DIA Your Way! Part 2, Antibodies Against Membrane Protein Targets New this year, we are delighted to announce a third conference

info@cshl.edu We have applied for funds from industry to partially support graduate students and postdocs.

Cold Spring Harbor Laboratory Maps & Directions. Joe Fraone, Business Development Manager, Oligonucleotides. crooke Assess the impact of regulations and guidance on oligonucleotide drug development and discuss possible avenues for changes and improvements. Please click the button below to register for this event.

BOOK BY 30TH JUNE AND SAVE 200, Additional Contact Info: 5:30 Oligonucleotides with Charge-Neutralizing Branched Groups on the Backbones That Enhance Cellular Uptake, David R. Tabatadze, Ph.D., President and CEO, ZATA Pharmaceuticals. Antisense oligonucleotides (ASOs) that directly target RNA of disease-associated genes may be therapeutically beneficial. Facilitate open discussions among all industry researchers and regulatory agencies regarding oligonucleotide drug development. all rights reserved. Adequate therapies are lacking for neurological disorders, including Alzheimers. Privacy Cancellations are accepted until September 2nd and a $50 fee will be charged. regulatory specialists and technology providers to discuss advances in next-generation oligonucleotide and precision therapeutics throughout the drug development process. The status (talk/poster) of abstracts will be posted on our web site as soon as decisions have been made by the organizers. Professional Membership = $100

As novel conjugated oligonucleotides emerge in the field, simple and reproducible methodologies are required to facilitate and accelerate systematic assessment of these compounds.

Please upgrade to a newer version of Internet Explorer or use an alternate browser such as Chrome or Firefox. This website uses cookies. The .gov means its official.Federal government websites often end in .gov or .mil. Dr. Woodcock is now the FDA's Principal Deputy Commissioner. CRISPR for Disease Modeling and Target Discovery, Artificial Intelligence in Drug Discovery Instagram, Meetings & Courses Program Selection of material for oral and poster presentation will be made by the organizers and individual session chairs. This site is not optimized for Internet Explorer 8 (or older). We use cookies so that we can offer you the best possible website experience. Full payment is due four weeks prior to the meeting. ABL, which fights against infectious diseases and cancers, through the complementary approaches of its companies in the fields of diagnosis, immunotherapy, food safety and nutrition, maintains global GMP facilities meeting U.S. and European regulatory standards, providing, virus-based oncolytic therapies, gene therapies, vaccines, and protein-based immunotherapeutic products. Please apply in writing via email to Maureen Morrow and state your financial needs. The translation of RNA from an effective genomic tool into a novel therapeutic modality has been hindered by the difficulty to deliver RNA molecules into specific target tissues by systemic administration, especially to leukocytes. are the challenges and opportunities in modulating the immune system with oligonucleotide Please note, we are also offering that flexibility to our speakers so you will experience some speakers presenting remotely. Emerging Immune Modulation Strategies NEW, Target Identification and Validation Clear understanding of the PK/PD behavior is essential for characterizing the performance of RNAi-based drug candidates in vivo. Samuel Gunderson, Ph.D., President and Co-Founder, SilaGene Inc. U1 Adaptors oligonucleotides (U1AOs) interfere with polyadenylation of gene-specific mRNA, causing their selective destruction inside the nucleus. Regular packages are all-inclusive and cover registration, food, housing, parking, a wine-and-cheese party, cocktail reception, and lobster banquet. Global experts from big pharma and leading biotechs will explore the emerging therapeutic applications of oligonucleotides with case studies surrounding the discovery and development of oligonucleotide therapeutics as well as CMC and regulatory standpoints. *You may become a member during the registration process and fees will automatically adjust. Rockville, MD 20852 9:35 Using Single-Stranded Donor DNA for Homology Directed Repair Catalyzed by CRISPR/Cas9 Activity, Eric. (516) 367-8346 ABL has extensive experience working with diverse organizations , including industry, government, and academic entities to support their quest in improving public health. 8:00 Interactive Breakout Discussion Groups with Continental Breakfast. Developed collaboratively by regulators, industry professionals, and academics, the program covers a wide range of topics from the nonclinical, CMC, and clinical areas. No-Housing packages include all costs except housing. Please click here for more information regarding our vaccine policy and mask requirement. The Oligo Meeting returns in person in 2022, The 18th Annual Oligo Meeting is a hybrid event, which includes a robust in-person program live from, (prices will increase by $200 after September 2nd). The conference offers a unique experience with multiple perspectives presented, and the opportunity to interface with regulators from around the globe. The 2022 DIA/FDA Oligonucleotide-based Therapeutics Conference is intended for a diverse group of scientists responsible for the evaluation of safety and efficacy of oligonucleotide products including interested industry, academic and government parties involved in ongoing efforts in the research and development of oligonucleotides. Explore the benefits of becoming a member. Twitter: @SMiPharma | #SMiOligonucleotides, The rights of trade mark owners are acknowledged. RNAi triggers are routed on cell entry through endosomes, which are well equipped to metabolize RNA and to initiate host immune responses. Silver Spring, MD 20993 The Oligonucleotide Therapeutics Society is excited to announce that we are planning an in-person meeting in Montreal for our 18th Annual Meeting. Without a subpoena, voluntary compliance on the part of your Internet Service Provider, or additional records from a third party, information stored or retrieved for this purpose alone cannot usually be used to identify you. Abstracts are welcomed on all scientific topics related to the development of RNA and other oligonucleotide-based therapeutic approaches. from siRNA, modified mRNA and CRISPR /Cas will be considered. How to improve payer engagement with tailored value communication. Annual Meeting of the Oligonucleotide Therapeutics Society. Part 1, Targeting KRAS and Other Small G Proteins, Artificial Intelligence in Drug Discovery Use tab to navigate through the menu items.

