomersen
2/27/2024
Refrence ; Drug bank
8
 Familial hypercholesterolemia is a genetic disorder caused by a
defect on chromosome-19, that makes the body unable to remove
LDL. Or bad cholesterol this results in high level of LDL in blood.
 The FDA label includes a black box warning of hepatoxicity.
 Administer through subcutanious 200 mg/1mL
 Half-life: 1-2 months.
 Drug interaction:
Acetaminophen, Amiodarone, Demeclocycline, Doxycycline,
Eravacycline,
They may increase the hepatotoxic activities of Mipomersen.
 Food Interactions;
Avoid excessive or chronic alcohol consumption. Alcohol may increase
the risk of liver injury.
Eteplirsen
2/27/2024
Refrence ; Drug bank
9
 Used to treat Duchenne muscular dystrophy (DMD)
 Duchenne muscular dystrophy is a rare genetic disorder
characterized by progressive muscle deterioration and premature
death most commonly due to respiratory or cardiac complications.
 It is caused by loss-of-function mutations in the DMD gene coding
for dystrophin, an essential protein involved in maintaining the
structural integrity and function of muscle fibres.
 Approved by the FDA in September 2016.
 Administer Intravenous strength 50 mg/mL
 Half-life (t1/2) 3-4 h.
Nusinersen
2/27/2024
Refrence ; Drug bank
10
 Spinal muscular atrophy. It is a disorder affecting the motor
neurons-nerves that control voluntary muscle movement.
 It is administrated as direct 2.4 mg/1mL Intrathecal.
 Half-life: 135 to 177 days in CSF, and 63 to 87 days in plasma.
 Toxicity ; Acute neurological impairment.
Inotersen
2/27/2024
Refrence ; Drug bank
11
 Used in the treatment of polyneuropathy caused by
hereditary transthyretin-mediated amyloidosis in
adults.
 It was FDA approved in October 2018.
 Dose 284 mg Subcutaneous.
 Food Interactions; Adm. of vitamin A supplement.
ADVANTAGES
2/27/2024
Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7
12
 Oligonucleotides are manufactured quickly (within a week).
 Sensitivity of therapy can be easily measured.
 Potential to produce longer lasting responses.
 Potential for enhanced binding affinity to target.
Challenges and LIMITATIONS
2/27/2024
Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7
13
 Off-Target Effects
 Unintended binding to non-target RNA sequences can lead to adverse effects or reduced
specificity.
 Delivery Efficiency
 Ensuring effective uptake and distribution of oligonucleotides to target tissues while minimizing
off-target accumulation.
APPLICATIONS
2/27/2024
Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7
14
 Inflammation therapeutics [Duchenne muscular dystrophy] (eteplirsen, golodirsen,
viltolarsen, and casimersen)
 CVS [Homozygous familial hypercholesterolemia] (mipomersen),
 Antiviral [Cytomegalovirus retinitis] (fomivirsen),
 Spinal muscular atrophy (nusinersen).
 Hereditary transthyretin amyloidosis (inotersen) and amyotrophic lateral sclerosis (tofersen).
References
2/27/2024
ISF college of pharmacy
15
 Chan JHP, Lim S, Wong WSF. Antisense oligonucleotides: from design to therapeutic
application. Clin Exp Pharmacol Physiol. 2006;33:533–40.
 Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus.
Nat Rev Drug Discov. 2021;20:427–53.
 Alama A, Barbieri F, Cagnoli M, Schettini G. Antisense oligonucleotides as therapeutic agents.
Pharmacol Res. 1997;36:171–8.
 https://www.researchgate.net/publication/377597137_Splice-
Modulating_Antisense_Oligonucleotides_as_Therapeutics_for_Inherited_Metabolic_Diseases
 https://link.springer.com/article/10.1007/s40259-024-00644-7
2/27/2024
ISF college of pharmacy

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Antisense oligonucleotides ppt.ppt ISF COLLEGE OF PARMACY

  • 1. TOPIC: ANTISENSE OLIGONUCLEOTIDE PRESENTED BY: Omkar Kumar Kuwar Department of Pharmacology ISF College of Pharmacy, MOGA omkar.official.955@gmail.com GUIDED BY: Dr. Arti Singh Associate Professor Department of Pharmacology ISF College of Pharmacy, MOGA 1
  • 2. CONTENTS 2/27/2024 ISF college of pharmacy 2  Introduction  Definition  Mechanism of ASO action  Advantages  Challenges & Limitations  Applications  Refrences
  • 3. INTRODUCTION to antisense oliGonucleotide 2/27/2024 Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7 3  Antisense : complementary or negative base sequence to a portion of the coding or positive sense mRNA.  Oligo : means scant or few  Nucleotide : 5 organic molecule that make up the backbone of the DNA & RNA. ( ATCGU)  Antisense oligonucleotide therapy is a new way to block protein function is to prevent the translation of protein by inhibiting translation step.  Antisense oligonucleotides (ASOs) are synthetic single stranded oligonucleotides that can bind to precursor mRNA (pre-mRNA) and/or mRNA through Watson-Crick base pairing.  ASOs can modulate the function of RNA, there by regulating gene expression.
