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7:30am - 7:45am

Registration and Coffee

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Showing of Streams
Showing of Streams
10:00am - 10:45am

Networking Refreshment Break in Poster and Exhibit Hall

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Showing of Streams
12:15pm - 12:20pm
Transition to Spotlight Presentation Rooms

Transition to Spotlight Presentation Rooms

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Showing of Streams
12:50pm - 1:55pm

Networking Luncheon in Poster and Exhibit Hall

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Showing of Streams
3:30pm - 3:35pm
Close of TIDES Conference

Close of TIDES Conference

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7:30am - 7:45am 15 mins
Registration and Coffee
7:45am - 8:15am 30 mins
Spotlight Presentations 1
Breakfast Spotlight Presentation
8:25am - 8:30am 5 mins
Oligonucleotide Discovery, Preclinical and Clinical
Chairperson's Remarks
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.
8:25am - 8:30am 5 mins
Oligonucleotide Chemistry Manufacturing and Controls
Chairman’s Remarks
8:25am - 8:30am 5 mins
Peptide Discovery, Preclinical and Clinical
Chairman’s Remarks
  • Trishul Shah, MS - Director Business Development, PolyPeptide Group
8:25am - 8:30am 5 mins
Peptide Chemistry Manufacturing and Controls
Chairman’s Remarks
  • Trishul Shah, MS - Director Business Development, PolyPeptide Group
8:25am - 8:30am 5 mins
Drug Delivery Innovations and Strategies
Chairperson's Remarks
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.
8:25am - 8:30am 5 mins
mRNA Therapeutics and CRISPR Therapeutics
Chairman's Remarks
8:30am - 9:00am 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
RNAi Delivery: Overcoming a Billion Years of Evolutionary Defenses
  • Steven Dowdy, PhD - Professor, UCSD School of Medicine

RNAi therapeutics have great potential to selectively treat human disease, especially cancer. However, due to their 14,000 Dalton size and 40 negative phosphate charges, siRNAs have limited to no bioavailability to overcome a billion years of evolutionary defenses to prevent RNAs from entering cells. Consequently, delivery remains The Technological Problem to solve for development of RNAi therapeutics, especially escape from endosomes.

8:30am - 9:00am 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Regulatory Agency Questions and Learnings from Recent Commercial Submissions
  • Jennifer Franklin - Director, CMC Regulatory Affairs, Ionis Pharmaceuticals

Recent CMC questions received from global regulatory agencies for an antisense oligonucleotide drug will be detailed, along with a discussion of the response strategies that led to their successful resolution and ultimately to drug approval.  Regulatory challenges and learnings from the review and approval process in various regions will also be examined.  

8:30am - 9:00am 30 mins
Info
Peptide Discovery, Preclinical and Clinical
A New Source of Frameshift Neoantigens for Vaccines and Diagnostics
  • Stephen Johnston, PhD - Chief Executive Officer, Calviri Professor, Arizona State University

We have discovered that RNA processing in tumors is a rich, unrecognized source of neoantigens. We make peptide arrays representing all frameshift neoantigens made in tumors. This allows a direct readout of which tumor neoantigens the immune system recognizes. These arrays are useful in designing vaccines and for diagnosis of cancer.

8:30am - 9:00am 30 mins
Info
Peptide Chemistry Manufacturing and Controls
A New Source of Frameshift Neoantigens for Vaccines and Diagnostics
  • Stephen Johnston, PhD - Chief Executive Officer, Calviri Professor, Arizona State University

We have discovered that RNA processing in tumors is a rich, unrecognized source of neoantigens. We make peptide arrays representing all frameshift neoantigens made in tumors. This allows a direct readout of which tumor neoantigens the immune system recognizes. These arrays are useful in designing vaccines and for diagnosis of cancer.

8:30am - 9:00am 30 mins
Info
Drug Delivery Innovations and Strategies
RNAi Delivery: Overcoming a Billion Years of Evolutionary Defenses
  • Steven Dowdy, PhD - Professor, UCSD School of Medicine

RNAi therapeutics have great potential to selectively treat human disease, especially cancer. However, due to their 14,000 Dalton size and 40 negative phosphate charges, siRNAs have limited to no bioavailability to overcome a billion years of evolutionary defenses to prevent RNAs from entering cells. Consequently, delivery remains The Technological Problem to solve for development of RNAi therapeutics, especially escape from endosomes.

