Australia's Future in Global Peptide Drug Development

Paul Watt, Chief Scientific Officer, Phylogica Ltd, Australia sat down with the TIDES team to discuss the future of global peptide drug development in Australia as well as the current challenges for intracellular drug delivery and the important roles that academic institutions have in the development and commercialization of peptide therapeutics. Below you will find a brief excerpt from this exclusive interview. To access the complete interview, click here.


In terms of investment and innovation, where do you see Australia's future in global peptide drug development?

Australians have had a long history in peptide discovery. For example the pioneering work of Professor Mario Geysen enabled the parallel synthesis of peptides in the early 80's. More recently Australia has accumulated multiple high achieving peptide scientists such as Paul Alewood (eg. synthesis toxin peptides including use of selenolanthionine bridges), Richard Lewis and Glenn King (ion channel active venom peptides) (including ion channel inhibitors). David Craik (cyclotides and structural biology of complex peptides), John Wade (neuropeptides/relaxins). Australian peptide innovation has been successfully commercialised with the establishment of multiple companies, including Mimotopes, Auspep, Protagonist and Phylogica. As many of these companies mature, I see a bright future in the development of the Australian peptide industry. In addition several peptides discovered in Australia have been subject of multiple alliances with big Pharma offering further commercial potential as they progress through clinical development.


To learn more from Paul, join him at TIDES, May 9-12, 2016 in Long Beach, CA. Where he will be discussing how to design oligonucleotides with better drug-like properties to accelerate your products from discovery/preclinical to the clinic and to market.  

Register for TIDES now and save $100, use the code B16180BLOG100.

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Peptides for CNS and Alzheimer's Disease Therapy

Exclusive TIDES 2016 interview with Dieter Willbold, Ph.D., Director, ICS-6 Structural Biochemistry, Forschungszentrum Jülich, Germany. Dr. Willbold gives us some exclusive insights into the topics he will be addressing onstage at the 18th annual TIDES in just 4 weeks time. Don't miss out on this record setting meeting; join over 800 global attendees over 4 days full of insights and learnings. Register for TIDES by Friday, 4/15 and save $300 - Use the code B16180BLOG.


Dr. Willbold Interview: 

What current challenges are you facing for advancing your promising peptide for Alzheimer's disease through clinical trials?

Our main focus at the moment is to finish all the necessary preclinical tox and safety tests in order to soon get permission for a clinical phase I study with our drug candidate.

One challenge for developing therapeutics like ours that target specific aggregate species, lies in the lack of suitable analytics to measure target engagement, because most methods don't differentiate between different aggregate sizes. In addition to cognitive behavioral tests, we ourselves have therefore developed novel methods to measure efficacy and target engagement .e.g. QIAD (short for quantitative determination of interference with aggregate size distribution; Brener et al., Scientific Reports 2015), which measures Abeta oligomer elimination efficiency.


What are the advantages of peptide therapeutics over small molecules, antibodies and other protein therapeutics?

Small molecules often lack specificity, and this can lead to serious side effects and autoimmune responses. On the other hand protein-based therapeutics like antibodies possess high target selectivity and are made of natural components, but they are very big and expensive molecules. They often suffer from low oral bioavailability.

Small peptides can offer the best of both worlds in some respects: They are more target specific than small molecules but easier to produce than protein based drugs. Our drug candidate is made solely from D-enantiomeric amino acid residues and has surprisingly good oral bioavailability.


Where do you see innovation happening in the peptide field over the next 5 years?

Innovation will certainly happen in the production of peptides (cost reduction) and in the formulation of peptides (increase oral bioavailability).

______________________________________________________

Reserve your seat today to attend TIDES 2016 in Long Beach, CA and hear Dr. Willbold deliver a live presentation "Efficiency and Oral Bioavailability of Abeta Oligomer Directed D-Enantiomeric Peptides Developed for Therapy of Alzheimer's Disease", where he will delve into small soluble Abeta oligomers are suspected to be the major toxic species responsible for development and progression of Alzheimer's disease (AD). We developed highly potent D-enantiomeric peptide compounds that specifically eliminate Abeta oligomers and improve cognitive performance and stop or slow down neurodegeneration of AD transgenic mice. Data on stability and oral bioavailability clearly support the superiority of D-peptides over L-peptides.

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Record Attendance Expected! Don't Miss IBC's 18th Annual TIDES

Register by Friday, April 15th and save $300! To save, use the code B16180BLOG100

Experts Provide Key Success Factors for Drug Development and Manufacturing:

Mixed-Incretin Receptor Biochemical Signaling at Multiple Receptors
Richard DiMarchi, Ph.D. Standiford H. Cox Professor of Chemistry, Indiana University

Drisapersen: Case Study
Robert Baffi, Ph.D. Executive VP, Technical Operations, BioMarin Pharmaceuticals

Quark's Long History of Oligonucleotide Development
Danny Zurr, Ph.D. Chairman and CEO, Quark Pharmaceuticals Inc. 

