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February 26-28, 2013 -
February 25 - March 01, 2013
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TIDES: Oligonucleotide and Peptide® Therapeutics from Research through Commercialization
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Oligonucleotide Therapeutics Discovery
- Agenda summary: Go to the agenda summary to view the detailed agenda for each track, course, keynote or plenary session.
- To view the complete agenda: Please download the PDF brochure.
Oligonucleotide Therapeutics Discovery
Courses: Sunday | Plenary Sessions: Monday | Main Conference: Tuesday | Wednesday
Monday keynote and plenary sessions are shared across all tracks. Please click on the respective links below for the full Monday agenda.
7:30
Registration and Coffee
12:50
Lunch on your own
5:30
Networking, Wine and Cheese Reception in Poster and Exhibit Hall
Courses: Sunday | Plenary Sessions: Monday | Main Conference: Tuesday | Wednesday
7:15
Registration and Coffee
8:15
Chairman's Remarks
Erich Koller, Ph.D., Assistant Director Core Research, Isis Pharmaceuticals, Inc.
Erich Koller, Ph.D., Assistant Director Core Research, Isis Pharmaceuticals, Inc.
Single Stranded RNAs as Novel Active Compounds
8:30
Chemically Modified Single Stranded RNA Activates RNA Interference in Animals
We have done an extensive chemical structure activity relationship (SAR) study of single stranded RNAs (ss-siRNAs) and coupled this to biochemical studies on the mechanism of activation of the RNA induced silencing complex (RISC). We identified ss-siRNAs as active in animals. The details of these results are presented.
Thazha P. Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals
We have done an extensive chemical structure activity relationship (SAR) study of single stranded RNAs (ss-siRNAs) and coupled this to biochemical studies on the mechanism of activation of the RNA induced silencing complex (RISC). We identified ss-siRNAs as active in animals. The details of these results are presented.
Thazha P. Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals
9:00
Targeting Long Non-coding RNA Interactions with PRC2 to Activate Single Gene Expression
Histone H3K27 trimethylation is generated by Polycomb repressive complex 2 (PRC2) and represses transcription. Our platform is based on the discovery that PRC2 is recruited locally by non-coding RNAs. We have identified the genome-wide pool of RNA sequences that interact with PRC2. Short, single-stranded oligonucleotides can sterically block PRC2 recruitment to a single lncRNA, thereby de-repressing the transcription of individual genes. This technology can be utilized to selectively upregulate gene expression for therapeutic benefit.
James Barsoum, Ph.D., Chief Scientific Officer, RaNA Therapeutics
Histone H3K27 trimethylation is generated by Polycomb repressive complex 2 (PRC2) and represses transcription. Our platform is based on the discovery that PRC2 is recruited locally by non-coding RNAs. We have identified the genome-wide pool of RNA sequences that interact with PRC2. Short, single-stranded oligonucleotides can sterically block PRC2 recruitment to a single lncRNA, thereby de-repressing the transcription of individual genes. This technology can be utilized to selectively upregulate gene expression for therapeutic benefit.
James Barsoum, Ph.D., Chief Scientific Officer, RaNA Therapeutics
Novel Chemistries and PK/PD Modulation
9:30
Automated Parallel Solid-phase Synthesis of 5'-triphosphate and 5'-diphosphate Oligonucleotides - Useful Tools for Therapeutics and Diagnostics Applications
A fully automated method for the parallel and high-throughput synthesis of 5'-triphosphate and 5'-diphosphate oligonucleotides is presented. The desired full-length oligonucleotides were constructed using standard DNA/RNA synthesis. Then, an uninterrupted sequential cycle afforded the unmodified or chemically modified 5'-triphosphates and 5'-diphosphates, useful tools in number of therapeutics and diagnostics applications.
