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Oligonucleotide Therapeutics and Delivery 2016
- 치료용 올리고뉴클레오티드 전달 컨퍼런스 2016 -
2016년 4월 4일 - 2016년 4월 5일
미국 매사추세츠주 케임브리지, Hyatt Regency Cambridge

Oligonucleotide Therapeutics and Delivery


올리고뉴클레오티드 기반 치료제는 이전부터 제3의 중요 의약품 개발 플랫폼이었으며, 특히 RNA 및 게놈 자체를 표적화함으로써 유전자 발현을 조절하는 기술에 초점이 맞추어지고 있습니다. 치료제로서 핵산의 중요 특징은 소분자 약제 및 생물제제에서는 불가능한 영역에도 대응할 수 있다는 점으로, 치료법이 제한되어 있거나 존재하지 않는 영역을 포함한 다양한 질병을 치료하기 위한 의약품의 개발이 가능합니다. 따라서 이 분야에 대한 관심은 많이 높아졌으나, 제1세대 분자는 효능과 안전성면에서 문제를 가지고 있으므로 의약품 개발 세계에 큰 영향을 미치지는 못하고 있습니다. 그러나 최근 핵산 화학 및 전달 기술의 진보에 의해 안정성, 생체이용률(bioavailability), 특이성, 효능 등이 개선됨에 따라 성공을 전망할 수 있는 신세대 치료제를 단기간에 개발, 임상에서의 평가를 받는 것이 가능해졌습니다.

2016년 4월 4일부터 5일까지 미국 매사추세츠주 케임브리지에서 개최되는 Oligonucleotide Therapeutics and Delivery에서는 의약품 개발과 Discovery에 종사하고 있는 주요 연구자가 참가하여 올리고뉴클레오티드 기반 치료제에 활용되고 있는 기술 및 과학적 진보 등에 대해 논의합니다.

  

4월 4일(월)

 

7:00 등록 & 커피

 


치료용 올리고뉴클레오티드의 진보

8:10 의장 개회사

Dmitry Samarsky, Ph.D., Senior Vice President, Technology Development, RiboBio Co, China

8:15 기조 강연:올리고뉴클레오티드 치료제의 새로운 패러다임으로서의 N-아세틸갈락토사민(GalNAc) 공액 siRNA

Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.

During this presentation, I will discuss the progress in the advancement of RNAi therapeutics and review delivery of RNAi and where the field is going. I will also discuss conjugated delivery of oligonucleotides to the liver and combining novel chemical modifications with conjugation strategies.

8:45 입체적 순수(Stereopure) 핵산 치료제 개발

Chandra Vargeese, Ph.D., Senior Vice President and Head, Drug Discovery, WAVE Life Sciences

WAVE Life Sciences is utilizing its innovative and proprietary synthetic chemistry drug development platform to design, develop and commercialize stereopure nucleic acid therapeutics that precisely target the underlying cause of rare genetic diseases, delivering exceptional treatment options for patients. Given the unique versatility of its chemistry platform, WAVE's pipeline will span multiple oligonucleotide modalities including antisense, exon-skipping and single-stranded RNAi.

9:15 유전병과 감염증 치료용 새로운 PMO(Phosphorodiamidate Oligomers)

Bruce Wentworth, Ph.D., Vice President, Biology, Sarepta Therapeutics

PMOs are being tested in advanced clinical trials for the treatment of patients with Duchenne muscular dystrophy (DMD), a rare, X-linked disease that results in progressive muscle loss and premature death. Research has shown that for other disorders, including viral and bacterial infection as well as rare diseases such as Pompe disease, modified PMOs may be more appropriate due to their potential for enhanced delivery and tissue targeting. The PMO-based technology has the potential to be a versatile, modifiable, and widely applicable treatment in any number of disease states.

9:45 스폰서 제공 프레젠테이션(연사 모집중)

10:15 전시회장 휴식시간, 포스터 발표 관람

 

합성과 의약화학

10:45 특별 강연:3가 GalNAc 공액 안티센스 올리고뉴클레오티드의 구조 활성 상관

Punit Seth, Ph.D., Executive Director, Medicinal Chemistry, Isis Pharmaceuticals

Trivalent GalNAc, a high affinity ligand for the hepatocyte-specific Asialoglycoprotein receptor (ASGR), enhances the potency of antisense oligonucleotides (ASOs) for inhibiting gene targets expressed in hepatocytes. We undertook a comprehensive structure-activity relationship study to determine the optimal structural requirements for enhancing ASO potency via ASGR mediated delivery to hepatocytes. As part of this effort, GalNAc clusters assembled from six distinct branched or amino acid scaffolds were synthesized and attached to ASOs using simplified solution-phase or phosphoramidite based methods. Within each cluster, the length and hydrophobicity of the linker attaching the GalNAc sugar to the branching point on the scaffold was varied. The effect of reducing backbone phosphorothioate content (PS) and changing the linker moiety between the GalNAc cluster and the ASO was also evaluated. Details from this work which resulted in the selection of a simplified trivalent GalNAc ASO conjugate for evaluation in human trials will be presented.