Technologies for Targeting Molecules to Sites of Diseases
John Reed MD, Ph.D Global Head of Roche Pharma Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd.


TIDES 2016 is on pace for record attendance with 800+ global attendees expected to attend this year’s meeting, held May 9-12, 2016 in Long Beach, California – will you be there?

Register your team today and access the science, technologies and contracts you need to accelerate your oligonucleotide and peptide products through the clinic towards commercial success.

Your registration grants you access to:

• 800+ attendees representing 25 countries help you forge new business and scientific collaborations
• 100+ speaker presentations across 5 tracks provide a customized agenda of solutions for your company’s current challenges
• 70+ exhibitors demonstrate the latest products and technologies to accelerate your therapeutic to market
• 40+ poster presentations keep you abreast of cutting-edge industry and academic research

Plus, be sure to register this week to gain access to Attendee Connect powered by partneringONE, the digital networking platform that allows you to view the full attendee list, pre-schedule meetings, and send private messages to attendees before, during and after the event.

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Exclusive Interview with Dr. Bruce Riser, CEO, BLR-Bio, LLC

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Bruce L. Riser, Ph.D., CEO, BLR-Bio, LLC for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

What are the biggest challenges of bringing new agents with novel targets to the market?

BR: There are really two big challenges when bringing new agents with novel targets to the market. First, many investors and potential partners are not familiar with the target, or they do not have the comfort that exists with an established target. Second, there can be misperceptions in the scientific community and in the biopharmaceutical industry where an agent is associated with another type of development creating a negative halo. On the first challenge, it is easy to see a proven target and to line up, jumping on the bandwagon with others trying to either win the race to launch first or to attempt to find a niche in an already established area. The problem of course is that lining up with everyone else does not provide the opportunity to develop and launch a true breakthrough therapy that can change the treatment paradigms and have a breakthrough impact on peoples' lives. Instead, investors and companies that line up for the established target often find they are trailing others to the market and are never able to successfully establish themselves in the market. It really takes a visionary to see the potential of a new target and have the insight and strength to invest in it. The second challenge, dealing with miss-associations of a novel therapy with other developments can be truly vexing. Our peptides target the novel CCN signaling pathway, leveraging the unique qualities of CCN3 to target the control of ECM changes in response to injury, blocking fibrosis and creating an environment for re-establishment of complete tissue homeostasis. By creating peptide libraries and conducting diverse in vitro and in vivo screens we have identified specific regions with the CCN3 protein responsible for blocking single, or multiple, pathways to fibroblast and cancer growth, and fibrosis formation. Unlike other products such as antibodies directed against the pro-fibrotic CCN2, that appear to partially block a single pathway, our novel therapy regulates vs. simply blocking, thus allowing reestablishment of the body's healthy equilibrium.

With several peptide products in the pipeline in preclinical development and a wide range of possible disease applications, how have you selected your lead indication target(s)?

BR: Having several products covering a wide variety of diseases is great and choosing lead indications to pursue is a wonderful challenge to have. In reality, the two lead indications that we have chosen to pursue have as much to do with the research that we have done with my team as it does the extreme health care need. My long background in academia, clinical nephrology/public health, and pharmaceutical drug R&D has driven the way we do drug discovery at BLR Bio. First, working to clearly understand the pathways to disease before selecting a target, and trying not to get caught up by targets in vogue. Then, testing our peptides in multiple related diseases before moving into the clinic. Our pipeline was created out of our long-term interest in fibrosis and the extracellular matrix in biology and disease, particularly in renal fibrosis as a complication of diabetes an area of great medical need. This has led to the selection of diabetic nephropathy as a main target for one of our lead products. Since our target, and the peptide inhibitor of our target, are involved in many other forms of fibrosis and other disease involving the miss-regulation of matricellular signaling, this creates other exciting therapeutic indications down the road. These include NASH, IPF, MD, and scleroderma to name a few.

I began my career in cancer research, and we know now that the extracellular matrix and matricellular signaling proteins such as the CCNs are critical also in the formation and resolution of tumors. The pro-fibrotic CCNs, CCN2 and CCN4 in particular have been shown to be important for the establishment and likely invasion and metastases in numerous tumors. The opportunity to use CCN3-based therapy to, not only halt the cancer, but also to target the microenvironment is an exciting one. Our company is particularly excited about the potential in pancreatic cancer. We have an established relationship with thought leaders in the cancer field and are moving forward with development work, partnering with some of these world experts. To add to the excitement of the therapies, our company also is developing a diagnostic/theranostic based on the same technology platform. It holds the promise of either stand-alone or companion diagnostics for development.

What are the key advantages of peptide therapeutics over small molecules, antibodies and other protein therapeutics?