Ivan Zlatev, Ph.D., Scientist, Ontorii Inc. (This presentation will be based on work performed at Alnylam Pharmaceuticals)
A fully automated method for the parallel and high-throughput synthesis of 5'-triphosphate and 5'-diphosphate oligonucleotides is presented. The desired full-length oligonucleotides were constructed using standard DNA/RNA synthesis. Then, an uninterrupted sequential cycle afforded the unmodified or chemically modified 5'-triphosphates and 5'-diphosphates, useful tools in number of therapeutics and diagnostics applications.
Ivan Zlatev, Ph.D., Scientist, Ontorii Inc. (This presentation will be based on work performed at Alnylam Pharmaceuticals)
Oligonucleotide Delivery: Mechanisms and Emerging Strategies
10:00
Physical Characterization of Lipid Nanoparticles for Efficient Delivery of Dicer Susbtrate siRNAs to Tumors
Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals
Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals
10:30
Networking Refreshment Break in Poster and Exhibit Hall
11:15
Delivering on the Promise of Therapeutic Oligos: An ADME Perspective
As more oligos reach late stage clinical trials, the combination of narrow therapeutic index and significant cost of goods is increasing the pressure to understand and develop strategies for improving productive delivery. This presentation reflects on these efforts and introduces a new European IMI initiative, COMPACT, that intends to address this challenge.
Steve Hood, Ph.D., Director, GSK Oligonucleotide Delivery (GOLD) Team, GlaxoSmithKline, United Kingdom
As more oligos reach late stage clinical trials, the combination of narrow therapeutic index and significant cost of goods is increasing the pressure to understand and develop strategies for improving productive delivery. This presentation reflects on these efforts and introduces a new European IMI initiative, COMPACT, that intends to address this challenge.
Steve Hood, Ph.D., Director, GSK Oligonucleotide Delivery (GOLD) Team, GlaxoSmithKline, United Kingdom
11:45
Mechanisms of Single-stranded Phosphorothioate Modified Antisense Oligonucleotide Accumulation in Hepatocytes
Single stranded antisense oligonucleotides (SSOs) are used to modulate the expression of genes in animal models and are being investigated as human therapeutics. The mechanisms through which these antisense drugs accumulate in tissues and gain access to their target RNAs is not well understood. Results are presented, elucidating the roles of various genes and pathways on SSO uptake into cells.
Erich Koller, Ph.D., Assistant Director Core Research, Isis Pharmaceuticals, Inc.
Single stranded antisense oligonucleotides (SSOs) are used to modulate the expression of genes in animal models and are being investigated as human therapeutics. The mechanisms through which these antisense drugs accumulate in tissues and gain access to their target RNAs is not well understood. Results are presented, elucidating the roles of various genes and pathways on SSO uptake into cells.
Erich Koller, Ph.D., Assistant Director Core Research, Isis Pharmaceuticals, Inc.
12:15
RNAi Therapeutics as Personalized Medicine for the Treatment of Hepatocellular Carcinoma
β-catenin and c-MET have been associated with the development of hepatocellular carcinoma. Using our SMARTT Polymer Technology®, siRNAs can be effectively delivered to HCC cells. Polymers formulated with β-catenin and c-MET siRNAs were evaluated in a HCC mouse model. Dose-dependent mRNA and protein KD following a single dose were observed in tumors in vivo. Repeat dose studies have shown potent anti-tumor efficacy as well as increased survival with good tolerability.
Mary Prieve, Ph.D., Director of Biology, PhaseRx, Inc.
β-catenin and c-MET have been associated with the development of hepatocellular carcinoma. Using our SMARTT Polymer Technology®, siRNAs can be effectively delivered to HCC cells. Polymers formulated with β-catenin and c-MET siRNAs were evaluated in a HCC mouse model. Dose-dependent mRNA and protein KD following a single dose were observed in tumors in vivo. Repeat dose studies have shown potent anti-tumor efficacy as well as increased survival with good tolerability.
Mary Prieve, Ph.D., Director of Biology, PhaseRx, Inc.
