Tuesday, March 31, 2015

BDP Week 2015: BPSA Roundtable

By: Maribel Rios, Managing Editor of BioProcess International

The Bio-Process Systems Alliance (BPSA) presented a single-use applications roundtable on Monday, 30 March 2015, at the BioPharmaceutical Development and Production (BDP) Week meeting in Huntington Beach, California. Mark Petrich, PhD (Merck & Co., Inc.) moderated the panel consisting of Jerry Martin (Pall Corporation), Jeffrey Carter, PhD (GE Healthcare), James Vogel (The BioProcess Institute), and Todd A. Kapp (Parker-Hannifin Corporation). Martin started with a historical review of the BPSA, a trade organization formed in 2005 and now consisting of 46 member companies (suppliers and end users) whose goal is to “advance single-use worldwide and facilitate implementation of single-use technologies.” 

Carter and Vogel then reviewed BPSA’s active and recent projects, consisting of the following:
·         Single-Use Manufacturing Component Quality Test Matrices (currently under final review and should be available for the BPSA Annual Summit, 13-15 July 2015, in Washington DC)
·         Change Notification Practices (currently in the initial stages of documenting)
·         Extractables (currently continuing collaboration with other organizations such as ASTM, ASME-BPE, and USP)

·         Quality Agreement Template for Single-Use Biopharmaceutial Manufacturing Products (published July 2014, currently soliciting feedback from users of the template, with possible future revisions)
·         Particulates Guide, a 12-chapter compendium addressing why particulates are an issue, how to identify them, and so on (currently soliciting further input)

The roundtable concluded with Knapp presenting the future focus of BPSA, including the annual summit and its new scholarship fund.


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Thursday, March 26, 2015

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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Wednesday, March 18, 2015

Antigen Express on Cancer Immunotherapy and Peptides

In today's featured interview from TIDES 2014, Eric von Hofe, Ph.D., President, Antigen Express, Inc. discusses peptides, cancer immunotherapy, peptide vaccines, and his experience at the TIDES 2014 event. He states that peptides constitute the minimum unit required to generate in response to solid advantages when developing therapeutic materials and that peptides “rev-up” the immune system to go out to be specific canto cells. Do you want to learn more? Watch this interview:


TIDES 2015 will take place May 3-6, 2015 in San Diego, California.  At this year's event, we're offering two days of content focusing on Peptide Chemistry Manufacturing and Controls and Peptide Discovery, Preclinical and Clinical featuring insights from companies such as PolyPeptide Group, Amgen, NOXXON Pharma, Zealand Pharma and more. For more this year's program by downloading 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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Wednesday, March 11, 2015

Advances in siRNA Developing Delivery Solutions

In today's featured interview from TIDES 2014, Jared A. Gollob, M.D., Vice President, Clinical Research, Alnylam Pharmaceuticals, Inc. discusses exciting advances in siRNA.  He says there are several different delivery formulations and the company goes after targets they have confidence in.  He also talks about focusing on moving siRNA therapies into Phase III trials.  To learn more, watch the interview:


TIDES 2015 will take place May 3-6, 2015 in San Diego, California.  This year,John Maraganore, Ph.D.; Chief Executive Officer, Alnylam Pharmaceuticals, will be giving the keynote presentation Strategies and Challenges in the Development of Oligonucleotide and Peptide Drugs: Perspectives on DrugDevice Combinations and Drug Product.  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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Wednesday, March 4, 2015

New Tools for Genome-Wide Functional Screens Needed to Develop New Drugs

Guest Post: Paul Diehl, PhD, Director of Business Development at Cellecta - Paul will be speaking at IBC's Genome Editing Applications conference March 18-19 in Boston.


Despite the need for new innovative therapies, the rate of novel drug development has remained level with about 35-40 new approvals each year (see figure below). One crucial factor restricting the rate of new drug discovery is the limited set of therapeutic gene targets. There are approximately 1,500 approved therapeutic compounds, but they target only about 300 gene products. Most new compounds in testing are directed toward these same known targets.

However, the potential number of drug targets is at least an order of magnitude greater. DrugBank associates over 4,000 targets with the 7,500 compounds in its database, and GeneCards catalogues over 10,000 genes that have data indicating they “cause, predispose, or protect from diseases.” Why does the current pharmacopeia only exploit less than 10 percent of the potentially targetable gene products then?

The problem is that there is still too much unknown about how most gene products actually work. A tremendous amount of genetic information has been amassed and deposited into online databases. Data about how, under certain conditions in some systems, transcription of certain genes increase or decrease, proteins that interact, and genes that are mutated more frequently in certain systems. Almost all of this data, however, is simply correlative. These massive databases simply do not contain the key information required to really understand how genes produce biology. No amount of deep and thorough analysis of the massive amount of information currently stocked away can illuminate how most genes function because there is a critical dearth of information indicating which genes actually drive the biology.