BR: As you know, peptide-based drugs are one of the most rapidly growing areas in therapeutic development. More than 100 peptide-based drugs are on the market with several effective in cancer treatment. There is good reason for this. In size, falling between small molecule drugs and "biologics", they have many of the benefits of both while shedding many of the deficiencies. For example, because of their small size they gain many delivery benefits over biologics. First, better penetration of tissue at the site of action. This is crucial in fibrotic disease and cancer where there is a very strong barrier to penetration. Second, it allows for many more delivery options, including oral formulation. The earlier assumption of poor metabolic stability of peptides has just not proven to be the case. Although there is often rapid clearance from circulation, the previous assumptions about required PK by classic definition has also proven to be not relevant, since a specific and sustained biological activity at the target receptor is characteristic of many peptides. Unlike small molecules they are based on sequences that have evolved to react with specific receptors and often at very low concentrations. This results in very low off target effects. Last, since they can be chemically synthesized the ease and cost to manufacture are more nearly like that of small molecules. The majority of approved peptides are in the 15 amino acids (aa) range or smaller, and they treat a broad range of diseases.

How do you see academia and industry collaborating in the future to advance promising peptide therapeutics into commercially viable products?

BR: First, we think that academia will play a significant role in the creation of new peptide-based therapies in the future. Since the last decade has seen a decline in the number of newly approved drugs from large pharma, and recently there has been a strong trend for these same companies to dramatically reduce their discovery effort, this creates a large opportunity for novel peptide therapies and academic-initiated translational research. This does not necessarily mean that the new paradigm will be that large strategic companies will license early stage assets directly from Universities. Rather, we anticipate that the most promising discoveries will lead to the creation of start-up companies and the building of defendable IP. Then, large pharmaceuticals will court these start-ups as they progress to become true biotechnology companies with completion of phase I, phase II clinical trials or beyond. We think that the best of these emerging biotech companies will then expand their ability to do discovery and early stage development. They will also be able to clearly evaluate and in-license additional opportunities in academia. These are exactly the skills now lacking in big pharma. These successful discovery biotech's will become the future pipelines for drugs that will go to the mid and large pharma companies centered on late development and marketing. I envision BLR Bio as becoming one of these discovery companies able to contribute significantly to filling this gap.

Want to hear more from Dr. Riser? Join him this May in Long Beach, CA for the 18th annual TIDES - Save $300 when you register using the code B16180BLOG100. Click here to register. 

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The Current Value of the Global Peptide Therapeutics Market

"The number of companies specializing in the manufacture of therapeutic peptides has been shrinking over the last 15 years with fewer new companies entering the market than those leaving it."  

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Rodney Lax, Ph.D., Business Consultant, PolyPeptide Group for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA. 


In this interview, Dr. Lax discusses:

• - The current estimated value of the global peptide therapeutics market.
• - The key factors that contribute to the constant growth of the global peptide therapeutics market.
• - The advantages/disadvantages to a consolidated peptide manufacturing industry.
• - Which emerging markets are leading the forefront of peptide therapeutics research and development
• - Opportunities that are emerging due to recent advancements in manufacturing and delivery technology

 Access the complete interview here.

PLUS! As a blog reader, you are eligible to save $300 when registering for TIDES. Use the code B16180BLOG100 to activate your savings. Click here to register.

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Analytical Controls in an Evolving Technology and Regulatory Landscape

 
IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Rachel Orr, Senior Scientist, GlaxoSmithKline R&D, United Kingdom for an exclusive interview that delves into analytical controls in an evolving technology and regulatory landscape, the topic of her presentation at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.


1. What key analytical challenges do oligonucleotide developers need to overcome in order to achieve regulatory approval?

Oligonucleotides are inherently difficult to analyze - they are neither traditional, small nor large molecules, and the techniques and well established methodologies for pharmaceutical analysis are all focused on these distinct classes. In addition, there is very limited regulatory guidance when it comes to oligonucleotides, so there are no set rules to follow. There are often discussions as to whether oligonucleotide analysis should follow small molecule rules and regulations or align with biopharmaceuticals; opinions are divided.

With limited experience across the industry in marketing applications it is difficult to assess what is needed in order to gain regulatory approval and this is one of the biggest challenges in CMC oligonucleotide development. There is no recent precedence of global oligonucleotide marketing applications and as such it is difficult to ascertain what may and may not be acceptable. Close engagement with the regulators is something which helps to form strategies and controls and hopefully this will increase the likelihood of success of regulatory approvals going forwards.


2. How have advancements in technology improved the analytical process? Can you provide examples?

Since the last successful oligonucleotide marketing application there have been considerable enhancements in technologies. UHPLC is now common throughout the industry and techniques such as high resolution NMR and mass spectrometry are much more affordable and quick. These advances in technology mean that the resolution and robustness of methodologies can be much improved, however has a downside that the regulators may well expect us to present them with these improvements by first intent.

Increased understanding of non "typical" molecules throughout the industry has enabled wider thinking and collaboration between different pharmaceutical companies, with more people working together on the problem progress will inevitably come quicker. As more people engage with the concept of oligos and as technology continues to evolve, I anticipate a large shift in the way we look to control oligonucleotides analytically in coming years.


