Delivery Of Nucleic Acid Therapeutics II: Biology, Engineering And Development

An ECI Conference Series

POSTPONED

Our apologies, but given the COVID-19 pandemic, we are postponing this conference to March 2022 date at the same hotel. 

For future meetings on delivery of Nucleic Acid Therapeutics, we direct you to the Oligonucleotide Therapeutics Society (OTS) annual meeting Sept. 27-30, 2020, in Montreal, Canada, where there will be two sessions on delivery: https://www.oligotherapeutics.org/2020-annual-meeting

Please accept our apologies for this inconvenience and we look forward to seeing you in 2022.

Sincerely,

Conference Chairs

Steve Dowdy (UCSD) and Laura Sepp-Lorenzino  (Intellia Therapeutics)         

March, 2022
Grand Hotel Minareto — Siracusa, Sicily

Keynote by Mano Manoharan, Ph.D., Alnylam

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About This Conference

Nucleic Acid Therapeutics (NATs), including ASOs, siRNAs, mRNA, Gene Therapy and Genome Editing, can precisely regulate, and potentially correct, gene expression in disease. As such, they can enable the development of novel therapeutic options for disease targets previously considered un-druggable by conventional small molecule and biologics means. 

The NAT therapeutic revolution is ALMOST upon us. While major advances and recent regulatory drug approvals have been made with liver, CNS and eye delivery, delivery outside of these tissues remains the bioengineering technological problem to solve. Even for the most advanced clinical candidates using lipid nanoparticles, currently less than 1% of the NAT cargo is escaping from the endosome. For viral delivery, novel vectors with specific tropism, improved transduction efficiency and safety remain a major need. It is widely recognized that there are significant gaps in our basic understanding of the mechanisms used by non- viral and viral delivery vehicles for tropism, uptake and trafficking in vivo, and how-to best harness this knowledge to advance delivery for therapeutic use. 

The ECI “Delivery of Nucleic Acid Therapeutics: Biology, Engineering and Development” conference will provide a forum for increasing our mechanistic understanding of the biological processes underpinning non- viral and viral NAT delivery, including molecular and cellular biology, virology, in vivo pharmacology and toxicology, rational optimization strategies, targeting, formulation, bioengineering and process development solutions for scale-up manufacturing, regulatory considerations and preclinical and clinical NAT programs. 

The conference will bring together biologists, chemists, pharmacologists, biophysicists, drug developers, nanotechnologists, process engineers from academia and industry developing viral and non-viral delivery strategies for the antisense, RNA interference, mRNA, Gene Therapy and Genome Editing, ex vivo and in vivo. 

The major topics to be discussed are: 

• Nucleic acid chemical modifications and conjugates to enable and enhance delivery
• Liposomes and polymeric nanoparticle delivery vehicles 
• Viral delivery technologies 
• Targeting 
• Mechanisms of cellular uptake and endosomal escape 
• Biophysical characterization methods 
• NAT delivery vehicle toxicology and ADME 
• Local and systemic NAT delivery case studies 

The program will consist of invited and abstract chosen oral presentations and poster presentations. 

Conference Organization

Conference Chairs

Steven F. Dowdy, Ph.D., Professor, Department of Cellular & Molecular Medicine, University of California San Diego School of Medicine, San Diego, CA 

Laura Sepp-Lorenzino, Ph.D., Executive Vice President, Chief Scientific Officer, Intellia Therapeutics, Cambridge, MA 

Keynote Address

Mano Manoharan, Ph.D., Senior Vice President, Distinguished Fellow, Innovation Chemistry, Alnylam Pharmaceuticals, Cambridge, MA 

Confirmed Speakers 

  • Paul Burke, Ph.D., Principal, Burke Bioventures LLC, Boston, MA
  • Pieter Cullis, Ph.D., FRSC, Professor, University of British Columbia, BC, Canada
  • Tony De Fourgerolles, Ph.D., CEO, Evox Therapeutics, Oxford, UK
  • Dirk Grimm, Ph.D., Professor, Hiedelberg University Hospital, Germany
  • Art Levin, Ph.D., Chief Scientific Officer, Avidity Biosciences, La Jolla, CA
  • Mark Kay, M.D./Ph.D., Professor, Depts of Pediatrics & Genetics, Stanford University, Palo Alto, CA
  • Anastasia Khvorova, Ph.D., Professor, RNA Therapeutics Inst., Univ. Mass. Medical School, MA
  • Tom Kirchhausen, Ph.D., Professor, Harvard Medical School
  • Melissa Moore, Ph.D., Chief Scientific Officer, Moderna Therapeutics, Cambridge, MA
  • Cyrus Safinya, Ph.D., Professor, Materials and Physics, UC Santa Barbara, CA
  • Margrit Schwarz Ph.D. MBA, CSO and Head of R&D, Genevant Sciences, Cambridge MA
  • Punit Seth, Ph.D., Vice President Medicinal Chemistry, Ionis Pharmaceuticals, Carlsbad, CA
  • Matt Stanton, Ph.D., Chief Scientific Officer, Generation Bio, Cambridge, MA
  • Ernst Wagner, Ph.D., Chair, Center for Nanoscience, Ludwig-Maximilians-Universität, Germany
  • Jesper Wengel, Ph.D., Professor, Nucleic Acid Center, Univ. Southern Denmark, Odense, Denmark 
  • Kristy Wood, Ph.D., Senior Director, Intellia Therapeutics
  • Anders Wittrup,M.D., Asst. Prof. Lund University, Sweden

This second conference on NAT delivery will be held at the Grand Hotel Minareto in Siracusa, Sicily, Italy. To receive the next mailing, please email laura.sepplore@intelliatx.com

Call for Abstracts

The major topics to be discussed are:

  • Mechanisms of cellular uptake and endosomal escape
  • Nucleic acid chemical modifications and conjugates to enable and enhance delivery
  • Liposomes and polymeric nanoparticle delivery vehicles
  • Viral delivery technologies
  • Targeting
  • Biophysical characterization method
  • Local and systemic nucleic acid therapies delivery case studies

Please use these titles to pre-select one or two sessions where you believe your work fits best.