12:45
Networking Luncheon in Poster and Exhibit Hall
1:55
Chairman's Remarks
Martin Maier, Ph.D., Senior Director, RNA Synthesis, Alnylam Pharmaceuticals
Martin Maier, Ph.D., Senior Director, RNA Synthesis, Alnylam Pharmaceuticals
Technologies for Cell and Tissue Targeting or Improved Delivery
2:00
Lipid Nanoparticle siRNA Systems: Gene Silencing in Non-liver Tissues
Lipid nanoparticle (LNP) systems containing siRNA have shown clinical utility for silencing genes in liver (hepatocytes) following i.v. administration. LNP siRNA systems for gene silencing in non-liver tissues requires incorporation of features to avoid uptake into the liver as well as targeting agents to facilitate uptake into target cells. This talk outlines progress made to develop LNP siRNA systems for silencing genes in tissues such as immune cells, bone marrow and brain.
Pieter R. Cullis, Professor and Director, Nanomedicines Research Group, Department of Biochemistry and Molecular Biology, University of British Columbia, Canada
Lipid nanoparticle (LNP) systems containing siRNA have shown clinical utility for silencing genes in liver (hepatocytes) following i.v. administration. LNP siRNA systems for gene silencing in non-liver tissues requires incorporation of features to avoid uptake into the liver as well as targeting agents to facilitate uptake into target cells. This talk outlines progress made to develop LNP siRNA systems for silencing genes in tissues such as immune cells, bone marrow and brain.
Pieter R. Cullis, Professor and Director, Nanomedicines Research Group, Department of Biochemistry and Molecular Biology, University of British Columbia, Canada
2:30
Combinatorial Development of Synthetic siRNA Delivery Systems
High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing siRNA delivery systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery siRNA, both in vitro and in vivo. A number of siRNA delivery formulations have been developed with in vivo efficacy and show potential therapeutic application for the treatment of genetic disease, viral infection and cancer.
Daniel G. Anderson, Ph.D., Associate Professor Chemical Engineering, Koch Institute, Massachusetts Institute of Technology
High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing siRNA delivery systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery siRNA, both in vitro and in vivo. A number of siRNA delivery formulations have been developed with in vivo efficacy and show potential therapeutic application for the treatment of genetic disease, viral infection and cancer.
Daniel G. Anderson, Ph.D., Associate Professor Chemical Engineering, Koch Institute, Massachusetts Institute of Technology
3:00
Development of DPC Technology for TherapeuticsiRNA Delivery
Dynamic Polyconjugate (DPC) technology is a targeted, polymer-based siRNA delivery platform. Key to this technology are environmentally-sensitive polymer masking chemistries which enable activation of the polymer only after reaching the target cell. This enables greatly improved polymer circulation times and targetability, as well as a wider safety margins. We are currently developing DPC technology for use in an RNAi-based treatment for chronic Hepatitis B virus infection.
David Lewis, Ph.D., Vice President, Biology, Arrowhead Research Corporation
Dynamic Polyconjugate (DPC) technology is a targeted, polymer-based siRNA delivery platform. Key to this technology are environmentally-sensitive polymer masking chemistries which enable activation of the polymer only after reaching the target cell. This enables greatly improved polymer circulation times and targetability, as well as a wider safety margins. We are currently developing DPC technology for use in an RNAi-based treatment for chronic Hepatitis B virus infection.
David Lewis, Ph.D., Vice President, Biology, Arrowhead Research Corporation
3:30
Networking Refreshment Break in Poster and Exhibit Hall
Panel Discussion
4:15
What Does it Take to Bring an Oligonucleotide to Market?