3. What are the most common mistakes that oligonucleotide drug developers encounter during CMC submissions?

Again, due to there being so few oligonucleotides which have gone through the full CMC process I think it is difficult to make too many sweeping statements, however the key issues that I have come across all stem from attempting to fit oligonucleotides into a box. Whether it is the small molecule group or the biopharmaceutical groups who take on the challenge of oligonucleotides, it is generally only a few "experts" within that team who work on the molecules. It is impossible to fit existing small or large molecule platforms onto oligonucleotides and a lot of time, resources and money can be wasted trying to do so! Due to the small pool of people working on the oligo projects it can also be challenging when it comes to elements such as wider reviews of submissions as the reviewers are not always as well informed about the challenges associated with oligonucleotide analysis. Education of these reviewers and key stakeholders is an activity that cannot be underestimated in time or importance! This emphasizes the benefits of cross-pharmaceutical company collaborations.


Want to hear more from industry experts like Rachel? See our exclusive TIDES speaker Q&A series here.

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Accelerate Your Peptide from Discovery to Commercialization

Accelerate your peptide product from early discovery through late-stage development and commercialization by attending IBC's 18th Annual TIDES meeting, held May 9-12, 2016 in Long Beach, California.

TIDES offers two tracks dedicated for peptide scientists to learn about the latest strategies and technologies needed to overcome challenges in targeting, drug discovery and peptide design, process development, regulatory, scale-up manufacturing, investment landscape and more:

Keynote Speaker:
Richard DiMarchi, Standiford H. Cox Professor of Chemistry, Indiana University
Mixed-Incretin Receptor Biochemical Signaling at Multiple Receptors

Track 1: Peptide Chemistry, Manufacturing & Controls
Speaking Organizations Include: Lonza, Bachem, Teva, Novartis, Peptron

Track 2: Peptide Discovery, Preclinical and Clinical
Speaking Organizations Include: Corden, Galena, Neon, Celerion, Phylogica

Learn about the Therapeutic Landscape of Peptides in 2016 and Beyond:
Rodney Lax, Ph.D., Business Consultant, PolyPeptide Group
Redefining the Therapeutic Peptide Business in the 21st Century

Plus, TIDES offers the largest and most focused exhibit hall for the peptide industry, where you can access 70+ vendor booths, 40+ posters and evening cocktail networking receptions to help grow your business. You won't want to miss out on this rare opportunity to connect with 800+ like-minded researchers from across the world.

As a reader of our blog, you are eligible to save $300 when registering for TIDES - Simply use the code B16180BLOG100. Click here to register.  

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From Critical Quality Attribute Assessment to Specification and Characterization

"The reason for assessing the critical quality attributes is not only to fulfill an expectation, but also because it serves as a better understanding of the product under development"

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Renata Varga, Ph.D., Scientist - Characterization, Analytical Sciences, Global Biological CMC, Teva Pharmaceutical for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

In this interview, Dr. Varga discusses;

• The key regulatory challenges facing peptide developers
• What regulators look for when establishing CQAs in genetics, and any variation between generics and novel therapeutics.
• Peptide innovation in the next 5 years

From Critical Quality Attribute Assessment to Specification and Characterization
Characterization and comparability assessment of peptide based generic products is driven by critical quality attributes (CQAs). Standard CQAs are identified based on regulatory requirements. Additional CQAs are determined by understanding the molecule itself: sensitive amino acids or which are involved in receptor-binding or mechanism of activation, are identified and based on uncertainty and impact the criticality of that attribute is defined. All standard- and additional CQAs are included in the specification while potential CQAs are further characterized by orthogonal techniques.

Access the complete interview

Register for TIDES now and save $500 - to save, use the code B16180BLOG100

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Efficient Chemoenzymatic Peptide Synthesis (CEPS) in Water

"When peptides are ligated in water, the crude reaction mixture can immediately be loaded on the preparative HPLC column to purify the product" 

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Timo Nuijens, Ph.D., Lead Scientist, EnzyPep BV, The Netherlands for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

Efficient Chemoenzymatic Peptide Synthesis (CEPS) in Water
We describe a novel, organic cosolvent-stable peptide ligase (peptiligase), which couples peptide fragments with exceptional efficiency in water. Because peptiligase does not need a particular recognition motif, this novel technology is traceless and very broadly applicable. Besides fragment condensation, the enzyme is able to efficiently catalyze head-to-tail cyclization of peptides to create macrocyclics. It has also been shown to couple peptide esters efficiently to proteins. Peptiligase could be a useful tool for the (industrial) synthesis of numerous (pharmaceutical) peptides and conjugates.

In this interview, Dr. Nuijens also discusses;

• The importance of fragment condensation of peptides in water
• What the future holds for the Netherlands in the global peptide drug development market
• The factors to consider when determining the optimal synthesis strategy when screening an enzyme

Access the complete interview

Register for TIDES now and save $500 - To save, use the code B16180BLOG100

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Peptide Vaccine Manufacturing | Interview with Dr. Mimoun Ayoub of CordenPharma

"The concept of designing immunomodulating peptide cocktails and trigger the immune response has been mainly initiated by biotech companies who owns more than 80% of the current cancer vaccine pipeline."