Ekkehard Leberer, Senior Life Sciences Consultant, Elbicon Website: www.oligonucleotide.co.uk/pharmaphorum, Advancements in extra-hepatic delivery of oligonucleotides. The 18th Annual Oligo Meeting is a hybrid event, which includes a robust in-person program live from Hilton Phoenix Resort at the Peak in Phoenix, Arizona and a virtual component allowing delegates who are unable to attend in-person to enjoy the Annual Meeting from the comfort of their home or office. 10903 New Hampshire Avenue Managing CMC Activities For The Development Of Oligonucleotide Therapeutics led by Mia Kiistala, CEO/Owner/Principal Consultant, Aurora CMC Consulting, CHAIRS FOR 2022: Please note that based on your settings, it is possible that not all functionalities of the website will be available. Food and Drug Administration Global Program Officer program dedicated to Emerging mRNA Therapeutics to complement our established Oligo Discovery & Delivery and CMC & Regulatory programs. In what indications? Considerations for Therapeutic Translation of CRISPR/Cas9, Eric B. Kmiec, Ph.D., Director, Gene Editing Institute and Senior Research Scientist, Center for Translational Cancer Research, Helen F. Graham Cancer Center & Research Institute, Christiana Care Health System, Designing and Delivering Better Oligonucleotide Drugs, Moderators: Dan Peer, Ph.D., Director, Laboratory of Precision NanoMedicine, Tel Aviv University, Exploring CRISPR and Other Innovative Drug Modalities, Thomas D. Madden, Ph.D., President and CEO, Acuitas Therapeutics, 9:05 Therapeutic Protein Expression in vivo Using Messenger RNA-Lipid Nanoparticles. No on-site registrations will be accepted. Now in its 8th year, this unique event brings together leading discovery scientists, developers, CMC experts, Alongside the innovative programme, attendees can engage in a variety of event features and make the most of participation in a range of virtual, remote experiences.. Learn more.