8:30am - 8:50am 20 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Inhibition and Degradation of Drug Targets Using bioPROTAC mRNAs – A Novel Approach with Broad Therapeutic Potential
  • Anthony Partridge, PhD - Principal Scientist, Early Discovery Pharmacology, Merck, Sharp & Dohme

To tackle historically intractable targets, we have developed a platform employing targeted degradation. Specifically, we have engineered fusion constructs involving two components I) mini-proteins/peptides with high-affinity against therapeutic targets linked to II) truncated E3 ligase receptors. These ‘bioPROTACs’ have proven broadly successful with many constructs showing robust degradation activity. Currently, we aim to apply this technology as research tools and therapeutically by pursuing delivery strategies of bioPROTAC mRNAs.

8:50am - 9:15am 25 mins
mRNA Therapeutics and CRISPR Therapeutics
Evaluation of Modified Interferon alpha mRNA Constructs for the Treatment of Non-melanoma Skin Cancer
  • Markus Mandler, PhD - CSO, Accanis Biotech
9:00am - 9:30am 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
Moving Antibody-Oligonucleotide Conjugates (AOCs) into Development
  • Andrew Geall, PhD - Vice President of Formulations, Analytics and Chemistry, Avidity Biosciences

Translating the AOC technology from the laboratory to the clinic will require technologic advancements in multiple areas including scale up, development of human payloads, selection of monoclonal antibodies that target human receptors and analytical techniques for assessing quality and stability. The presentation will cover how Avidity is tackling each of those areas.

9:00am - 9:30am 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Quality Development in European Early Access Approaches (PRIME)
  • René Thürmer, PhD - Deputy Head, Unit Pharmaceutical Biotechnology, BfArM Federal Institute for Drugs and Medical Devices

The European Medicines Agency (EMA) and the US FDA launched the PRIME and Breakthrough Therapy schemes to strengthen their support for the development of medicines that address unmet medical needs with the aim to help patients to benefit from these therapies as early as possible. The presentation will discuss quality challenges and possible scientific and regulatory approaches which could be used to facilitate development and preparation of robust quality data packages, to enable timely access to medicines for patients whilst providing assurance that patient safety and product quality are not compromised. Risk-based approaches regarding pharmaceutical development programmes including, e.g. product characterisation, specification setting, validation and stability testing as well as early identification of quality issues / attributes that are critical to the clinical use of the medicinal product will be highlighted.

9:00am - 9:30am 30 mins
Info
Peptide Discovery, Preclinical and Clinical
Improving Formulation Consistency of Individualized, Peptide-based Therapies Using Self-assembling Nanoparticles
  • Geoffrey Lynn, M.D, PhD - CEO, Avidea Technologies

Peptides have a broad range of physical and chemical properties, which can complicate manufacturing and lead to variable activity when used in individualized therapies. To overcome this challenge, Avidea has developed an approach for chemically programming peptide-based therapies to self-assemble into nanoparticles (SNP) with uniform properties (i.e. size, charge and drug loading) irrespective of peptide composition. The results of Avidea’s efforts to systematically develop a personalized cancer vaccine (“AVT01”) based on SNP technology will be presented as a case study.

9:00am - 9:30am 30 mins
Info
Peptide Chemistry Manufacturing and Controls
Improving Formulation Consistency of Individualized, Peptide-based Therapies Using Self-assembling Nanoparticles
  • Geoffrey Lynn, M.D, PhD - CEO, Avidea Technologies

Peptides have a broad range of physical and chemical properties, which can complicate manufacturing and lead to variable activity when used in individualized therapies. To overcome this challenge, Avidea has developed an approach for chemically programming peptide-based therapies to self-assemble into nanoparticles (SNP) with uniform properties (i.e. size, charge and drug loading) irrespective of peptide composition. The results of Avidea’s efforts to systematically develop a personalized cancer vaccine (“AVT01”) based on SNP technology will be presented as a case study.

9:00am - 9:30am 30 mins
Info
Drug Delivery Innovations and Strategies
Moving Antibody-Oligonucleotide Conjugates (AOCs) into Development
  • Andrew Geall, PhD - Vice President of Formulations, Analytics and Chemistry, Avidity Biosciences

Translating the AOC technology from the laboratory to the clinic will require technologic advancements in multiple areas including scale up, development of human payloads, selection of monoclonal antibodies that target human receptors and analytical techniques for assessing quality and stability. The presentation will cover how Avidity is tackling each of those areas.

9:15am - 9:40am 25 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Messenger RNA Therapeutics for Bone Regeneration and for Pulmonary Diseases
  • Christian Plank, PhD - Chief Technology Officer, Ethris GmbH

mRNA transcript therapy is envisaged to enable novel therapeutic approaches for numerous disease targets. Ethris is specialized in topical administration of mRNA. Evidence for therapeutic efficacy of mRNA therapeutics in bone regeneration and a genetic pulmonary disease will be presented.

