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Oligonucleotide Therapeutics:
Brief Overview of the State of the Market
The Promise and Opportunities
• Oligonucleotides offer promising treatment for a wide range of medical conditions
• Abilit...
Early Challenges
• Modern age of oligonucleotides and the birth of Antisense Oligonucleotide (ASO) work began in
the early...
Enabling Technologies
• Reversed-phase (RP) separations has facilitated reasonable mass spectroscopy (MS) sensitivity
• Ma...
Drug Delivery & Toxicology Challenges
• Delivering active oligonucleotide to correct sites within target cells of target t...
Regulatory Complexity
• Oligonucleotides are chemically synthesized are manufactured in ways similar to
other small-molecu...
The Pipeline
• FDA has approved three oligonucleotide drugs
• Currently, there are 135 oligonucleotide clinical trials lis...
BioTechLogic’s Oligonucleotide Experience
BioTechLogic has done a great deal of working with oligonucleotide products in r...
717 Indian Road
Glenview, Illinois 60025
Phone: 847-730-3475
Fax: 847-730-3498
www.biotechlogic.com
Contact: Peter Dellva,...
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Oligonucleotide Therapeutics: Brief Overview of the State of the Market

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Presentation discusses the history, future and current state of the oligonucleotide therapeutics arena.

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Oligonucleotide Therapeutics: Brief Overview of the State of the Market

  1. 1. Oligonucleotide Therapeutics: Brief Overview of the State of the Market
  2. 2. The Promise and Opportunities • Oligonucleotides offer promising treatment for a wide range of medical conditions • Ability to affect protein targets that cannot be effectively treated by small-molecule or protein therapeutics • Specific malfunctioning genes can be targeted, manipulated, silenced and/or modulated • Immune system modification is possible • Scaling up for commercial-scale GMP production is more feasible than it is for many cell therapies or other biologic therapies • Side effects are often more manageable than other classes of drugs
  3. 3. Early Challenges • Modern age of oligonucleotides and the birth of Antisense Oligonucleotide (ASO) work began in the early 1970s after Nobel laureate Gobind Khorana published his ground-breaking work • Despite early progress, Antisense Oligonucleotide (ASO) therapeutics as a promising set of entities for commercialization began in the early 1990s • Limited synthesis methods sharply limited the amount of available drug substance and caused supply chain delays • Analytical methods were not well developed and analytical instrumentation technology was often not advanced enough to support the needs of the market
  4. 4. Enabling Technologies • Reversed-phase (RP) separations has facilitated reasonable mass spectroscopy (MS) sensitivity • Market leaders have dedicated significant resources toward improving manufacturing efficiency and capacity increasing quantities of drug substance “By developing several proprietary chemical processes to scale up our manufacturing capabilities, we have greatly reduced the cost of producing oligonucleotide drugs. For example, we have significantly reduced the cost of raw materials through improved yield efficiency, while at the same time increasing our capacity to make the drugs. Through both our internal research and development programs and collaborations with outside vendors, we may achieve even greater efficiency and further cost reductions,” – -Stanley T. Crooke, M.D., Ph.D., Chairman, Chief Executive Officer and President, Ionis Pharmaceuticals (then Isis Pharmaceuticals) in its 2014 annual report •
  5. 5. Drug Delivery & Toxicology Challenges • Delivering active oligonucleotide to correct sites within target cells of target tissues in challenging • Nucleic acid molecules are charged and larger than traditional small molecules, so productive uptake into target organs and cells is often difficult • Off-target impacts have also been a historical and ongoing concern - progress has and is continuing to be made in this area
  6. 6. Regulatory Complexity • Oligonucleotides are chemically synthesized are manufactured in ways similar to other small-molecule drugs • However, they interact in vivo in a manner more typical of biologic therapies • FDA has released recommendations for approaching synthetic oligonucleotide drug substance and drug product approval—“Points To Consider For The Submission of Chemistry, Manufacturing, and Controls (CMC) Information in Oligonucleotide-Based Therapeutic Drug Applications.” • FDA and EMA disagree on how Oligonucleotide therapeutics should be classified – FDA regulates as a small molecule drug, EMA regulates oligonucleotides with similar mechanisms under which biologics are regulated
  7. 7. The Pipeline • FDA has approved three oligonucleotide drugs • Currently, there are 135 oligonucleotide clinical trials listed on ClinicalTrials.gov registered by more than 30 companies • Of the 135, 37 are open studies either actively recruiting, active but not recruiting or active and enrollment is by invitation only • Many of registered clinical trials using antisense or siRNAs are for treatment of different forms of cancer. However, there are other conditions being addressed, including conditions affecting the eyes and liver, Duchenne Muscular Dystrophy, HIV, Mesothelioma, Rheumatoid Arthritis and many more
  8. 8. BioTechLogic’s Oligonucleotide Experience BioTechLogic has done a great deal of working with oligonucleotide products in recent years, including process validation work for drug substance, drug product and an oligo adjuvant • CMO on-site support • Equipment qualification protocols • Commercial-ready batch records • Support validation protocols and studies • Process validation • Manufacturing support • Technical support • Facility validation reports • Microbial monitoring strategies • Commercialization plans • Formulation development reports • Process control strategies • Chromatography column troubleshooting • Multiproduct facility CV site policies and strategies
  9. 9. 717 Indian Road Glenview, Illinois 60025 Phone: 847-730-3475 Fax: 847-730-3498 www.biotechlogic.com Contact: Peter Dellva, Head of Business and Finance PD@biotechlogic.com

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