In a recent interview with TIDES, Mimoun Ayoub, Ph.D., Director, Global Peptides & Injectables Platform, CordenPharma, Switzerland, shifts the focus towards the topics that will be addressed in his talk at the upcoming TIDES event. In his talk he will be examining the peptide vaccines that are designed to activate B-cells and killer T-cells to recognize cancer cells as foreign agents. Cancer cells have surface protein modifications such as phosphorylations and glycosylations that differentiate them from healthy cells. The peptide epitopes used are usually fragments from these modified proteins. These "cocktails" of peptide fragments can be further engineered to increase their immune activity and plasma half-life by constraining the peptide backbone through introduction of cyclization or non-natural amino acids in the sequence. The production and analytical challenges related to cocktail peptide vaccines will be discussed.

In the interview, Dr. Ayoub also addresses:

• The role peptide vaccines play in the in the development of novel cancer therapies
• Regulatory challenges during the development and manufacturing of peptide vaccine "cocktails"
• Future delivery methods for administering peptide vaccines

Access the complete interview

To learn more from Dr. Ayoub, join him at IBC's 18th Annual TIDES event this May in Long Beach, CA where he will be chairing the Peptide Chemistry Manufacturing and Controls track as well as speaking in a session titled "Peptide Vaccine Manufacturing : Drug Substance and Drug Product CMC Challenges" Download the brochure to access the complete agenda and speaking faculty. 


Register for TIDES now and save $500 - To activate your savings, use the code B16180BLOG100  

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Impurity Guidelines to Oligonucleotides | An Interview with Andrew Teasdale of AstraZeneca

In a recent interview with TIDES, Andrew Teasdale, AZ Impurities Advisory Group Chair, AstraZeneca, United Kingdom examines the scope and applicability of both new guidance (ICH Q3D and ICH M7) and existing guidance (ICH Q3A / Q3B) to Oligonucleotides, looking into practical challenges and how they might be addressed and the potential application of generic, platform risk assessment approaches.


When discussing the impurities that face oligonucleotide developers, Andrew said, "The most common impurities in oligonucleotides are still related substances (oligomeric impurities)". However, this still begs to question what does one need to consider when mitigating risk for these impurities? Access the complete interview now.

Access the complete interview

To learn more from Andrew about oligonucleotide impurities, join him at IBC's 18th Annual TIDES event this May in Long Beach, CA where he will be speaking in his session titled "Assessment of the Relationship of Impurity Guidelines to Oligonucleotides - Opportunities for Rationale Approaches to Management of Risk". Download the brochure to access the complete agenda and speaking faculty.



Register for TIDES now and save $500 - To activate your savings, use the code B16180BLOG100

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Novel Delivery Strategies for Oligos and Peptides - $600 Savings End Friday, 1/29

Register by this Friday, January 29th and receive up to a $600 savings – Use the code B16180BLOG100 to save. Click here to register.

Recent advances in delivery strategies have paved the way for improved targeting, efficacy and patient usability for the oligonucleotide and peptide therapeutic industry. Ensure that your team is up-to-date on the latest drug delivery strategies for accelerated product development by attending IBC's 18th Annual TIDES meeting, held May 9-12, 2016 in Long Beach, California.

Register today to attend the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization.

Industry Experts Present Novel Delivery Strategies for Oligonucleotides and Peptides:


Centyrins as Targeting Agents for Next-Generation Modular Therapeutics
Vadim Dudkin, Ph.D., Scientific Director, Centyrex, Johnson & Johnson

The biophysical properties of Centyrins make them ideal as targeting agents for a variety of novel delivery technologies. Centyrex is exploiting this platform via novel therapeutic applications aimed at addressing outstanding challenges in...(continue reading)
________________________________________

Treatment Opportunities for Cystic Fibrosis Using Aerosolized Antisense Oligonucleotides
Shuling Guo, Ph.D., Director, Antisense Drug Discovery, Isis Pharmaceuticals

Cystic fibrosis (CF) is one of the most common life-threatening genetic diseases affecting ~30,000 people within US and ~70,000 worldwide. Lung disease is the major cause of morbidity and mortality in CF. Antisense oligonucleotides (ASOs) administered by aerosol delivery distribute broadly into...(continue reading)
________________________________________

ITCA 650 (Exenatide Delivered Continuously via a Matchstick-Size Subcutaneous Osmotic Mini-Pump) an Investigational Therapy for Type-II Diabetes
Scott Peterson, Ph.D., Executive Director, Corporate Development, Intarcia Therapeutics, Inc.

ITCA 650, an investigational therapy for T2DM currently in Phase 3 clinical trials, is an osmotic mini-pump that can subcutaneously deliver exenatide (a GLP-1 receptor agonist) for extended periods of time. This presentation will cover the technology behind...(continue reading)


For more session information, download the complete agenda.