9:30am - 10:00am 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
Ocular Delivery of siRNA Conjugates: Efficient and Durable Gene Silencing of TTR after Single Intravitreal Administration of siRNA Conjugates
  • Jayaprakash Nair, PhD - Associate Director, Research, Alnylam Pharmaceuticals

Ocular transthyretin produced locally in retinal pigment epithelium (RPE) and ciliary epithelia (CE) can cause amyloid deposits, resulting in significant visual impairment, including blindness, in approximately 10% of hereditary transthyretin-mediated (hATTR) amyloidosis patients. Liver transplantation does not resolve ocular amyloidosis and liver-directed therapies are not expected to be efficacious against ocular manifestations. Silencing the expression of TTR in the eye using small interfering RNA’s (siRNAs) would represent a novel treatment approach for development. Here we show that siRNA conjugates targeting TTR can be delivered to the relevant cell types in the eye and produce efficient and durable gene silencing after single intravitreal administration. Preclinical efficacy and safety of siRNA conjugates in rodents and nonhuman primates will be presented.

9:30am - 10:00am 30 mins
Oligonucleotide Chemistry Manufacturing and Controls
Spinraza Regulatory CMC Experiences
  • Mia Kiistala - Associate Director Regulatory Affairs, Biogen
9:30am - 10:00am 30 mins
Peptide Discovery, Preclinical and Clinical
Pioneering NeoAntigen Immunotherapies: via ATLAS & Rethinking Peptide NeoAntigens
  • Daniel DeOliveira, PhD - Director of Pharmaceutical Sciences & Manufacturing, Genocea Biosciences
9:30am - 10:00am 30 mins
Peptide Chemistry Manufacturing and Controls
Pioneering NeoAntigen Immunotherapies: via ATLAS & Rethinking Peptide NeoAntigens
  • Daniel DeOliveira, PhD - Director of Pharmaceutical Sciences & Manufacturing, Genocea Biosciences
9:30am - 10:00am 30 mins
Info
Drug Delivery Innovations and Strategies
Ocular Delivery of siRNA Conjugates: Efficient and Durable Gene Silencing of TTR after Single Intravitreal Administration of siRNA Conjugates
  • Jayaprakash Nair, PhD - Associate Director, Research, Alnylam Pharmaceuticals

Ocular transthyretin produced locally in retinal pigment epithelium (RPE) and ciliary epithelia (CE) can cause amyloid deposits, resulting in significant visual impairment, including blindness, in approximately 10% of hereditary transthyretin-mediated (hATTR) amyloidosis patients. Liver transplantation does not resolve ocular amyloidosis and liver-directed therapies are not expected to be efficacious against ocular manifestations. Silencing the expression of TTR in the eye using small interfering RNA’s (siRNAs) would represent a novel treatment approach for development. Here we show that siRNA conjugates targeting TTR can be delivered to the relevant cell types in the eye and produce efficient and durable gene silencing after single intravitreal administration. Preclinical efficacy and safety of siRNA conjugates in rodents and nonhuman primates will be presented.

9:40am - 10:00am 20 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Novel mRNA Immunotherapies
  • Robert Jabulowsky, PhD - Deputy Head of Project Management, BioNTech AG

Due to its unique characteristics, mRNA may be easily employed for potent cancer immunotherapy. The potential of mRNA-based cancer therapeutics will be presented and discussed.

10:00am - 10:45am 45 mins
Networking Refreshment Break in Poster and Exhibit Hall
10:45am - 11:15am 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
Targeting RNA with Antisense Oligonucleotides to Treat Diseases of the CNS
  • Frank Rigo, PhD - Executive Director, Neuroscience Drug Discovery, Ionis Pharmaceuticals

The genetic cause for many neurodegenerative diseases is known, yet for most of them no therapies exist that directly exploit this information. An attractive approach to reduce gene expression is via the use of ASOs that harness the RNase H mechanism, which have shown success in both preclinical models and in clinical trials for diseases of the CNS.

10:45am - 11:15am 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Saving GalNAc: Improvements on Solution-Phase Conjugation of Oligonucleotides
  • Simon Breitler, PhD - Process Chemist, Roche

The conjugation of therapeutic oligonucleotides with GalNAc-clusters for hepatocyte targeting is a well-known method in today’s business. GalNAc-triclusters are relatively large in size and their physico-chemical properties render them difficult and costly to synthesize on kg-scale. It is therefore important that the conjugation step with the oligonucleotide is efficient as possible in order to not waste valuable GalNAc-triclusters. The talk will explain how we were able to reduce the amount of GalNAc-tricluster necessary for conjugation to an aminohexyl-modified oligonucleotide by almost 50% on multi-100g scale.