use an integrated array of optimized immunology and molecular laboratory assay platforms, to extract immunological correlates of activity and response in both preclinical studies and clinical trials., To stay informed on the latest updates, please follow us on. BOOK BY 29TH APRIL AND SAVE 400 Benefit from the insight of our expert speakers via live and pre-recorded presentations. Please click here for full details on all breakouts. 6:00 Characterization of Novel Conjugated RNAi drugs, Bruno M.D.C. Godinho, PhD., Milton-Safenowitz Post-Doctoral Fellow, Laboratory of Dr. Anastasia Khvorova, RNA Therapeutics Institute, University of Massachusetts Medical School. Oligonucleotide Therapeutics and Delivery Conference 2022 Can However mRNA is labile and requires a delivery system to access cells. Hilton Phoenix Resort at the Peak in Phoenix, Arizona. The technical storage or access is necessary for the legitimate purpose of storing preferences that are not requested by the subscriber or user. Examples will be provided outlining the strategy for carrying out genetic surgery more precisely and how they may enable productive outcomes in the clinic. We are thrilled to able to offer flexibility to our attendees to attend in-person or virtually. In that position, she has led many of the FDAs groundbreaking drug initiatives. Drug Information Association (DIA) DIA is a global association that mobilizes life science professionals from across all areas of expertise to engage with patients, peers and thought leaders in a neutral environment on the issues of today and the possibilities for tomorrow. FDA clears Incyte's Opzelura as first vitiligo therapy, Novartis faces tougher decision on Sandoz, as finances recover, Roche CEO Severin Schwan to stand down after 14 years, VBL craters as ovarian cancer gene therapy fails phase 3 test, Ex-GSK vaccine chief to lead new UK biotech Vicebio, Twill taps YourCoach for DtX-health coaching integration, After cough drug setback, Merck partners Hyfes tracking app, As FDA inspections return, upholding compliance and maintaining GMP readiness is more crucial than ever, Allergan bolsters aesthetics pipeline with Exicure hair loss deal, How pharma can help the post-COVID recovery of European cancer care.

Many genetic diseases can be treated by restoring defective gene expression. Recent research implicates Wnt/-catenin signaling as a mechanism of resistance to cancer immunotherapy. The two-day conference will bring you high-quality insights and industry connections on the latest clinical trial candidates and a platform for exchanging ideas for tackling the biggest challenge: DELIVERY. If the capacity limit is reached, we will only be able to offer the virtual option. Part 2 NEW, 250 First Avenue, Suite 300Needham, MA 02494P: 781.972.5400F: 781.972.5425 Corporate No-Housing Package: $1035, Meetings The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. zon hogrefe rick jerry ripples tides conference standup blogger trilinkbiotech Deepen your understanding of oligonucleotide therapeutic delivery and explore the latest innovations in extrahepatic oligonucleotide delivery and target specificity The technical storage or access is strictly necessary for the legitimate purpose of enabling the use of a specific service explicitly requested by the subscriber or user, or for the sole purpose of carrying out the transmission of a communication over an electronic communications network. Phone: (619) 795-9458. The initial indication is to treat skin hypertrophic scar and then extend it to liver fibrosis, such as Primary Sclerosing Cholangitis, and liver cancer, such as Cholangicarcinoma, as well as other fibrotic conditions. Twitter CSHL Courses I will discuss the unique advantage of this polypeptide nanoparticle technology for efficient siRNA delivery, its pharmaceutical properties for manufacturing and its preclinical safety profile.

The primary audience includes leading academic experts, interested pharmaceutical companies, regulatory agencies, patient advocacy groups, non-profit organizations, scientists, clinicians from regulatory, academic, industrial and other healthcare sectors, scientists involved in drug development in those same industries, regulatory scientists, experts and leaders from industry, academia, regulatory government agencies in the US and abroad, patient representatives, healthcare providers, and pharmacokineticists (clinical, preclinical, and toxicokinetics) who are involved in oligonucleotide drug development or supporting research., Robin M. Weinick, Senior Vice President and Managing Director, Americas All participants at in-person DIA Meetings, Workshops, Forums and Conferences, are required to be fully vaccinated. Advances in genomics, gene editing, oligonucleotide synthesis, delivery and manufacturing, and an increase in collaborations and licensing opportunities have all contributed to a resurgence in the development of DNA and RNA-based drugs. Location: London, UK Plus, two interactive half day pre-conference workshops on 20th September 2022: Oligonucleotide Therapies- Overcoming The Challenges Of Delivery led by Nick Lench, Executive Director, NATA, Martin Kerr, Head of Business Development & Partnerships, Nucleic Acid Therapy Accelerator, Ritwick Sawakar, MRC Investigator, MRC Toxcology Unit, University Of Cambridge