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Announcing: TIDES 2016 Keynote Lineup | Brochure is Now Available

Download the complete TIDES event brochure now. 

IBC’s 18th Annual TIDES meeting is coming to Long Beach, CA May 9-12, 2016. TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization.

RVSP by January 29th, 2016 with code B16180LINK100 and receive $600 savings off the standard rate. Secure your best rate now.

Keynote and Featured Speakers Provide Novel Strategies to Accelerate Product Approval:

• Mixed-Incretin Receptor Biochemical Signaling at Multiple Receptors
• • Richard DiMarchi, Ph.D. Professor of Chemistry | Indiana University
• Drisapersen: Case Study
• • Robert Baffi, Ph.D., Executive VP, Technical Operations | BioMarin Pharmaceutical
• Quark's Long History of Oligonucleotide Development
• • Danny Zurr, Ph.D., Chairman and CEO | Quark Pharmaceuticals Inc.
• Technologies for Targeting Molecules to Sites of Disease
• • John Reed, M.D., Ph.D. Global Head | Roche Pharma, Switzerland

TIDES by the numbers:

• 800+ attendees representing global innovators, suppliers and thought-leaders who can expand your pipeline and grow your business
• 100+ case studies and new data presentations providing novel strategies to accelerate your product to market
• 70+ exhibitors showcasing new technologies to improve your process development, analytical and manufacturing/scale-up efforts

Sponsorship and Exhibition Opportunities
Showcase your expertise to 800+ global buyers by becoming an event sponsor or exhibitor at TIDES. A wide variety of options are available to meet your company's goals and budget. Contact Patrick Gallagher pgallagher@ibcusa.com for pricing and package details.

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Are You Keeping Up with Your Competition at TIDES?

Over 70 leading and emerging oligonucleotide and peptide companies will be Exhibiting, providing cutting-edge Spotlight Presentations and/or Sponsoring networking sessions at TIDES 2015, coming to San Diego this May. What do they know?

They know:

·         TIDES is the largest gathering of the oligonucleotide and peptide community where networking and business gets done. Over 50% of the attendees are President’s, VPs, C-Level, Directors and Managers that hold the true buying decision power in their hands. Meeting face-to-face with these decision makers will accelerate business, partnerships and sales.

·         Whether in the Exhibit Hall, between sessions or during the scheduled Networking Receptions, you have direct access to hundreds of leading minds and businesses.

TIDES is the leading source of advances in technology, strategies and case studies that span from R&D through Commercialization. Your company needs to be an important part of this community and conversation.

Why let your competition have all the glory?

·         Secure one of the remaining 10 exhibit spaces left >> Request an updated floor plan

·         Sponsor a Spotlight Presentation and prove that your company is a thought-leader in the industry. We have a few select spots remaining in the agenda. Contact me for subject and topic areas.

·         Brand your company through sponsorship of the TIDES Lounge or other branding opportunities.

·         Capitalize on this unique opportunity to highlight your company's expertise to 800+ qualified attendees from 25+ countries.   As our valued event sponsor/exhibitor, your company's brand awareness extends beyond our attendees to our thousands of targeted email subscribers, website visitors and event prospects.   Give me a call or shoot me an email today to discuss how your participation at this year's TIDES meeting will help you achieve your company's targeted growth goals.

Join us in San Diego next month. Register for TIDES today: http://bit.ly/1OBPwgA

Best,
The TIDES Team

Futurebiopharma.blogspot.com

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Visionary Keynote Interviews + New Poster Award at TIDES

TIDES is coming to San Diego in 6 weeks!  Get a sneak peek of this year's meeting by listening to exclusive interviews with a few of this year's visionary keynote speakers, who provide their perspectives on the current state of the oligonucleotide and peptide industry.

Start planning your team's trip to attend this year's meeting and access 800+ like-minded attendees, 100+ speaker presentations, 70+ exhibitors and 40+ scientific poster presentations.  The $200 registration savings deadline expires on April 10, so secure your seat today: http://bit.ly/1bxzMyX

Plus, all registered attendees gain immediate access to Attendee Connect, the partnering tool that allows you to communicate with fellow attendees before, during and after the meeting.

Stanley T. Crooke, M.D., Ph.D. CEO, Isis Pharmaceuticals, Inc.

The progress of technology has yielded new chemistries like Generation 2.5.  How will that improve the potential of antisense technology?

SC:  Second generation drugs today that we make are significantly better than second generation [drugs] of just a few years ago in that we get about twice as much potency and we have much better tolerated drugs and that's a product of advances in technology.  Generation 2.5, we would expect would give us an increase in potency of about ten-fold...(Listen to the full interview)

You say that you are focused on creating the maximum value for your drugs and technology.  Tactically, how are you going that?

SC:  I think focusing on maximum value is the only hope for the industry in general.  With the technology we have at Isis, we have the benefit of very rapidly being able to take genetic information and convert it to drug.  So step one is to...(Listen to the full interview)

Kunwar Shailubhai, Ph.D., Co-Founder and CSO, Synergy Pharmaceuticals, Inc.