10:45am - 11:15am 30 mins
Info
Peptide Discovery, Preclinical and Clinical
FlowVax: A Synthetic, Adjuvanted Microsphere Peptide Vaccine Platform
  • Scott Burkholz - Bioinformatics Data Scientist, FlowPharma

Flow Pharma is developing the FlowVax adjuvanted microsphere peptide vaccine platform to generate a safe, reliable immune response to unmodified class I and class II epitopes using its patented Size Exclusion Antigen Presentation Control (SEAPAC™) technology. FlowVax eliminates the need for single long peptides, reducing manufacturing issues and potential frame-shift errors from SLP post processing in the antigen presenting cell. The adjuvants used are each part of FDA approved vaccines.

10:45am - 11:15am 30 mins
Info
Peptide Chemistry Manufacturing and Controls
FlowVax: A Synthetic, Adjuvanted Microsphere Peptide Vaccine Platform
  • Scott Burkholz - Bioinformatics Data Scientist, FlowPharma

Flow Pharma is developing the FlowVax adjuvanted microsphere peptide vaccine platform to generate a safe, reliable immune response to unmodified class I and class II epitopes using its patented Size Exclusion Antigen Presentation Control (SEAPAC™) technology. FlowVax eliminates the need for single long peptides, reducing manufacturing issues and potential frame-shift errors from SLP post processing in the antigen presenting cell. The adjuvants used are each part of FDA approved vaccines.

10:45am - 11:15am 30 mins
Info
Drug Delivery Innovations and Strategies
Targeting RNA with Antisense Oligonucleotides to Treat Diseases of the CNS
  • Frank Rigo, PhD - Executive Director, Neuroscience Drug Discovery, Ionis Pharmaceuticals

The genetic cause for many neurodegenerative diseases is known, yet for most of them no therapies exist that directly exploit this information. An attractive approach to reduce gene expression is via the use of ASOs that harness the RNase H mechanism, which have shown success in both preclinical models and in clinical trials for diseases of the CNS.

10:45am - 11:15am 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Nucleoside-modified mRNA Immunization against Infectious Diseases
  • Norbert Pardi, PhD - Research Assistant Professor of Medicine, University of Pennsylvania

Lipid nanoparticle-encapsulated nucleoside-modified mRNA vaccines have demonstrated immunogenicity and protective efficacy against various infectious pathogens such as Zika, ebola and influenza viruses. Dr. Pardi’s presentation will give an update on the recent findings of this promising novel vaccine type.

11:15am - 11:45am 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
Extrahepatic Targeting and the Future of Oligonucleotide Therapeutics
  • David Rozema, PhD - Head of Therapeutic Chemistry, Empirico Therapeutics

Empirico is building a next-generation therapeutics company founded on utilizing huge biological datasets, human genetics and programmable biology to power novel target discovery and development. Oligonucleotides can be an ideal translational partner for targets identified through human genetics, however extrahepatic delivery of oligonucleotides is challenging. Oligonucleotide development for diseases requiring delivery outside of the liver will rely on the identification of efficient targeting ligands and/or the use of local administration.

11:15am - 11:45am 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Control of Oligonucleotide Drugs: Product Quality Attribute Assessment to Ascertain Criticality
  • Robert Duff, PhD - Principal Scientist, Attribute Sciences, Amgen

The presentation will focus on the assessment of the critical quality attributes for siRNA as based on mechanism of action, forced degradation experiments and the literature. The principles of Quality-by-Design (QbD) apply so the attributes must be assessed and scored for criticality. A severity score reflects the expected or perceived impact on the drug’s safety or efficacy if the attribute levels were to be out of control (elevated or reduced in comparison to expected levels).

11:15am - 11:45am 30 mins
Peptide Discovery, Preclinical and Clinical
FDA Regulatory View on Personalized Medicine Concepts for Therapeutic Peptides
  • Syed Husain, PhD - Research Chemist, Division of Cellular/Gene Therapies, Office of Tissues and Advanced Therapies, CBER, FDA
11:15am - 11:45am 30 mins
Peptide Chemistry Manufacturing and Controls
FDA Regulatory View on Personalized Medicine Concepts for Therapeutic Peptides
  • Syed Husain, PhD - Research Chemist, Division of Cellular/Gene Therapies, Office of Tissues and Advanced Therapies, CBER, FDA
11:15am - 11:45am 30 mins
Info
Drug Delivery Innovations and Strategies
Extrahepatic Targeting and the Future of Oligonucleotide Therapeutics
  • David Rozema, PhD - Head of Therapeutic Chemistry, Empirico Therapeutics

Empirico is building a next-generation therapeutics company founded on utilizing huge biological datasets, human genetics and programmable biology to power novel target discovery and development. Oligonucleotides can be an ideal translational partner for targets identified through human genetics, however extrahepatic delivery of oligonucleotides is challenging. Oligonucleotide development for diseases requiring delivery outside of the liver will rely on the identification of efficient targeting ligands and/or the use of local administration.