Abstracts (one page maximum) that include specific results and conclusions to allow a scientific assessment of the proposed oral presentation are invited.  Please prepared your abstract according to this template: docx or doc.

Oral abstract submission deadline: TBA

Poster abstract submission deadline: TBA

Abstracts of all presentations will be made available to conference participants prior to the start of the conference.

Note: Only a limited number of oral presentation slots are available and thus all submissions for oral sessions will be considered for both oral and poster presentation.

Awards will be presented to the top student posters.

Venue and Transportation Information

The conference will be held at the Grand Hotel Minareto near Siracusa in Sicily, Italy.

The resort’s 93 rooms and 4 suites are cottages scattered around the luxurious Mediterranean grounds, a splendid setting dominated by the sea and the sky which provides a serene setting for conferences. The hotel has ample meeting space for both technical and poster sessions. Rooms are air-conditioned and have televisions, mini-bars, safes and other amenities.

Transportation

Transporation Options from the Catania Airport to the Grand Hotel Minareto

Siracusa is in the southeast corner of Sicily, on Sicily’s east coast, about an hour south of Catania.  The closest airport to the Grand Hotel Minareto is Catania-Vincenzo Bellini Airport (CTA, http://www.aeroporto.catania.it/?lang=eng). It is 71 km from the hotel (about a one-hour drive). 

You have several choices of transportation: Individual transfer, group transfer, train (and taxi), bus (and taxi), and rental car.

Nadia Davi (nadia.davi@liberty-int.com) of Liberty Incentives and Congresses, Italy is the contact person for those wanting to book transportation from the Catania Airport to the Gran Hotel Minareto.  Do not contact her for train, bus or rental cars.

Individual and Group Transfers

You can choose an individual transfer or a group transfer.  The group transfer will be organized by the expected time of arrival at Catania Airport within the range of one hour.  For example, if two people are arriving at 11:30 am and 6 people are arriving at 12:00 pm, then the transfer would be 8 people.  The rate of a “group” transfer is €45 per person for arrival hours between 6:00 am and 10:00 pm. For arrivals between 10:00 pm and 6:00 am there is a night supplement of €13.5 per person.

An individual transfer (for one – three persons) in a private vehicle with driver is €90.  The night supplement is €27

Other requests will be quoted individually by Ms. Davi.

Please use this form when contacting Ms. Davi. (Ben – link to the PDF from Liberty Travel)

By Train 

From Catania Station (Taxis can transport you from the airport to the train station), take a train to Syracuse (Siracusa). There are hourly trains from the train station in Catania to the train station in Siracusa.  The train ride is approximately 85 minutes. Taxis can be hired at the Siracusa station for transfer to the hotel (about a 10-minuted drive). For schedules and fares visit http://www.trenitalia.com 

Siracusa’s train station is on Via G. Rubino (where Corso Umberto I and Via Ermocrate come together) in the “New City” mainland.  It is just south of the archaeological zone and about a dozen blocks west of the bridges to Ortigia (the “old town” on an island).

By Bus

Interbus (www.interbus.it) runs buses to Sircusa regularly during the week from Catania to Siracusa and less frequently on Sundays.  Please visit their website for details. This is the most economical way, although you will need to add a taxi at each end of your journey.

By rental car from Catania Fontanarossa Airport 

(About 55 minutes) Get on E45 from SP701.  Follow E45 and the Catania-Siracusa highway (SS114) and take the “Siracusa Sud” exit from SS114 onto Via per Floridia/SS124 in Siracusa.  

Continue on Floridia/SS124 until the next roundabout where you take the 2nd right onto Via Antonio Ascari.  At the next roundabout (Largo Primo Levi), take the second exit onto Viale Pantanelli (essentially following the main road).  

At the roundabout at the Viale Paolo Orsi, turn to the right, Via Columba, (where there is a median strip of palms, and on the left you will see a McDonalds) and go straight until the crossroads where you make a right onto Via Elorina (on the left you will see a Bingo) which is now SS115.  

Pass the Agip and QB petrol stations on your right and after about 500 meters pass the QB, you will come to a roundabout where you will take the second exit onto Via Lido Sacramento. 

At the next roundabout, take the first exit and stay on Via Lido Sacramento. 

Continue straight onto Via La Maddalena which turns right and becomes Via del Faro Massollivieri.  You will see a sign for the Minareto and Nesos. 

Go straight on the coastal road and the hotel will be on the right.

Google directions from Catania Fontanarossa Aiport to Grand Hotel Minareto 

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General Information about ECI

Engineering Conferences International (ECI) is a not-for-profit, global engineering conferences program, originally established in 1962 that provides opportunities for the exploration of problems and issues of concern to engineers and scientists from many disciplines.

The format of the conference provides morning and late afternoon or evening sessions in which major presentations are made. Poster sessions will be scheduled for evening discussion as well. Available time is included during the afternoons for ad hoc meetings, informal discussions, and/or recreation. This format is designed to enhance rapport among participants and promote dialogue on the development of the meeting. We believe the conferences have been instrumental in generating ideas and disseminating information to a greater extent than is possible through more conventional forums.

All participants are expected both to attend the entire conference and to contribute actively to the discussions. The recording/photographing of lectures and presentations is forbidden. As ECI conferences take place in an informal atmosphere, casual clothing is the usual attire.

Smoking is prohibited at ECI conferences and conference functions.