11:15 RNA 기반 치료제용 Phosphorodithioate RNA

Xianbin Yang, Ph.D., Director, R&D, AM Biotechnologies

During this presentation I will discuss the chemistry for synthesizing phosphorodithioate (PS2)-modified siRNAs, aptamer, and anti-miRNAs; crystal structures of PS2-modified siRNAs and protein-RNA complexes; therapeutic aptamers with remarkably improved binding affinity (from nM to pM) with a single PS2 substitution; and in vitro and in vivo gene silencing activity of PS2-substituted RNA.

11:45 구아닌 사중나선 구조를 안정시키고 백혈병 세포내에서의 성장 저해 활성을 강화하는 c-Myc 프로모터 표적화 올리고뉴클레오티드의 지질 수식

Gilles Tapolsky, Ph.D., CSO, Advanced Cancer Therapeutics

We have shown that Pu27 reduces c-MYC transcription in leukemia cell lines and consequently inhibits cell growth and promotes apoptosis. In this study, we evaluated the effect of Pu27 modification using polyethylene glycol (PEG), tocopherol (Toco) and the lipid palmitate in order to increase G-quadruplex stability and lessen blood clearance. Our finding suggests that modification of the c-MYC targeted oligonucleotide by addition of lipids stabilizes the 3D structure (G-quadruplex) and improve its function at inhibiting cell growth most likely by down-regulating c-MYC.

12:15 런천 프레젠테이션 또는 개별 점심식사

 

암 면역치료와 병용요법

1:25 의장 발언

Art Krieg, M.D., Founder and CEO, Checkmate Pharma

1:30 특별 강연:체크포인트 저해요법의 효과율 향상 가능성:TLR9의 역할

Art Krieg, M.D., Founder and CEO, Checkmate Pharma

Many immunologists have speculated that combining a strong Th1 immune activator known to be capable of inducing multifunctional anti-tumor CD8+ T cell responses in cancer patients together with anti-PD-1/PD-L1 would greatly increase the response rates to therapy compared to either agent alone. Checkmate's TLR9 agonist program has shown such a response in humans with excellent safety, and will be moving into clinical development in combination with an anti-PD-1 antibody in advanced cancer patients in early 2016.

2:00 체크포인트 저해제와의 병용에 의한 효과적인 치료를 가능하게 하는 종양내 imo-2125(TLR9 아고니스트)를 이용한 종양미세환경 조절

Sudhir Agrawal, D.Phil., President, Research, Idera Pharmaceuticals

 

2:30 CureVac의 배열 최적화 mRNA - 차세대 생물제제 개발 루트

Mariola Fotin-Mleczek, Ph.D., CSO, CureVac

Recent advances strongly suggest that mRNA is the basis for a new class of vaccines and drugs. RNActive®, one of CureVac's technologies has been developed on this basis and provides potent prophylactic vaccines and novel immunotherapies against cancer. These successes could be extended preclinically to mRNA protein and gene replacement therapy. The production of mRNA-based vaccines and drugs is highly flexible, scalable and cost competitive, and eliminates the requirement of a cold chain. Furthermore CureVac's proprietary optimization process allows the generation of sequence optimized yet natural mRNA that provides a safe and efficient method for enabling the human body to produce its own medicine.

3:00 전시회장 휴식시간, 포스터 발표 관람

 

3:30 RNAi를 이용한 면역 체크포인트의 사일런싱(Silencing)

Alexey Wolfson, Ph.D., Founder and CSO, MirImmune

 

4:00 면역 조절 기능을 가진 구형 핵산

David Giljohann, Ph.D., CEO, Exicure

Immunomodulatory nucleic acids act by agonizing or antagonizing endosomal toll-like receptors (TLR3, TLR7/8, and TLR9), proteins involved in innate immune signaling. Immunomodulatory spherical nucleic acids (SNAs) that stimulate (immunostimulatory, IS-SNA) or regulate (immunoregulatory, IR-SNA) immunity by engaging TLRs have been designed, synthesized, and characterized. IR-SNAs exhibit up to eightfold increases in potency and 30% greater reduction in fibrosis score in mice with nonalcoholic steatohepatitis (NASH). Given the clinical potential of SNAs due to their potency, defined chemical nature, and good tolerability, SNAs are attractive new modalities for developing immunotherapies.

4:30 스폰서 제공 프레젠테이션(연사 모집중)

5:00 전시회장 환영 리셉션, 포스터 발표 관람

6:00 첫째날 종료


4월 5일(화)

7:30 조찬 그룹 토론

 

항바이러스제 개발

8:25 의장 발언

Andrew Vaillant, Ph.D., CSO, Replicor Inc.