11:15am - 11:45am 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
mRNA Based Vaccines Expressing RSV are Immunogenic and Protective in Preclinical Models of Respiratory Syncytial Virus Infection
  • Andrew Geall, PhD - Vice President of Formulations, Analytics and Chemistry, Avidity Biosciences

Respiratory syncytial virus (RSV) is the leading cause of severe lower respiratory tract infections worldwide. In collaboration with Moderna, we are developing a vaccine consisting of a modified mRNA (Moderna Technology), formulated in a lipid nanoparticle. Preclinical models of immunogenicity (mice, cotton rats, African Green Monkeys (AGM)) and protection (cotton rats, AGM) were utilized to evaluate a panel of mRNA vaccines targeting different forms of RSV F. The leading candidate, a full-length, prefusion stabilized version of RSV F was demonstrated to be immunogenic and protective in these models. These preclinical data indicate that mRNA vaccines targeting RSV are a promising vaccine approach and should be evaluated in clinical trials.

11:45am - 12:15pm 30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Inhibition of Glutathione S-Transferase P (GSTP) for the Treatment of KRAS-Driven NSCLC using a Novel siRNA Lipid Nanoparticle
  • Roger Adami, PhD - Senior Director, Pharm. Sci, Nitto BioPharma, Inc.
11:45am - 12:15pm 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Delivering Answers Through Innovation
  • Brian Carothers - Vice President and General Manager, Agilent Technologies Inc

Over the past 16 years the oligo therapeutic field has seen continued growth in many areas.  The number of companies has increased from 40 to 142 and programs from 121 to 468 meaning more than 180 specific diseases are being targeted by therapeutic oligo programs today.  This presentation will introduce Agilent Nucleic Acid Solutions Division (NASD) newest facility in Frederick, CO that will more than double our commercial manufacturing capacity to meet the increasing global demand today and in the future.

11:45am - 12:15pm 30 mins
Peptide Discovery, Preclinical and Clinical
Late Breaking Presentation
11:45am - 12:15pm 30 mins
Peptide Chemistry Manufacturing and Controls
Late Breaking Presentation
11:45am - 12:15pm 30 mins
Drug Delivery Innovations and Strategies
Inhibition of Glutathione S-Transferase P (GSTP) for the Treatment of KRAS-Driven NSCLC using a Novel siRNA Lipid Nanoparticle
  • Roger Adami, PhD - Senior Director, Pharm. Sci, Nitto BioPharma, Inc.
11:45am - 12:15pm 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Program on Self-amplifying RNAs for Prophylactic Vaccination
  • Dong Yu, PhD - Director & Function Head, Antigen Identification, GSK Vaccines

GSK has developed a self-amplifying mRNA (SAM) based vaccine platform, which offers an opportunity to tackle disease areas that remain challenging to a traditional vaccine approach. SAM vaccines have been shown to elicit robust, persistent, and broad immune responses against different disease targets in multiple animal models. This presentation will overview the principal, preclinical proof of concept, and future direction of this platform at GSK.

12:15pm - 12:20pm 5 mins
Transition to Spotlight Presentation Rooms
12:20pm - 12:50pm 30 mins
Info
Spotlight Presentations 1
Sumitomo’s Approach for Large-Scale Synthesis of Long RNA Oligos with High Purity
  • Yuki Tanaka - Researcher, Sumitomo Chemical Co Ltd
12:20pm - 12:50pm 30 mins
Info
Spotlight Presentations 2
Intertek Scientific Briefing
12:50pm - 1:55pm 65 mins
Networking Luncheon in Poster and Exhibit Hall
1:55pm - 2:00pm 5 mins
Oligonucleotide Discovery, Preclinical and Clinical
Chairperson's Remarks
1:55pm - 2:00pm 5 mins
Oligonucleotide Chemistry Manufacturing and Controls
Chairman’s Remarks
1:55pm - 2:00pm 5 mins
Peptide Discovery, Preclinical and Clinical
Chairman’s Remarks
  • Christopher Rhodes, PhD - President and CEO, Drug Delivery Experts
1:55pm - 2:00pm 5 mins
Peptide Chemistry Manufacturing and Controls
Chairman’s Remarks
  • Marc Jacob, Ph.D. - Business Development Manager, Purification Technologies - North America, Phenomenex
1:55pm - 2:00pm 5 mins
Drug Delivery Innovations and Strategies
Chairman’s Remarks
  • Christopher Rhodes, PhD - President and CEO, Drug Delivery Experts
1:55pm - 2:00pm 5 mins
mRNA Therapeutics and CRISPR Therapeutics
Chairman’s Remarks
2:00pm - 2:30pm 30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Update on the DCR-PHXC and DCR-HBV Clinical Programs and Other Pipeline Progress
  • Bob Brown, PhD - Chief Scientific Officer, Senior Vice President, Research, Dicerna Pharmaceuticals
2:00pm - 2:30pm 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Characterization of Critical Quality Attributes of mRNA
  • Penggao Duan, PhD - Principal Scientist, Moderna Therapeutics