Arthur A. Levin, Ph.D., Executive Vice President, R&D, Miragen Therapeutics
Panelists:
Amy Lee, Director, Research, Tekmira Pharmaceuticals
Rachel Meyers, Ph.D., Vice President Research and RNAi Lead Development, Alnylam Pharmaceuticals
Hans-Peter Vornlocher, Ph.D., Managing Director Research, Axolabs GmbH, Germany
- Phase-specific considerations from discovery to clinic and GMP manufacturing
- Lessons learned from clinical programs to date
- The holy grail: solving the oligonucleotide delivery problem
- Regulatory considerations - the transition from good idea to safe dosing in man
Arthur A. Levin, Ph.D., Executive Vice President, R&D, Miragen Therapeutics
Panelists:
Amy Lee, Director, Research, Tekmira Pharmaceuticals
Rachel Meyers, Ph.D., Vice President Research and RNAi Lead Development, Alnylam Pharmaceuticals
Hans-Peter Vornlocher, Ph.D., Managing Director Research, Axolabs GmbH, Germany
5:15
Networking, Wine and Cheese Reception in Poster and Exhibit Hall
Dedicated Poster Viewing - Poster presenters will be at their posters to discuss results with you. Sponsored by
Dedicated Poster Viewing - Poster presenters will be at their posters to discuss results with you. Sponsored by
Courses: Sunday | Plenary Sessions: Monday | Main Conference: Tuesday | Wednesday
7:30
Coffee
Featured Presentation
Unpublished Data
8:00
Tackling RNAi Delivery with Bioreversible PhosphoTriester siRNN ProdrugsIn the world of RNAi therapeutics, siRNA delivery remains the technological problem to solve. We pioneered a RNAi delivery technology that radically shrinks the size to the smallest possible, self-delivering monomeric RNAi molecule by synthesis of neutral bioreversible phosphotriester siRNN prodrugs that are converted by intracellular restricted enzymes into charged phosphodiester siRNAs.
Steven F. Dowdy, Ph.D., Professor, Cellular and Molecular Medicine, UCSD School of Medicine
8:25
Chairman's Remarks
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark
Discovery and Preclinical Strategies
8:30
Novel Chemical Modifications for siRNAs
Although several well-tolerated chemical modifications for short interfering RNAs (siRNAs) have been identified, little is known about the positional tolerance of different chemical modifications and how various chemistries can effectively be combined for the design of potent siRNAs with improved properties. To further elucidate the design principles for chemically modified siRNAs, we systematically studied the position-dependent effect of various novel chemical modifications on siRNA potency.
Martin Maier, Ph.D., Senior Director, Drug Discovery, Alnylam Pharmaceuticals
Although several well-tolerated chemical modifications for short interfering RNAs (siRNAs) have been identified, little is known about the positional tolerance of different chemical modifications and how various chemistries can effectively be combined for the design of potent siRNAs with improved properties. To further elucidate the design principles for chemically modified siRNAs, we systematically studied the position-dependent effect of various novel chemical modifications on siRNA potency.
Martin Maier, Ph.D., Senior Director, Drug Discovery, Alnylam Pharmaceuticals
9:00
Efficacy, PK and Tox Data Supporting the Move to IND for Mirna Therapeutics
miR-34 is a tumor suppressor miRNA that indices cell cycle arrest and apoptosis in cancer cells by regulating the expression of more than twenty oncogenes, including MET, MYC, WNT and MYB. We developed a liposome-formulated mimic of miR-34 that causes complete tumor regression in orthotopic models of liver cancer at dose levels that are 100-fold below the no adverse effects level that was observed in GLP tox studies in non-human primates and rats. Clinical studies scheduled to begin in the second quarter of 2013 will provide the first measure of the therapeutic potential of a tumor suppressor miRNA.
David Brown, Ph.D., Director of Discovery, Mirna Therapeutics
miR-34 is a tumor suppressor miRNA that indices cell cycle arrest and apoptosis in cancer cells by regulating the expression of more than twenty oncogenes, including MET, MYC, WNT and MYB. We developed a liposome-formulated mimic of miR-34 that causes complete tumor regression in orthotopic models of liver cancer at dose levels that are 100-fold below the no adverse effects level that was observed in GLP tox studies in non-human primates and rats. Clinical studies scheduled to begin in the second quarter of 2013 will provide the first measure of the therapeutic potential of a tumor suppressor miRNA.