8:30 핵산 폴리머:만성 B형 간염, B형 간염/D형 간염 감염증 치료에서의 항바이러스 기서와 응용

Andrew Vaillant, Ph.D., CSO, Replicor Inc.

Nucleic acid polymers (NAPs) are a newly emerging antiviral technology for the treatment of chronic HBV infection and HBV / HDV co-infection. NAPs have the unique ability to clear HBsAg from the blood of human patients, a critical step in achieving a functional cure in HBV and HBV / HDV infection. Replicor will present its current mechanistic data underlying the basis for this unique antiviral effect of NAPs as well as updated clinical data showing Replicor's progress in using NAP-based combination therapy in patients with chronic HBV infection and HBV / HDV co-infection towards achieving functional cure for these infections.

9:00 만성 B형 간염 감염증과 인자 12 매개 질환용 RNAi 치료제에서의 DPC 기술 이용

David Lewis, Ph.D., CSO, Arrowhead Research Corporation

9:30 스폰서 제공 프레젠테이션(연사 모집중)

9:45 전시회장 휴식시간, 포스터 발표 관람

 

RNA 기반 치료제와 전달 기술의 진보

10:25 의장 발언

Balkrishen (Bal) Bhat, Ph.D., Vice President, Chemistry, RaNA Therapeutics

10:30 장쇄 논코딩 RNA(lncRNA) : 의약품 개발의 새로운 영역

Balkrishen (Bal) Bhat, Ph.D., Vice President, Chemistry, RaNA Therapeutics

The ss-siRNA activity in vivo requires a metabolically stable 5'-phosphate analog. Here, we used crystal structure of the 5'-phosphate binding pocket of Ago-2 bound with guide strand to design and synthesize ss-siRNAs containing various 5'-phosphate analogs. Chemically modified ss-siRNA targeting human apoC III mRNA demonstrated good potency for inhibiting ApoC III mRNA and protein in transgenic mice. Moreover, ApoC III ss-siRNAs were able to reduce the triglyceride and LDL cholesterol in transgenic mice demonstrating pharmacological effect of ss-siRNA.

11:00 프로프라이어터리 마이크로RNA의 독특한 기능 기반 비약적인 신규 암치료법 개발

Roel Q.J. Schaapveld, Ph.D., MBA, CEO, InteRNA Technologies BV

To explore miRNAs as therapeutic angiogenesis-inhibitors, we performed a functional screen to identify miRNAs that are able to decrease EC viability. We identified miRNA-7 (miR-7) as a potent negative regulator of angiogenesis. This study provides a comprehensive validation of miR-7 as novel anti-angiogenic therapeutic miRNA that can be systemically delivered to both EC and tumor cells and offers promise for miR-7 as novel anti-tumor therapeutic.

11:30 지질 기반 올리고뉴클레오티드 전달 시스템 개발

Volker Fehring, Ph.D., Director, Formulation Development, Silence Therapeutics GmbH

Posttranscriptional gene silencing by RNA interference can be therapeutically exploited to inhibit pathophysiological gene expression. However, in contrast to the established effectiveness of RNAi in vitro, safe and effective delivery of siRNAs to specific organs and cell types in vivo remains the major hurdle. Here, we report the development and in vivo characterization of a novel siRNA delivery system (DACC lipoplex) suitable for modulating target gene expression.

12:00 런천 프레젠테이션 또는 개별 점심식사

 

중추신경계로의 전달

1:00 의장 발언

Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University, South Korea; CEO, OliX Pharmaceuticals

1:05 비강 투여 후 세로토닌 수송체의 사일런싱을 시행하는 공액 siRNA의 치료용 항우울제로서의 가능성

Andres Montefeltro, Ph.D., CEO, nLife Therapeutics, S.L.

nLife Therapeutics has developed different nucleic acid chemical modifications with the aim to optimize cell specific delivery capabilities to neurons. We have combined siRNAs and antisense oligonucleotides (ASOs) with some specific and potent small molecule ligands to neuronal receptors or transporters, named nOligos (neuronal specific oligonucleotides). These combinations proved to deliver the nucleic acid to the target neuron in an effective way. Also, the intranasal administration of the modified nucleic acids reached the targeted brain area and neurons in mice and monkeys.

1:35 신경변성질환 치료에 대한 치료용 올리고뉴클레오티드 엑소좀을 매개로 한 전달

Anastasia Khvorova, Ph.D., Professor, Molecular Medicine, RNA Therapeutics Institute, University of Massachusetts Medical School

Oligonucleotide therapeutics is a new class of drugs, the clinical utility of which has been limited by inefficient tissue distribution and cellular uptake. Through our research, we have developed a novel methodology that enables the loading of hydrophobically modified oligonucleotides (hsiRNA) into exosomes. These hsiRNAs show efficient cellular uptake in vitro as well as broad brain distribution and in vivo efficacy. Exosome-formulated oligonucleotide therapeutics might be a solution for the development of novel therapeutics for the treatment of neurodegenerative disorders.