Already a DIA Member? FDA/CDER/OMPT/OND Social distancing measures may be in place at the time of the event. We can activate gene expression by three distinct technology platforms: 1) Targeting repressive cis-acting long noncoding RNA, 2) Enhancing stability of mRNA transcript, or 3) Supplementing in vitro transcribed mRNA. Advances in RNAi trigger chemistry and targeting allow effective knockdown without requiring active endosomal escape, without toll receptor activation and allowing subcutaneous delivery. Here, I will describe some of the challenges and opportunities in modulating leukocytes response using RNA molecules and discuss adverse effects such as immuno-toxicity. One Bungtown Road, Cold Spring Harbor, NY 11724, If you are a US federal employee impacted by the government shutdown and are unsure how to proceed with registering for this meeting, please contact. Engage in the latest innovations in oligonucleotide therapeutics such as utilising AI machine learning to drive the discovery of novel therapeutics. These and other data are moving neuro-focused ASOs from bench to bedside, with one FDA approved ASO and several others in human clinical trials for neurodegeneration. Recent progress in the discovery and preclinical development of all three platforms will be presented. Click here for details on short courses offered. An intensive 1-day meeting that delves into the latest in oligonucleotides chemistry, process & analytical development, therapeutics, antisense therapy & a meeting place for experts working within cutting-edge oligo research & therapeutic development.

DCR-BCAT is an RNAi-based experimental drug targeting -catenin, formulated in a tumor-selective nanoparticle. We are eager to have as many young people as possible to attend since they are likely to benefit most from this meeting. United States.

We produce cutting edge congresses and summits for the Life Sciences Industry, bringing together industry leaders and solution providers at a senior level, creating the opportunity to partner, network and knowledge share. September 26-27 Conference: CRISPR for Disease Modeling and Target Discovery, September 27-28 Conference: Emerging Oligonucleotide Therapeutics, September 28-29 Symposium: Tackling Rare Diseases, 2:00 Refreshment Break in the Exhibit Hall with Poster Viewing, Development of New Oligonucleotide Platforms and Chemistries, Tanuja Koppal, Ph.D., Conference Director, Cambridge Healthtech Institute, Bruce Given, M.D., COO, Arrowhead Pharmaceuticals, 2:55 Chemistry Developments and Therapeutic Applications of GalNAc-siRNA Conjugates, Ivan Zlatev, Ph.D., Senior Scientist, Research, Alnylam Pharmaceuticals, 3:25 RNAi Based Human Therapy for Chronic Hepatitis B Infection. The designated hashtag for this meeting is #cshlRNATX. By clicking SUBSCRIBE, you are agreeing to our. This event offers two learning avenues depending on your preference! The technical storage or access is required to create user profiles to send advertising, or to track the user on a website or across several websites for similar marketing purposes. Cambridge Healthtech Institutes Inaugural conference on Emerging Oligonucleotide Therapeuticstracks both the scientific and clinical progress being made in developing new molecular entities, improving synthesis and delivery technologies and extending stability and safety profiles for oligonucleotide-based therapies. RNA interference (RNAi) occurs in the cytoplasm. 3:10 From Local to Systemic: Delivering Novel siRNA Therapeutics for Multiple Clinical Indications. 1:50 Refreshment Break in the Exhibit Hall with Poster Viewing, Dan Peer, Ph.D., Director, Laboratory of Precision NanoMedicine, Tel Aviv University, 2:40 Moving from Intravenous to Subcutaneous Delivery in RNAi Why and How, Bruce D. Given, M.D., COO, Arrowhead Pharmaceuticals. In this role she works closely with the Commissioner of Food and Drugs to develop and implement key public health initiatives and helps oversee the agencys day-to-day functions.