David Brown, Ph.D., Director of Discovery, Mirna Therapeutics
9:30
Nucleic Acid Polymers and Their Application in Hepatitis B Infection (HBV): Efficacy and Safety (9:40)
Nucleic acid polymers (NAPs) utilize the sequence independent interaction of PS-ONs with apolipoproteins to block HBsAg-release into the blood. A second generation NAP (REP 9AC') clears the blood of HBsAg in HBV-infected patients which dramatically improves the efficacy of immunotherapy to establish durable immunological control of HBV infection off treatment in most patients. The safety profile of NAPs are presented to demonstrate the toxicological properties of PS-ONs which do not target protein expression.
Andrew Vaillant, Ph.D., Vice President and Chief Scientific Officer, REPLICor Inc., Canada
Nucleic acid polymers (NAPs) utilize the sequence independent interaction of PS-ONs with apolipoproteins to block HBsAg-release into the blood. A second generation NAP (REP 9AC') clears the blood of HBsAg in HBV-infected patients which dramatically improves the efficacy of immunotherapy to establish durable immunological control of HBV infection off treatment in most patients. The safety profile of NAPs are presented to demonstrate the toxicological properties of PS-ONs which do not target protein expression.
Andrew Vaillant, Ph.D., Vice President and Chief Scientific Officer, REPLICor Inc., Canada
10:00
Therapeutic Opportunities for Interferon Regulatory Factor 5 (IRF5) Antisense Inhibitors in Systemic Lupus Erythematosus (SLE)
Recent data supports IFNa as a major driver of SLE pathology. IRF5 is known to induce transcription of IFNa and other immune genes downstream of TLR activation. Interestingly, genetic variants in the IRF5 gene have been associated with SLE susceptibility and increased levels of IFNa. We have evaluated antisense oligonuleotides (ASOs) targeting IRF5 in several murine SLE models and observed significant improvement of pathology. We suggest that IRF5 ASOs can be considered as an attractive approach to treat SLE.
Alexey Revenko, Ph.D., Senior Scientist, Isis Pharmaceuticals
Recent data supports IFNa as a major driver of SLE pathology. IRF5 is known to induce transcription of IFNa and other immune genes downstream of TLR activation. Interestingly, genetic variants in the IRF5 gene have been associated with SLE susceptibility and increased levels of IFNa. We have evaluated antisense oligonuleotides (ASOs) targeting IRF5 in several murine SLE models and observed significant improvement of pathology. We suggest that IRF5 ASOs can be considered as an attractive approach to treat SLE.
Alexey Revenko, Ph.D., Senior Scientist, Isis Pharmaceuticals
10:30
Networking Refreshment Break in Poster and Exhibit Hall
11:15
Structure Activity Determinants of Locked Nucleic Acids: Platform, Discovery, Clinic
LNA is based on a bicyclic chemical structure and is an efficient platform for inhibiting coding and non-coding RNA. The differentiated properties of LNAs cannot be understood from a reductionist view point, i.e. nucleobase sequence determinants - alone. Up-take and structure activity relationships are illustrated and possible underlying mechanisms determining compound individuality are discussed. The latest pre-clinical/clinical data on Miravirsen is highlighted. MicroRNA-122 inhibition by Miravirsen leads to efficient HCV titer reductions in infected individuals.
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark
LNA is based on a bicyclic chemical structure and is an efficient platform for inhibiting coding and non-coding RNA. The differentiated properties of LNAs cannot be understood from a reductionist view point, i.e. nucleobase sequence determinants - alone. Up-take and structure activity relationships are illustrated and possible underlying mechanisms determining compound individuality are discussed. The latest pre-clinical/clinical data on Miravirsen is highlighted. MicroRNA-122 inhibition by Miravirsen leads to efficient HCV titer reductions in infected individuals.
