Isis will make numerous presentations at the TIDES conference May 22-25, 2011 in Boston, MA. Here is the schedule and complete abstracts (Isis personnel will also participate in a number of forum discussion sessions - see the Isis website for complete information on these):
Sunday, May 22, 2011
8:30 a.m. E.T.
ION PAIR-HPLC-MS FOR QUALITY CONTROL OF OLIGONUCLEOTIDE THERAPEUTICS
Claus Rentel, Ph.D., Director, Analytical Development and Quality Control, Isis
Pharmaceuticals, Inc.
Oligonucleotide drugs contain many structural very similar impurities which are difficult or impossible to detect by most analytical techniques. We have developed an LC-MS based QC method determining assay, identity and impurity profile of oligonucleotides. This presentation will provide an overview of the method featuring advantages regarding identification and quantitation of unknown and coeluting impurities, accuracy and limits of detection.
9:10 a.m. E.T.
THE LEARNING CURVE: EXPERIENCES IN RUNNING ISIS’ ION PAIR-HPLC-MS METHOD
Cynthia T. Sanderson, Ph.D., Research Scientist, PPD
This presentation will include lessons learned and practical advice on implementing and running Ion-Pair HPLC-MS for oligonucleotides. Topics will include critical factors as well as tips for successful implementation and trouble shooting of this technology. The presenter will draw from experiences in setting up and qualifying a dedicated LC-MS system, method transfers and method development.
9:40 a.m. E.T.
IMPACT OF MASS SPECTROMETER DESIGN ON ION PAIRHPLC-MS ANALYSIS OF OLIGONUCLEOTIDES
Claus Rentel, Ph.D., Director, Analytical Development and Quality Control, Isis
Pharmaceuticals, Inc.
Two HPLC systems equipped with Diode Array and single quadrupole MS Detectors
(Agilent and Waters) were evaluated for routine QC use release and stability studies of a Phophorothioate oligonucleotide. It will be shown, that the design of the ion source has a major impact on generating meaningful and reproducible results.
Monday, May 23, 2011
Time 2:00 p.m. ET
CLINICAL DEVELOPMENT OF MIPOMERSEN, AN ANTISENSE SECOND-GENERATION OLIGONUCLEOTIDE TARGETING APOLIPOPROTEIN B
Tejdip Singh, M.D., Medical Director, Global Patient Safety and Risk Management,
Genzyme Corporation
This presentation will provide an overview of the clinical development of mipomersen, an antisense second-generation oligonucleotide being investigated for patients with severely elevated cholesterol such as those with familial hypercholesterolemia, including patients with homozygous and severe heterozygous familial hypercholesterolemia. Mipomersen targets hepatic production of apolipoprotein B (Apo B), the structural core for particles such as LDL that carry cholesterol.
Tuesday, May 24, 2011
Time 9:00 a.m. ET
EFFECTIVE DELIVERY OF MICRORNA THERAPEUTICS
Balkrishen Bhat, Ph.D., Senior Director Chemistry, Regulus Therapeutics
MicroRNAs are small non-coding RNAs that regulate major biological functions in mammalian systems. Dysregulation of microRNAs can cause human pathologies such as cancer and inflammatory diseases. Regulus Therapeutics is taking a systematic approach to developing microRNA drugs with clinical grade properties. Here we present data on delivery and distribution of microRNA modulators after systemic or local delivery. A number of case examples including let-7, miR-122, and miR-34a will be presented to highlight the progress.
9:30 a.m. ET
CHARACTERIZATION OF FUNCTIONAL ANTISENSE OLIGONUCLEOTIDES UPTAKE INTO LIVER CELLS
Erich Koller, Ph.D., Assistant Director, Core Research, Isis Pharmaceuticals, Inc.
Most mammalian cell lines take up modified antisense oligonucleotides (ASOs) when added to culture media. However, it has been difficult to demonstrate antisense effects without the aid of transfection agents. We have isolated a mouse 3 hepatocellular carcinoma cell line that maintains the ability to take up ASOs. We demonstrate that functional uptake is blocked by inhibitors of the adapter protein AP2M1, but not clathrin or caveolin
2:00 p.m. ET
DEVELOPMENT OF 2’-MOE ANTISENSE OLIGONUCLEOTIDES AS A NOVEL APPROACH TO TREAT FIBROSIS AND SKIN SCARRING
Nicholas M. Dean, Ph.D., Chief Scientific Officer, Excaliard Pharmaceuticals, Inc.
Excaliard Pharmaceuticals is developing 2’MOE modified ASOs as novel therapeutics to treat scarring. The load ASO (EXC 001) inhibits expression of CTGF. The CMC issues encountered & the solutions developed will be presented, along with results from multiple Phase 1 and Phase 2 studies.