This lack of information about which genes are responsible for biological responses and disease progression stems from the difficulty in obtaining this data experimentally. Data is needed that causally links gene products to phenotypic changes. This requires assays that assess how the perturbation of certain genes changes the responses or characteristics of cells in a model system, and there are few experimental approaches to generate this sort of data efficiently.

Some techniques, such as genomic recombination technologies like transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), can provide the needed data by disrupting or otherwise altering genetic targets in a precise manner. However, functional analysis tools must be able to assay the roles of large numbers of genes simultaneously. Collecting data on this scale requires parallel measurements in genome-wide functional assays, which is difficult with these gene manipulation techniques.

There are really only two technologies that currently form the basis of techniques for broad-based genetic screens that assess the functions of large numbers of genes in a single assay: RNA interference (RNAi) and CRISPR knockout screens.

Although RNAi and CRISPR loss-of-function screening approaches are effective, their potential to elucidate gene function remains somewhat limited. One limitation is that either approach only looks at the effects produced by the disruption of a single gene within the system. In reality, though, it is rare that one gene produces one trait. Most phenotypes are caused by multiple interacting genes. While loss-of-function screens can be designed to look for interacting genes, such as Bassik, et al. has shown, paired gene screens can only be run on a limited set of a few hundred genes, so efficient combinatorial assessments of interacting genes across the genome is really out of reach at the moment.

Also, disruption of gene function by knockdown or knockout provides only a limited range of functional assays. Genes may also be activated or otherwise functionally modulated to produce biological responses. Although RNAi can only be used for loss-of-function screens, CRISPR has shown some potential in screening for the activation or gain of genetic function. There is the potential, then, for the development of more sophisticated CRISPR-based screens that assay for genetic changes activating new pathways or bringing about novel biological transformations that may well elucidate disease development.

Thus, while RNAi and CRISPR loss-of-function screens provide one of the few effective approaches currently available for genome-wide functional screening, they are not sufficient to meet the daunting challenge of understanding how genes produce biology. Progress in identifying novel therapeutic targets will continue at a glacial pace until more advanced techniques are developed to address the challenge of teasing out the complex genetic networks that drive and control cellular responses and processes.

  
New Drug Approvals and R&D Expenditures on Annual Basis. The number of new drugs with novel mechanisms approved each year by the FDA (left axis) as compared with the annual expenditures reported by pharmaceutical companies in the 2014 Biopharmaceutical Research Industry Profile published by PhRMA (http://www.phrma.org/profiles-reports).



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Application Space for Peptides

In today's featured interview from TIDES 2014, Patrick C. Reid, Ph.D., Chief Scientific Officer, PeptiDream, Inc of Japan talks about how a lot of work involving peptides are not published yet. That kind if info is obtained in this type of conference. He also states that the therapeutic side has commercial potential, application space for peptides are limitless, and that he does not see any disease that can’t be therapeutically targeted with peptides. He expects peptides to represent against therapeutic modality. To learn more, watch the interview here:


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 Strategies and Challenges in the Development of Oligonucleotide and Peptide Drugs: Perspectives on DrugDevice Combinations and Drug Product.  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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Friday, February 27, 2015

Has disposable technology been tested on a large scale yet?

Biopharmaceutical Development and Production Week speaker Siddhartha Shrivastava, Ph.D., Senior Scientist, Downstream Process Development, Patheon recently took some time to sit down with us and discuss his upcoming presentation centered around downstream processing.  Today, he shares  the importance of disposable membrane chromatography systems for mAb purification.


Has disposable technology been tested on a large scale yet at Patheon? 

As I said, there are two parts. One is the clarification and we are testing Clarasolve Filters and  he second part is we are replacing chromatographic columns with membranes. So, for our process --- in essence, we are not reinventing the wheel because Clarasolve Filters are from Millipore and the membranes that we are using are from Natrix, one of our collaborators. 
So, we have not reinvented the wheel. What we have done is we have merged both these technologies to come up with a hybrid process which is far more effective and far cheaper compared to the classical platform or traditional platform methods. 
Having said that, we have not yet tested the full process at scale. But, Clarasolve has been using multiple commercial products and they are being used multiple times successfully both by Patheon and by many other companies, as well. And Natrik members are also employed in many of the clinical trials. So, individual contributors are already being tested and are proven in the market that they are liable to be used for the production of human injectibles. We have mix them to produce the whole process and, of course, --- as you know, Patheon is a CDMO and we have got ample opportunities to test them and we are in the process of testing them. But, of course, they are not yet tested at scale.

In this interview Dr. Shrivastava also discusses disposable technology, why it's more appealing than traditional technology and it's cost effectiveness, .  Download the entire interview here.


Dr. Shrivastava will be presenting Disposable Membrane Chromatography Systems as a Platform for mAb Purification on Thursday, April 2 at Biopharmaceutical Development and Production Week.  As a reader of this blog,when you register to join us March 30-April 2, 2015, you can save 20% off standard rates when you mention code XB15155BLOGJP! Have any questions? Reach out to me at jpereira@iirusa.com.