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark
11:45
Intervening in Complex Disease Biology with microRNA Targeting Therapeutics
The abstract is unavailable at this time.
William S. Marshall, Ph.D., President and Chief Executive Officer, miRagen Therapeutics
The abstract is unavailable at this time.
William S. Marshall, Ph.D., President and Chief Executive Officer, miRagen Therapeutics
12:15
Targeting microRNAs and Biological Networks: An Innovative Approach to Treat Disease
The abstract is unavailable at this time.
Neil Gibson, Ph.D., Chief Scientific Officer, Regulus Therapeutics
The abstract is unavailable at this time.
Neil Gibson, Ph.D., Chief Scientific Officer, Regulus Therapeutics
12:45
Networking Luncheon in Poster and Exhibit Hall
1:55
Chairman's Remarks
Bo Rode Hansen, Vice President, Santaris Pharma A/S, Denmark
Bo Rode Hansen, Vice President, Santaris Pharma A/S, Denmark
Clinical Progress of Oligonucleotide Therapeutics
2:15
Update on Alnylam Clinical Programs
This presentation reviews the results of Alnylam's clinical programs that have established proof of concept for RNAi therapeutics in man.
Jared Gollob, M.D., Vice President, Clinical Research, Alnylam Pharmaceuticals, Inc.
This presentation reviews the results of Alnylam's clinical programs that have established proof of concept for RNAi therapeutics in man.
Jared Gollob, M.D., Vice President, Clinical Research, Alnylam Pharmaceuticals, Inc.
2:45
Inhibition of Toll-like Receptors as a Strategy for Treating Autoimmune Diseases
Endosomal Toll-like receptors (TLRs) 7, 8 and 9 are implicated in certain autoimmune diseases, including lupus, psoriasis and arthritis. Immune complexes containing self-nucleic acids have shown to initiate immune cascades through these TLRs. We have designed and synthesized oligonucleotide-based antagonists of TLRs 7, 8 and 9, which have shown potent activity by inhibiting Th1, Th17 and inflammasome pathway in preclinical disease models of lupus and psoriasis. IMO-3100, an antagonist of TLRs 7 and 9, is in Phase 2 trial in patients with psoriasis. IMO-8400, an antagonist of TLRs 7, 8 and 9, is in Phase 1 trial and its initial indication is lupus.
Sudhir Agrawal, CEO, Idera Pharmaceuticals, Inc.
Endosomal Toll-like receptors (TLRs) 7, 8 and 9 are implicated in certain autoimmune diseases, including lupus, psoriasis and arthritis. Immune complexes containing self-nucleic acids have shown to initiate immune cascades through these TLRs. We have designed and synthesized oligonucleotide-based antagonists of TLRs 7, 8 and 9, which have shown potent activity by inhibiting Th1, Th17 and inflammasome pathway in preclinical disease models of lupus and psoriasis. IMO-3100, an antagonist of TLRs 7 and 9, is in Phase 2 trial in patients with psoriasis. IMO-8400, an antagonist of TLRs 7, 8 and 9, is in Phase 1 trial and its initial indication is lupus.
Sudhir Agrawal, CEO, Idera Pharmaceuticals, Inc.
3:15
Networking Refreshment Break
3:45
Clinical Development of RXI-109 to Reduce Dermal Scarring
RXI-109 is a 'self-delivering' or sd-rxRNA® that targets a key regulator of fibrosis and scar formation (connective tissue growth factor) and is initially being developed to reduce dermal scarring following surgery. Preliminary safety and tolerability data from clinical trials of intradermal administration of RXI-109 at small incision sites are reviewed. Development plans include Phase 2 clinical investigation of RXI-109 in scar revision surgeries and continued research to support broader use as an anti-fibrotic agent.