Can you discuss the scientific rationale for developing a uroguanylin peptide analog to treat functional GI disorders?

KS: Two known agonists of GCC that are in clinical development are Linactotide, which is an FDA approved drug and our molecule, plecanatide for functional disorders/chronic constipation and IBS-C. Now, our molecule – plecanatide – is an analogue of uroguanylin whereas Linactotide is an analogue of e-coli enterotoxin. So, I would like to emphasize...(Listen to the full interview)

What challenges do you see the peptide industry facing in the next 5 years?

KS: The biggest challenge when we started working on this compound was to develop large-scale manufacturing.  The next thing that I believe is quite challenging for peptide drugs is formulation. Formulation should be done in a way that.....(Listen to the full interview)

The TIDES 2015 Poster Award

The TIDES 2015 Poster Award, sponsored by AUM LifeTech, Inc. will recognize TIDES 2015 poster presenters whose poster submission and research efforts demonstrate exceptional advances and breakthroughs in the following areas: discovery, pre-clinical and clinical aspects of oligonucleotide based therapeutic research including but not limited to mRNA silencing and manipulation, miRNAs, short and long non coding RNAs, exon skipping, delivery, genome edting, etc.

Three poster award recipients will receive $12,500, $7,500 and $5,000 respectively, in in-kind support/technology/products from AUM LifeTech, Inc. Through this support, awardees will have access to AUM’s next generation oligonucleotide technology that can be used to further awardee’s research. Please refer to www.aumlifetech.com for details on AUM’s RNA silencing and manipulation technology. Submissions from academic and research institutes are especially encouraged, as are submissions from industry. 

To apply for the poster award, you must be a registered TIDES attendee and submit your poster abstract by April 10, 2015 >> Submit your poster abstract today: http://bit.ly/1Iz2PND

Register by Friday, April 10 and receive a $200 Savings. Your $200 Savings Priority Code = 00DCL3R2B. Register here: http://bit.ly/1bxzMyX

Group rates are available for companies registering 4+ attendees.  Call 646-895-7445 to secure your team's seat today.

Sponsorship/Exhibition Opportunities: More than 70 leading and emerging oligonucleotide and peptide companies will be exhibiting or sponsoring this year's TIDES meeting.  Contact Sales Manager Steve Rooney at srooney@ibcusa.com to secure one of our few remaining exhibit spaces and/or spotlight speaking opportunities.

Cheers,

The TIDES Team

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[Video] Breakthrough Status: Are You Ready?

In today's featured presentation from TIDES 2014, Jeffrey Baker, Ph.D., Deputy Director, Office of Biotechnology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration talks about FDA SIA (Safety and Innovation Act). He presents a rundown of this act and talks about NDAs (Non-disclosure agreement), and BLAs (Biologics License Applications). Baker states that FDA SIA has made available a pathway for rapid approval of drugs critical to public health, however companies need to be prepared to manufacture and meet patient needs. Are you ready? For details, watch the presentation:



TIDES 2015 will take place May 3-6, 2015 in San Diego, California.  This year, Ravi S. Harapanhalli, Ph.D., Vice President, Technical, PAREXEL International and Former Branch Chief, Office of New Drug Quality Assessment, US FDA, will be on hand to present Progress in Advancement of RNAi Therapeutics.  For more information about this session and the rest of the program, download the agenda.  As a reader of this blog, when you register to join us and mention code XB15180BLOG, you can save 20% off standard rates.

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Announcing Your 2015 TIDES Keynote Lineup

IBC's 17th Annual TIDES meeting is coming to San Diego, CA on May 3-6, 2015.This is the leading forum for CMC, clinical and discovery scientists in the oligonucleotide and peptide industry to share best practices and new technologies to help you:

• • Accelerate your promising therapeutic to market
• • Ensure accurate CMC submissions
• • Comply with emerging regulatory expectations
• • Apply best practices from leading preclinical/clinical projects
• • Improve process development timelines

Download the preliminary agenda.

This year, we move coasts and provide you visionary keynotes sharing key success factors for drug development and manufacturing highlighting these areas:

• >Antisense Technology: Stanley T. Crooke, Ph.D., Chief Executive Officer, Isis Pharmaceuticals, Inc.
• >RNAi Therapeutics: John Maraganore, Ph.D., Chief Executive Officer, Alnylam Pharmaceuticals
• >Peptide Therapeutics: Torsten Hoffmann, Ph.D., Executive VP and CSO, Zealand Pharma A/S
• >Oral Peptide: Kunwar Shailubhai, Ph.D., Co-Founder & CSO, Synergy Pharmaceuticals, Inc.

And don't forget - as a reader of this blog, when you register to join us and mention code XB15180BLOG, you can save 20% off the standard rate!