Leaders in the field from across the globe alongside students will present on an array of topics, including chemistry, AI/machine learning, genome & RNA editing, and preclinical and clinical research. Associate Director for Pharmacology and Toxicology Join. In addition preclinical results illustrating the application of mRNA-LNP therapeutics in a several clinical areas will be presented. With these three different platforms, we can achieve specific gene upregulation and tailor to desired therapeutic profiles. Details on the topics and moderators are below. 4:25 Targeted Platform for RNA Therapeutics. DIA/FDA's Oligonucleotide-based Therapeutics Conference brings together leading experts to inform, educate, and share advancements in oligonucleotide-based therapeutic product development. Registration will close one week prior to the conference to allow time to process all registration forms and verify vaccination status. Academic/Student No-Housing Package: $835 Preview all session descriptions, speakers, and more all in one place! Ronald.Wange@fda.hhs.gov, More Meetings, 800 Enterprise Road, Suite 200 Therefore, we encourage you to register and make your housing arrangements early due to potential limited capacity. . Graduate/PhD Student Package: $1020 Click accept cookies to continue. This includes cookies which are necessary for the operations of the website, as well as cookies that are only used for anonymous statistical purposes, for comfort settings or to display personalized content. The Oligo Meeting returns in person in 2022 Novel phosphoramidite monomers enabling the incorporation of charge-neutralizing branched groups (CNBGs) on internucleoside phosphates of oligonucleotides during the automated synthesis have been developed. Cancellations are accepted until September 2nd and a $50 fee will be charged. Part 2, Potential human applications of genome editing to somatic cells, Scientific and technical considerations for design and application of genome editing strategies, Ethical and regulatory issues associated with somatic cell genome editing, Development of reproducible and robust strategies that can enable precise point mutation repair or gene insertion, Types of experimental variabilities with the use of different cell lines; established cell lines, iPSCs and primary cells in general. Fees are the same for in-person or virtual attendance, Deadline to register at Early Bird Prices September 2, 2022 Is delivery the only challenge that will change the fate of oligonucleotide therapies? ABL has extensive experience working with diverse organizations including industry, government, and academic entities to support their quest in improving public health. New generations of lipid nanoparticle systems (LNP) allow efficient delivery and expression of mRNA via different routes of administration. Our Symposiums are delivered through Swapcard, our market-leading and highly interactive digital event platform. Shanthi Ganesh, Ph.D., Associate Director, Pre Clinical Oncology, Dicerna Pharmaceuticals, Inc. Systemically delivered U1AO-peptide conjugates targeting KRAS and MYC lead to tumor shrinkage in xenograft mice harboring pancreatic cancer with no apparent toxicity. Upon registration, please indicate via the check box which avenue you are registering for: in-person or virtual. Courses Full Registration for the Oligonucleotide Chemistry & Therapeutics Symposium now open. In addition, I will detail the challenges of targeting lipid-based nanoparticles directly into specific cells. Jimmy Weterings, Principal Scientist, AstraZeneca, EARLY-BIRD RATES: In 1994, Dr. Woodcock was named Director of the CDER. ABLInc. a subsidiary of the FrenchbioindustrialgroupInstitutMrieux,which fights against infectious diseases and cancers, through the complementary approaches of its companies in the fields of diagnosis, immunotherapy, food safety and nutritionmaintains global GMP facilities meeting U.S. and European regulatory standards, providingcGMPbiomanufactureofvirus-based oncolytic therapies, gene therapies, vaccines, and protein-based immunotherapeutic products. 1-888-257-6457, If to FDA: Oligonucleotide Therapeutics Society 3:55 A Multi-Dimensional RNA-Based Approach to Treat Genetic Disorders, John Androsavich, Ph.D., Senior Scientist, Translate Bio. The Annual Meeting is open to anyone interested or involved in oligonucleotide therapeutics. T: +44 (0)20 7827 6154 Not a member? Sponsors These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic. Seek feedback from industry, academia, and other stakeholders on the current progress and challenges in oligonucleotide research and drug development. Sign in.