mRNA product-related impurities include short mRNAs resulting from either premature termination of transcription or in-process degradation, uncapped mRNAs, and point mutations, insertions/deletions. Multiple case studies utilizing a combination of biochemical and biophysical methods will be discussed on characterization of mRNA product- related impurities and variants for successful development of mRNA therapeutics

2:00pm - 2:30pm 30 mins
Info
Peptide Discovery, Preclinical and Clinical
Adhesive Dermally Applied Microarray (ADAM): Experience in Osteoporosis, Diabetes and Migraine
  • Mahmoud Ameri, PhD - Vice President, Research and Development, Zosano Pharma

Zosano has developed intracutaneous microneedle systems for parathyroid hormone, glucagon, and zolmitriptan. Hitherto, we reported data from a 6-month study in post-menopausal women with osteoporosis, reversal of insulin-induced hypoglycemia in Type 1 Diabetes Mellitus subjects and recently, announced results of a 589-subject placebo-controlled trial in subjects with migraine. Intracutaneously administered zolmitriptan was highly effective for the treatment of migraine, with statistical significance compared to placebo achieved for the two co-primary endpoints of pain freedom at 2 hours, and most bothersome symptom absence at 2 hours. In conjunction with our ongoing long term safety study we believe this trial will form the basis for approval. The results in the three diverse patient populations exhibit the effectiveness of the intracutaneous microneedle systems for delivering drugs rapidly and yielding pharmacologic effects quickly. It also shows that this route of administration has an immense possibility for the rapid delivery of a number of therapeutic compounds.

2:00pm - 2:30pm 30 mins
Info
Peptide Chemistry Manufacturing and Controls
Industrial Peptide Purification – Challenges and Concepts
  • Ralf Eisenhuth, PhD - Process Manager Technology Transfer and Chromatography, Bachem AG

The presentation will illustrate how Bachem faces the growing demands for purity and understanding of the behavior of peptide APIs by continuously investigating and optimizing downstream processes. The available technologies for downstream processing of crude synthetic peptides will be discussed in the context of maximizing throughput at low costs and constant quality.

2:00pm - 2:30pm 30 mins
Info
Drug Delivery Innovations and Strategies
Adhesive Dermally Applied Microarray (ADAM): Experience in Osteoporosis, Diabetes and Migraine
  • Mahmoud Ameri, PhD - Vice President, Research and Development, Zosano Pharma

Zosano has developed intracutaneous microneedle systems for parathyroid hormone, glucagon, and zolmitriptan. Hitherto, we reported data from a 6-month study in post-menopausal women with osteoporosis, reversal of insulin-induced hypoglycemia in Type 1 Diabetes Mellitus subjects and recently, announced results of a 589-subject placebo-controlled trial in subjects with migraine. Intracutaneously administered zolmitriptan was highly effective for the treatment of migraine, with statistical significance compared to placebo achieved for the two co-primary endpoints of pain freedom at 2 hours, and most bothersome symptom absence at 2 hours. In conjunction with our ongoing long term safety study we believe this trial will form the basis for approval. The results in the three diverse patient populations exhibit the effectiveness of the intracutaneous microneedle systems for delivering drugs rapidly and yielding pharmacologic effects quickly. It also shows that this route of administration has an immense possibility for the rapid delivery of a number of therapeutic compounds.