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TIDES 2015 Speaker Deadline + Early Registration Offer

Few speaking slots remain at IBC's 17th Annual TIDES meeting, held on May 3-6, 2015 in San Diego, CA. If you are working in any of the following scientific areas below, we encourage you to submit a speaker proposal by Wednesday, November 5, 2014 for a podium presentation:

• -Oligonucleotide Manufacturing Technology, Analysis, CMC & Regulatory
• -Peptide Manufacturing Technology, Analysis, CMC & Regulatory
• -Oligonucleotide Discovery & Clinical Development
• -Peptide Discovery & Clinical Development

To see an in-depth look of what we’re looking for in each of these categories and submit a presentation proposal here,

Your abstract and presentation must describe scientific results and must not contain marketing or business development content. If you are interested in speaking but cannot make the November 5 deadline, please email Michael Keenan as soon as possible.

How else can you participate in TIDES 2015?

• - Early Registration Offer Ends January 23, 2015. Save up to $400 when you register.
• - Sponsorship/Exhibition Opportunities: Highlight your company's expertise to this global audience of oligonucleotide and peptide decision makers by becoming an event sponsor or exhibitor. Email Sherry Johnson at  reach her at 508-614-1451 for pricing and package options.

Have any other questions on this year’s event? Feel free to reach out to me at jpereira@iirusa.com.

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What are some of the issues have occurred in the past regarding consent and genetic studies?

Next week, the TIDES Event will take place in Providence, Rhode Island.  This week, we're revisiting some of the speaker interviews we conducted to prepare for the event. Today we look at the interview with Bertha deLanda, CIP - IRB/SCRO Panel Manager, Research Compliance Office at Stanford University.  She will be presenting Informed Consent and Genetic Research as a part of the Nucleic Acids Technologies for Molecular Diagnostics Workshop.

Today, Bertha answers the question:
What issues have occurred in the past regarding consent and genetic studies?

Well, unfortunately there are many examples of how both subjects and researchers and the general public are affected by mishaps and compliance issues that are very current. Havasupai Tribe for example, contributed their blood samples in the hopes of finding out more about the high incidence of pervasive diabetes within their population. The consent forms that the researchers obtained contained very vague language, which was reviewed by the IRB and found – at the time – to be adequate. However, the vagueness and terms and the lack of communication regarding the consenting process really caused downstream legal issues when the tribe later discovered that their samples were being used for other research that they objected to.

Also, there is the case of newborns being tested via blood spots in Texas where parents sued the institutions involved for not disclosing that this material would be stored indefinitely for undisclosed research purposes and without the plaintiff’s knowledge or consent.

Unfortunately, we have many other examples of situations where informed consent didn’t meet the standard of adequacy by the public eye. Since we depend on subjects providing samples as volunteers in order to continue our research, we must look at these past issues in order to avoid them in the future. I’ll touch on these examples during my talk and provide examples of consent forms to help delineate these points.

Read the rest of Bertha's interview here.

If you would like to learn more about this topic, join Bertha next week, May 12-15, 2014, in Providence, Rhode Island for the TIDES event. As a reader of this blog, when you register to join us and mention code XB14180BLOG, you can save 20% off the standard rate. Have any questions? Reach out to Jennifer Pereira.

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What is the main challenge with the informed consent process as it relates to genetic research process?

Next week, the TIDES Event will take place in Providence, Rhode Island.  This week, we're revisiting some of the speaker interviews we conducted to prepare for the event. Today we look at the interview with Bertha deLanda, CIP - IRB/SCRO Panel Manager, Research Compliance Office at Stanford University.  She will be presenting Informed Consent and Genetic Research as a part of the Nucleic Acids Technologies for Molecular Diagnostics Workshop.

Today, Bertha shares her thoughts on the question:

What is an IRB and what do they have to do with the consenting process?

Well, in IRB or Institutional Review Board, it is the independent ethics review board that’s responsible for reviewing, approving, continuing with or rejecting any research that involves either human subjects or their identifiable private health information. It is composed of at least five individuals, one who must be an unaffiliated or public member, one who must be a non-scientist and other members who are doctors, lawyers, teachers, businessmen, etc. Because my panel also oversees studies that involve stem cell research, my panel includes BioE Thesis and researchers with a strong stem cell background. Also, sometimes when necessary, a panel member who is experienced in assisted reproductive technology. 

My background is in the genetics research field. Many of the PCR drug technologies I was involved with were genetic research-based. Their purpose was to diagnose, treat, identify or even, in some cases, cure diseases so that they would inevitably go to human clinical trials. Therefore, before writing a protocol that involves work with human samples, the scientists and researchers should do their best to avoid the pitfalls and retrospective issues that can occur when consenting, potential participants or their samples.

Read the rest of Bertha's interview here.

If you would like to learn more about this topic, join Bertha next week, May 12-15, 2014, in Providence, Rhode Island for the TIDES event. As a reader of this blog, when you register to join us and mention code XB14180BLOG, you can save 20% off the standard rate. Have any questions? Reach out to Jennifer Pereira.

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