ABLs CDMO services include bulk drug substance,. fill/finish of drug product, process and assay development, and bioanalytical testing. We will examine how genetic lesionsoften arise during the process of homology directed repair and point mutation resolution. The site is secure. In preclinical models, systemic administration of DCR-BCAT induced rapid increases in tumor T cells and dramatically improved responses to immunotherapy agents. Part 1NEW, Target Identification and Validation Part 1, Artificial Intelligence in Drug Discovery Certainly head and shoulders above the virtual meetings I have attended thus far, high marks for the team and all involved in rolling out the discussions! Muthiah (Mano) Manoharan, PhDSenior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Arthur Levin, PhDCSO, Avidity Biosciences, Lubo Nechev, PhDVice President Process and Analytical Sciences, Alnylam Pharmaceuticals, Ekkehard Leberer, PhDSenior Life Sciences Consultant, ELBIOCON, Key Contacts: Get Involved with OPT Congress, For more information on the conference, please contact:Gemma SmithSenior Conference DirectorCambridge Healthtech Institute+44 (0)7754-518-131Email: Gemma Smith, For sponsorship information, please contact:Aimee CrokeBusiness Development ManagerCambridge Healthtech Institute(+1) 781-292-0777Email: Aimee Croke, For media and association partnerships, please contact:Ellen FarrarSenior Marketing ManagerCambridge Healthtech Institute(+1) 781-972-5492Email: Ellen Farrar, 250 First Avenue, Suite 300Needham, MA 02494P: 781.972.5400F: 781.972.5425 DIA is offering both a live in-person and a live-stream virtual option for the DIA/FDA Oligonucleotide-Based Therapeutics Conference. Examples Conferences, and With INDsapproved by both U.S. FDA and China FDA, we have started clinical trials in both countries. Clinical results to date are promising, with multi-dosing resulting in stepwise, additive reduction in serum HBsAg. In 2020 Dr. Woodcock was asked to lend her expertise to Operation Warp Speed the initiative to develop therapeutics in response to the pandemic. Copyright 2022 Cold Spring Harbor Laboratory.

Provide a forum for open discussion between industry, academic, and government scientists regarding challenges and experiences encountered in oligonucleotide drug development. The Oligonucleotide Therapeutics and Delivery conference reveals the latest in oligonucleotide discovery and delivery, with presentations on targeted delivery mechanisms, oligonucleotide chemistries and important clinical advances. (Kris) Iyer, Ph.D., Co-Founder & CSO, Spring Bank Pharmaceuticals. These agents are designed to inhibit viral replication and lower all viral antigens. Global experts from big pharma and leading biotechs will explore the emerging therapeutic applications of oligonucleotides with case studies surrounding the discovery and development of oligonucleotide therapeutics as well as CMC and regulatory standpoints. Join your peers and industry experts as together we optimize drug design, delivery and performance, accelerate time to market and achieve commercial success. Ronald Wange, Ph.D. we design improved and local oligo therapeutics? News & Events for Human Drugs, Recalls, Market Withdrawals and Safety Alerts, 2022 FDA/DIA Oligonucleotide-based Therapeutics Conference - 04/25/2022. Listen to case studies presenting on pre-clinical and clinical research in areas such as oncology and what can be learned for future clinical trials Messenger RNA (mRNA) is an important new therapeutic modality. E: ngaloria@smi-online.co.uk, LinkedIn: SMi Pharma

Preference will be given to those submitting abstracts. Or you can disable cookies, but it will affect your experience. 10:35 Coffee Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced, Oligonucleotides for Cancer Immunotherapy, 11:20 RNAi-Mediated -Catenin Inhibition Promotes T Cell Infiltration and Potentiates Immune Checkpoint Blockade.