생체내 전달에 대한 새로운 어프로치

2:05 특별 강연:제2세대 RNAi 트리거를 이용한 치료제 개발

Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University, South Korea; CEO, OliX Pharmaceuticals

Recent studies came up with novel RNAi triggering molecular structures with unique structural features and functional advantages compared with the conventional siRNA. During this presentation I will introduce novel RNAi triggers developed in my laboratory, with improved features over conventional siRNA, such as reduced off-target effects, enhanced cellular delivery when complexed with cationic delivery vehicles, and specific target gene silencing combined with immunostimulation. One of these second generation RNAi triggers, asymmetric siRNAs (asiRNAs), were combined with specific set of chemical modifications to generate cell-penetrating asiRNAs (cp-asiRNAs), which can execute gene silencing without delivery vehicle both in vitro and in vivo. I will introduce current therapeutic development programs based on the cp-asiRNA structures.

2:35 올리고뉴클레오티드 Discovery와 전달을 위한 새로운 나노약품 플랫폼

Art Levin, Ph.D., Executive Vice President, Research and Development, Avidity NanoMedicines

Despite the considerable promise, delivery has proven to be one of the central challenges of oligonucleotide-based therapeutics. Oligonucleotides are large, hydrophilic and highly negatively charged, so they don't cross cell membranes. We have pioneered the development of Precision NanoMedicines, which are targeted, polymeric nanoparticles encapsulating siRNA drug payloads for delivery to specific tumor types. These self-assembling nanoparticles can be decorated with antibodies, proteins, peptides and small molecules to bind to extracellular receptors and facilitate cellular uptake.

3:05 전시회장 휴식시간, 포스터 발표 관람

3:45 복수 환자 유래 이종이식편 종양에 대한 Dicer-Substrate siRNA(DsiRNA) 전달

Bob Brown, Ph.D., CSO, Dicerna Pharmaceuticals

Lipid Nanoparticle (LNP) technology is an elegant solution for delivery of RNAi triggers, since it enables both bioavailability to target organs as well as the ability to transfect target cells. However, while LNPs are well characterized for delivery of RNA oligonucleotides to the normal liver, much remains to be explored regarding the mechanisms of LNP-mediated delivery to tumors. In this study, we investigated the ability of Dicerna's unique LNP platform, termed EnCore, to deliver Dicer- substrate siRNAs (DsiRNAs) to xenograft tumors of diverse origin.

4:15 메신저 RNA 기반 치료제의 전임상 연구에서 임상 연구로의 이동

Pad Chivukula, Ph.D., CSO & COO, Arcturus Therapeutics

Arcturus has developed a novel, potent and safe RNA Therapeutics platform called LUNAR™, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, antisense and microRNA oligotherapeutics. In addition, we incorporate Unlocked Nucleic Acid (UNA) chemistry into the oligonucleotide drug candidate enabling the targeting of any gene in the human genome. This presentation will provide an update on our lead asset, an UNA-modified, LUNAR-formulated siRNA targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

4:45 반흔형성을 억제하는 RXI-109의 임상 개발

Pamela Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals Corp.

RXI-109 is a self-delivering RNAi compound (sd-rxRNA®) in development as a therapeutic to target and reduce connective tissue growth factor (CTGF) in order to impede the fibrotic pathway. Preliminary results from Phase 2a dermal clinical trials indicate a better outcome (reduced scar formation) following hypertrophic scar revision surgery when the incision site is treated by intradermal injections of RXI-109. A summary of the ongoing dermal trials and an overview of a Phase 1/2 trial to prevent subretinal fibrosis in subjects with neovascular age-related macular degeneration will be discussed.

5:15 올리고뉴클레오티드의 약리 작용을 강화하는 소분자

Rudolph L. Juliano, Ph.D., Boshamer Distinguished Professor, Department of Pharmacology, University of North Carolina

Endosomal trapping is a key impediment to the effective use of oligonucleotides in therapy. We have used high throughput screening to identify small molecules that selectively release oligonucleotides from the late endosome compartment thus increasing access to the cytosol and nucleus. These compounds substantially enhance pharmacological effects of several types of oligonucleotides both in cell culture and in mouse models.

5:45 컨퍼런스 폐막

 

* 주최측 사정에 따라 사전 예고없이 프로그램이 변경될 수 있습니다.



Image Credit: Luminous BioSciences

Luminous BioSciences offers high quality custom DNA oligos that are sunthesized according to your needs. We provide DNA oligo synthesis from 10 base to 200 bases.
www.luminousbio.com


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