2:00pm - 2:30pm 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Characterization of Critical Quality Attributes of mRNA
  • Penggao Duan, PhD - Principal Scientist, Moderna Therapeutics

mRNA product-related impurities include short mRNAs resulting from either premature termination of transcription or in-process degradation, uncapped mRNAs, and point mutations, insertions/deletions. Multiple case studies utilizing a combination of biochemical and biophysical methods will be discussed on characterization of mRNA product- related impurities and variants for successful development of mRNA therapeutics

2:30pm - 3:00pm 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
The Oligonucleotide Agent BC 007 Neutralizes Agonistic Autoantibodies Directed Against β1-Adrenoceptors - Data of Clinical Phase 1 Trial
  • Annekathrin Haberland, PhD - Director, Regulatory Affairs, Berlin Cures GmbH

BC007 neutralizes functional pathogenic autoantibodies against G-protein coupled receptors (GPCR-AAb) including beta1-AAbs, which are the high prevalence cause of heart failure. At moderate doses, persistent neutralization of AABs can be obtained after infusion. This was found in the Phase-1 safety tests including older GPCR-AAb positive subjects. BC007 was well tolerated and showed no clinically relevant side effects. Transient elevated aPTT to subclinical values was observed in some subjects. BC007 is a powerful new drug for neutralizing GPCR-AAb with a favorable side effect profile that is being tested as the first causative drug for patients with β1-AAb associated heart failure.

2:30pm - 3:00pm 30 mins
Info
Oligonucleotide Chemistry Manufacturing and Controls
Defining Critical Quality Attributes for RNA Nanoparticle Manufacturing from Extended Characterization
  • Heinrich Haas, PhD - Vice President, RNA Formulation & Drug Delivery, BioNTech AG

For clinically applicable RNA nanoparticle products, criteria to define quality and specifications must be defined. Further to methods applied in regular quality control, advanced characterization can provide valuable data for defining critical product parameters and help to justify specifications. Here we present results from RNA nanoparticle products, where conditions for assembly were systematically varied. Combined analysis of result from various independent methods (e.g., small angle X-ray scattering (SAXS), binding studies, spectroscopic techniques) enabled to correlate structural parameters with biological activity and targeting selectivity. Such information can be helpful for defining and justifying quality criteria for the pharmaceutical products.

2:30pm - 3:00pm 30 mins
Info
Peptide Discovery, Preclinical and Clinical
Engineered Amphiphilic Peptides Enable Delivery of Protein and CRISPR Cargoes to Cells
  • David Guay, PhD - Research Director, Feldan Therapeutics

Among biologic cargoes, proteins offer promise but are limited by a lack of efficient delivery methods. We developed amphiphilic peptides that enable robust delivery of proteins to cells by a simple co-incubation. These carrier peptides are optimized to deliver peptides, antibodies and CRISPR ribonucleoprotein complex to cells, including hard-to-modify Natural Killer cells and mouse airway epithelia.

2:30pm - 3:00pm 30 mins
Info
Peptide Chemistry Manufacturing and Controls
Aggregation of Peptides in Preparative Chromatography
  • Anthony Zidell - Analytical Chemist, Corden Pharma

Aggregation of synthetic peptides is problematic for preparative chromatographic purification. In this work, we present various cases in which avoiding aggregation has played a commanding role in the development and scale-up of purification processes.

2:30pm - 3:00pm 30 mins
Info
Drug Delivery Innovations and Strategies
Engineered Amphiphilic Peptides Enable Delivery of Protein and CRISPR Cargoes to Cells
  • David Guay, PhD - Research Director, Feldan Therapeutics

Among biologic cargoes, proteins offer promise but are limited by a lack of efficient delivery methods. We developed amphiphilic peptides that enable robust delivery of proteins to cells by a simple co-incubation. These carrier peptides are optimized to deliver peptides, antibodies and CRISPR ribonucleoprotein complex to cells, including hard-to-modify Natural Killer cells and mouse airway epithelia.

2:30pm - 3:00pm 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Defining Critical Quality Attributes for RNA Nanoparticle Manufacturing from Extended Characterization
  • Heinrich Haas, PhD - Vice President, RNA Formulation & Drug Delivery, BioNTech AG

For clinically applicable RNA nanoparticle products, criteria to define quality and specifications must be defined. Further to methods applied in regular quality control, advanced characterization can provide valuable data for defining critical product parameters and help to justify specifications. Here we present results from RNA nanoparticle products, where conditions for assembly were systematically varied. Combined analysis of result from various independent methods (e.g., small angle X-ray scattering (SAXS), binding studies, spectroscopic techniques) enabled to correlate structural parameters with biological activity and targeting selectivity. Such information can be helpful for defining and justifying quality criteria for the pharmaceutical products.