Pamela Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals
RXI-109 is a 'self-delivering' or sd-rxRNA® that targets a key regulator of fibrosis and scar formation (connective tissue growth factor) and is initially being developed to reduce dermal scarring following surgery. Preliminary safety and tolerability data from clinical trials of intradermal administration of RXI-109 at small incision sites are reviewed. Development plans include Phase 2 clinical investigation of RXI-109 in scar revision surgeries and continued research to support broader use as an anti-fibrotic agent.
Pamela Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals
4:15
First Clinical Phase IIa Data of the Mirror-Image RNA Oligonucleotide NOX-A12
NOX-A12 is a functional mirror-image RNA oligonucleotide (a so-called Spiegelmer) that binds and inhibits the pro-angiogenic chemokine and homing factor SDF-1 (stromal cell-derived factor-1). SDF-1 is particularly involved in homing processes of hematopoietic stem cells as well as leukemic stem cells. After a broad characterization of NOX-A12 in preclinical models, Phase I studies demonstrated that the neutralization of SDF-1 leads to the mobilization of stem cells in healthy volunteers. Currently, NOX-A12 is evaluated in an open label study in patients suffering from chronic lymphatic leukemia (CLL) and multiple myeloma (MM) in combination with chemotherapy.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany
NOX-A12 is a functional mirror-image RNA oligonucleotide (a so-called Spiegelmer) that binds and inhibits the pro-angiogenic chemokine and homing factor SDF-1 (stromal cell-derived factor-1). SDF-1 is particularly involved in homing processes of hematopoietic stem cells as well as leukemic stem cells. After a broad characterization of NOX-A12 in preclinical models, Phase I studies demonstrated that the neutralization of SDF-1 leads to the mobilization of stem cells in healthy volunteers. Currently, NOX-A12 is evaluated in an open label study in patients suffering from chronic lymphatic leukemia (CLL) and multiple myeloma (MM) in combination with chemotherapy.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany
4:45
A Synthetic, Chemically Modified siRNA Targeting Caspase-2 as a Therapeutic Agent for Ocular Neuroprotection
Caspase-2 is a pro-apoptotic gene specifically expressed and activated in retinal ganglion cells following acute optic nerve injury or intraocular pressure increase. Intravitreal administration of QPI1007 (a synthetic siRNA targeting caspase-2) demonstrated local RNAi, lack of inflammatory effects and excellent preclinical and clinical safety profiles. Neuroprotective activity of QPI1007 has been demonstrated in five preclinical animal models involving RGC loss. In human Phase I trials in NAION patients, a single intravitreal injection of QPI1007 administered within 2 weeks following disease onset prevented further visual deterioration within one year observation. This is in contrast to the natural course of the disease resulting in 3 and more line loss of visual acuity in ~20% of patients within the first 6 months following the ischemic event.
Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel
Caspase-2 is a pro-apoptotic gene specifically expressed and activated in retinal ganglion cells following acute optic nerve injury or intraocular pressure increase. Intravitreal administration of QPI1007 (a synthetic siRNA targeting caspase-2) demonstrated local RNAi, lack of inflammatory effects and excellent preclinical and clinical safety profiles. Neuroprotective activity of QPI1007 has been demonstrated in five preclinical animal models involving RGC loss. In human Phase I trials in NAION patients, a single intravitreal injection of QPI1007 administered within 2 weeks following disease onset prevented further visual deterioration within one year observation. This is in contrast to the natural course of the disease resulting in 3 and more line loss of visual acuity in ~20% of patients within the first 6 months following the ischemic event.
Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel
5:15
Safety, Pharmacokinetics and Pharmacodynamics of PNT2258 (An anti-BCL2 DNA Targeted Drug) in Patients with Advanced Solid Tumors
The abstract is unavailable at this time.
Wendi V. Rodrigueza, Ph.D., CSO and VP, Product Development, ProNAi Therapeutics, Inc.
The abstract is unavailable at this time.
Wendi V. Rodrigueza, Ph.D., CSO and VP, Product Development, ProNAi Therapeutics, Inc.
5:45
Close of TIDES
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