  • 4. definition 2/27/2024 Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7 4  Oligonucleotides are chemically synthesized using phosphoramides.  The oligonucleotide chain proceeds in direction of 3’ to 5’ terminus.  Antisense RNA prevent protein translation of certain mRNA strands by binding to them.  Antisense DNA can be used to target a specific cRNA.
  • 7. Fomivirsensodium 2/27/2024 Refrence ; Drug bank 7  Fomivirsen is a 21-base phosphorothioate oligonucleotide.  Fomivirsen is marketed under the trade name Vitravene for intravitreal injection and was the first antisense drug to be approved by the Food and Drug Administration (FDA).  Indication Indicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS), • injected into the eye (intravitreal) 330 µg ( 0.05 mL/eye )  Half-life about 55-78 hours. CMV is a viral infection of the retina of the eye resulting if the eye inflammation.
  • 8. Mipomersen 2/27/2024 Refrence ; Drug bank 8  Familial hypercholesterolemia is a genetic disorder caused by a defect on chromosome-19, that makes the body unable to remove LDL. Or bad cholesterol this results in high level of LDL in blood.  The FDA label includes a black box warning of hepatoxicity.  Administer through subcutanious 200 mg/1mL  Half-life: 1-2 months.  Drug interaction: Acetaminophen, Amiodarone, Demeclocycline, Doxycycline, Eravacycline, They may increase the hepatotoxic activities of Mipomersen.  Food Interactions; Avoid excessive or chronic alcohol consumption. Alcohol may increase the risk of liver injury.
  • 9. Eteplirsen 2/27/2024 Refrence ; Drug bank 9  Used to treat Duchenne muscular dystrophy (DMD)  Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications.  It is caused by loss-of-function mutations in the DMD gene coding for dystrophin, an essential protein involved in maintaining the structural integrity and function of muscle fibres.  Approved by the FDA in September 2016.  Administer Intravenous strength 50 mg/mL  Half-life (t1/2) 3-4 h.
  • 10. Nusinersen 2/27/2024 Refrence ; Drug bank 10  Spinal muscular atrophy. It is a disorder affecting the motor neurons-nerves that control voluntary muscle movement.  It is administrated as direct 2.4 mg/1mL Intrathecal.  Half-life: 135 to 177 days in CSF, and 63 to 87 days in plasma.  Toxicity ; Acute neurological impairment.
  • 11. Inotersen 2/27/2024 Refrence ; Drug bank 11  Used in the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults.  It was FDA approved in October 2018.  Dose 284 mg Subcutaneous.  Food Interactions; Adm. of vitamin A supplement.
  • 12. ADVANTAGES 2/27/2024 Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7 12  Oligonucleotides are manufactured quickly (within a week).  Sensitivity of therapy can be easily measured.  Potential to produce longer lasting responses.  Potential for enhanced binding affinity to target.
  • 13. Challenges and LIMITATIONS 2/27/2024 Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7 13  Off-Target Effects  Unintended binding to non-target RNA sequences can lead to adverse effects or reduced specificity.  Delivery Efficiency  Ensuring effective uptake and distribution of oligonucleotides to target tissues while minimizing off-target accumulation.
  • 14. APPLICATIONS 2/27/2024 Refrencs; https://link.springer.com/article/10.1007/s40259-024-00644-7 14  Inflammation therapeutics [Duchenne muscular dystrophy] (eteplirsen, golodirsen, viltolarsen, and casimersen)  CVS [Homozygous familial hypercholesterolemia] (mipomersen),  Antiviral [Cytomegalovirus retinitis] (fomivirsen),  Spinal muscular atrophy (nusinersen).  Hereditary transthyretin amyloidosis (inotersen) and amyotrophic lateral sclerosis (tofersen).
  • 15. References 2/27/2024 ISF college of pharmacy 15  Chan JHP, Lim S, Wong WSF. Antisense oligonucleotides: from design to therapeutic application. Clin Exp Pharmacol Physiol. 2006;33:533–40.  Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021;20:427–53.  Alama A, Barbieri F, Cagnoli M, Schettini G. Antisense oligonucleotides as therapeutic agents. Pharmacol Res. 1997;36:171–8.  https://www.researchgate.net/publication/377597137_Splice- Modulating_Antisense_Oligonucleotides_as_Therapeutics_for_Inherited_Metabolic_Diseases  https://link.springer.com/article/10.1007/s40259-024-00644-7