3:00pm - 3:30pm 30 mins
Info
Oligonucleotide Discovery, Preclinical and Clinical
Anti-FGF2 aptamer RBM-007 in phase I/IIa trials for wet AMD
  • Yusuf Ali, PhD - CEO, Ribomic USA

RBM-007 is a novel oligonucleotide-based aptamer with potent anti-FGF2 activity. Intravitreal administration of RBM-007 in animals demonstrated anti-angiogenic and anti-scarring effects, consistent with a therapeutic effect desired in the treatment of wet (exudative) AMD. We have entered phase I/IIa clinical trial in the US. This clinical trial, named as SUSHI is an open label, dose escalation study of safety and ocular tolerability of a single intravitreal injection of RBM-007 in subjects with exudative age related macular degeneration.

3:00pm - 3:30pm 30 mins
Oligonucleotide Chemistry Manufacturing and Controls
Late Breaking Presentation
3:00pm - 3:30pm 30 mins
Info
Peptide Discovery, Preclinical and Clinical
Exploiting a Novel Cell Death Mechanism for Selective Killing of Cancer Cells Upregulating Homologous Recombination
  • Maria Soloveychik, PhD - CEO, SyntheX, Inc.

At SyntheX, we have developed ToRPPIDO, a plug-and-play drug discovery platform for the identification of short peptide sequences and macrocycles that are capable of disrupting or bridging intracellular protein-protein interactions (PPIs) of interest in a cell-based system. Homology-directed DNA repair (HDR) plays a crucial role in maintaining genomic stability in cancer cells and is typically induced by oncogenes such as Myc, CycE, and KRas. In accordance, overexpression of HDR pathway components correlates with poor prognosis and chemo-resistance in most tumors, including currently untreatable pancreatic and biliary tract cancers. Application of our screening platform, ToRPPIDO, towards a crucial PPI within the HDR pathway led to the discovery of STX100. Subsequent derivatives of STX100 that are cell penetrant and proteolytically stable were tested for selective activity against various cancer cell lines that overexpress HDR. Results from a panel of over 30 cancer and primary cell lines confirm selective cancer cell killing activity of STX100 derivatives in vitro. STX100-mediated cancer cell death is independent of canonical cell death mechanisms (apoptosis, necroptosis, pyroptosis, ferroptosis, etc.). Rather, the mechanism exploits the differential abundance of the HDR target in cancer cells relative to normal tissue to elicit an acute calcium-dependent cell death upon binding of STX100 to its target. ADME/PK studies indicate favorable in-vivo characteristics and further pre-clinical development work is currently ongoing.

3:00pm - 3:30pm 30 mins
Info
Peptide Chemistry Manufacturing and Controls
High-Throughput Approaches to Column Screening for the Preparative Purification of Peptides
  • Steven McIntyre - Peptide Process Development Department Manager, Almac Group

This presentation will detail a novel approach that Almac employ in process development for the selection of preferred conditions for peptide purification. Along with high throughput approaches, data is interpreted statistically which has allowed for a shorter path through process development and more robust purification methods being employed in manufacture. The new approach will be exemplified with case studies

3:00pm - 3:30pm 30 mins
Info
Drug Delivery Innovations and Strategies
Exploiting a Novel Cell Death Mechanism for Selective Killing of Cancer Cells Upregulating Homologous Recombination
  • Maria Soloveychik, PhD - CEO, SyntheX, Inc.

At SyntheX, we have developed ToRPPIDO, a plug-and-play drug discovery platform for the identification of short peptide sequences and macrocycles that are capable of disrupting or bridging intracellular protein-protein interactions (PPIs) of interest in a cell-based system. Homology-directed DNA repair (HDR) plays a crucial role in maintaining genomic stability in cancer cells and is typically induced by oncogenes such as Myc, CycE, and KRas. In accordance, overexpression of HDR pathway components correlates with poor prognosis and chemo-resistance in most tumors, including currently untreatable pancreatic and biliary tract cancers. Application of our screening platform, ToRPPIDO, towards a crucial PPI within the HDR pathway led to the discovery of STX100. Subsequent derivatives of STX100 that are cell penetrant and proteolytically stable were tested for selective activity against various cancer cell lines that overexpress HDR. Results from a panel of over 30 cancer and primary cell lines confirm selective cancer cell killing activity of STX100 derivatives in vitro. STX100-mediated cancer cell death is independent of canonical cell death mechanisms (apoptosis, necroptosis, pyroptosis, ferroptosis, etc.). Rather, the mechanism exploits the differential abundance of the HDR target in cancer cells relative to normal tissue to elicit an acute calcium-dependent cell death upon binding of STX100 to its target. ADME/PK studies indicate favorable in-vivo characteristics and further pre-clinical development work is currently ongoing.

3:00pm - 3:30pm 30 mins
mRNA Therapeutics and CRISPR Therapeutics
Late Breaking Presentation
3:30pm - 3:35pm 5 mins
Close of TIDES Conference