Sunday, May 22, 2011
8:30 a.m. E.T.
ION PAIR-HPLC-MS FOR QUALITY CONTROL OF OLIGONUCLEOTIDE THERAPEUTICS
Claus Rentel, Ph.D., Director, Analytical Development and Quality Control, Isis
Pharmaceuticals, Inc.
Oligonucleotide drugs contain many structural very similar impurities which are difficult or impossible to detect by most analytical techniques. We have developed an LC-MS based QC method determining assay, identity and impurity profile of oligonucleotides. This presentation will provide an overview of the method featuring advantages regarding identification and quantitation of unknown and coeluting impurities, accuracy and limits of detection.
9:10 a.m. E.T.
THE LEARNING CURVE: EXPERIENCES IN RUNNING ISIS’ ION PAIR-HPLC-MS METHOD
Cynthia T. Sanderson, Ph.D., Research Scientist, PPD
This presentation will include lessons learned and practical advice on implementing and running Ion-Pair HPLC-MS for oligonucleotides. Topics will include critical factors as well as tips for successful implementation and trouble shooting of this technology. The presenter will draw from experiences in setting up and qualifying a dedicated LC-MS system, method transfers and method development.
9:40 a.m. E.T.
IMPACT OF MASS SPECTROMETER DESIGN ON ION PAIRHPLC-MS ANALYSIS OF OLIGONUCLEOTIDES
Claus Rentel, Ph.D., Director, Analytical Development and Quality Control, Isis
Pharmaceuticals, Inc.
Two HPLC systems equipped with Diode Array and single quadrupole MS Detectors
(Agilent and Waters) were evaluated for routine QC use release and stability studies of a Phophorothioate oligonucleotide. It will be shown, that the design of the ion source has a major impact on generating meaningful and reproducible results.
Monday, May 23, 2011
Time 2:00 p.m. ET
CLINICAL DEVELOPMENT OF MIPOMERSEN, AN ANTISENSE SECOND-GENERATION OLIGONUCLEOTIDE TARGETING APOLIPOPROTEIN B
Tejdip Singh, M.D., Medical Director, Global Patient Safety and Risk Management,
Genzyme Corporation
This presentation will provide an overview of the clinical development of mipomersen, an antisense second-generation oligonucleotide being investigated for patients with severely elevated cholesterol such as those with familial hypercholesterolemia, including patients with homozygous and severe heterozygous familial hypercholesterolemia. Mipomersen targets hepatic production of apolipoprotein B (Apo B), the structural core for particles such as LDL that carry cholesterol.
Tuesday, May 24, 2011
Time 9:00 a.m. ET
EFFECTIVE DELIVERY OF MICRORNA THERAPEUTICS
Balkrishen Bhat, Ph.D., Senior Director Chemistry, Regulus Therapeutics
MicroRNAs are small non-coding RNAs that regulate major biological functions in mammalian systems. Dysregulation of microRNAs can cause human pathologies such as cancer and inflammatory diseases. Regulus Therapeutics is taking a systematic approach to developing microRNA drugs with clinical grade properties. Here we present data on delivery and distribution of microRNA modulators after systemic or local delivery. A number of case examples including let-7, miR-122, and miR-34a will be presented to highlight the progress.
9:30 a.m. ET
CHARACTERIZATION OF FUNCTIONAL ANTISENSE OLIGONUCLEOTIDES UPTAKE INTO LIVER CELLS
Erich Koller, Ph.D., Assistant Director, Core Research, Isis Pharmaceuticals, Inc.
Most mammalian cell lines take up modified antisense oligonucleotides (ASOs) when added to culture media. However, it has been difficult to demonstrate antisense effects without the aid of transfection agents. We have isolated a mouse 3 hepatocellular carcinoma cell line that maintains the ability to take up ASOs. We demonstrate that functional uptake is blocked by inhibitors of the adapter protein AP2M1, but not clathrin or caveolin
2:00 p.m. ET
DEVELOPMENT OF 2’-MOE ANTISENSE OLIGONUCLEOTIDES AS A NOVEL APPROACH TO TREAT FIBROSIS AND SKIN SCARRING
Nicholas M. Dean, Ph.D., Chief Scientific Officer, Excaliard Pharmaceuticals, Inc.
Excaliard Pharmaceuticals is developing 2’MOE modified ASOs as novel therapeutics to treat scarring. The load ASO (EXC 001) inhibits expression of CTGF. The CMC issues encountered & the solutions developed will be presented, along with results from multiple Phase 1 and Phase 2 studies.
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