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Wednesday, February 25, 2015

[Video] ALN-TTR Programs for Transthyretin Amyloidosis

In today's featured presentation from TIDES 2014, Jared A. Gollob, M.D., Vice President of Clinical Research at Alnylam Pharmaceuticals, Inc. presents how explains how Transthyretin Amyloidosis is formed and how it develops throughout the body.  Transthyretin (TTR) amyloidosis is caused by the deposition of liver-derived mutant and/or wild-type TTR in peripheral nerves, GI tract and the heart. Patisiran (ALN-TTR02), which is being developed for familial amyloidotic polyneuropathy (FAP), is a systemically administered lipid nanoparticle (LNP) formulation of a small interfering RNA (siRNA) targeting wild-type and all mutant forms of TTR that is currently in Phase 3. ALN-TTRsc, which is being developed to treat familial amyloidotic cardiomyopathy (FAC), is a subcutaneously administered N-acetylgalactosamine (GalNAc)-conjugated siRNA also targeting all forms of TTR that is currently in Phase 2. This presentation will review the clinical results to date with both patisiran and ALN-TTRsc. To learn more, watch the actual 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 Strategies and Challenges in the Development of Oligonucleotide and Peptide Drugs: Perspectives on DrugDevice Combinations and Drug Product.  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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Friday, February 20, 2015

How much more cost effective is a disposal technology approach?

Biopharmaceutical Development and Production Week speaker Siddhartha Shrivastava, Ph.D., Senior Scientist, Downstream Process Development, Patheon recently took some time to sit down with us and discuss his upcoming presentation centered around downstream processing.  Today, he sheds light on the cost effectiveness of disposable technology.


How much more cost effective is this approach?
Sid: To answer this question I would like to explain the mAb or the industrial standard of gold standard of antibody purification. So, of course, as I said that requires a full battery of chromatographic columns that would be paired with the expensive resins. Among those expensive resins, the major resins which are used for antibody purification is Protein A, which is super expensive. So, that can be a significant. A 100 liter column would run upwards of around $1.6 to $2.5 million dollars. So, it is super expensive and the whole process costs millions of dollars. But, in the approach that we are going to present, you will see that we have replaced these expensive chromatographic columns with our collaborator company Natrix’s, hydrogel membranes, which has reduced the cost significantly. 
And we have also change the classical centrifugation approach to purify the harvest material or the depth filtration material with high capacity new material from Millipore called: “Clarasolve filters”. With the combination of these two systems, we have seen and we have estimated that there would be a significant savings in cost that would run upwards of millions of dollars in whole process. There is a significant chances that this process is very lucrative and very appealing and is also working very nicely.

In this interview Dr. Shrivastava also discusses the appeal of disposable technology and how it applies to the large scale.  Download the entire interview here.

Dr. Shrivastava will be presenting Disposable Membrane Chromatography Systems as a Platform for mAb Purification on Thursday, April 2 at Biopharmaceutical Development and Production Week.  As a reader of this blog,when you register to join us March 30-April 2, 2015, you can save 20% off standard rates when you mention code XB15155BLOGJP! Have any questions? Reach out to me at jpereira@iirusa.com.


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Wednesday, February 18, 2015

[Video] TIDES: AZD9150 and its Effect on Tumors

In today's featured interview from TIDES 2014, David C. Blakey, Ph.D., the Chief Scientist of Oncology iMED at AstraZeneca discusses the next generation of antisense molecules, AZD9150 and its effect on tumors and why TIDES is a great place to be for the industry. He also talks about bigger traction, which tackle lower uptake tissues and a combination of both peptides and nucleotides. To learn more, watch this interview:



This year at TIDES 2015, AstraZeneca's Regina Fritsche-Danielson, Ph.D., Associate Professor and Senior Director and Head of Bioscience Heart Failure Department will present Modified mRNA as a Potential Therapeutic for Patients with Cardiovascular Disease.  For more information on 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 the standard rate.


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Friday, February 13, 2015

Why is disposable technology more appealing compared to conventional technology?

Biopharmaceutical Development and Production Week speaker Siddhartha Shrivastava, Ph.D., Senior Scientist, Downstream Process Development, Patheon recently took some time to sit down with us and discuss his upcoming presentation centered around downstream processing.  Today, he looks at the appeal of disposable technology.

Why is disposable technology more appealing compared to conventional technology?
Continuing from where I left off in my last sentence, as I said if the product has got a long shelf-life, a long demand, that makes sense for us to invest in it and create a good infrastructure, a good footprint and bear the high operational and capital expenditure. But, if the product is in the development phase and you need to have some quick lots of drugs so that we can try clinically, that does not make sense. And, by moving to the disposable technology we are also reducing the chances of cross contamination.

As I said before, again, cleaning, and associated cleaning validation, basically reduced maintenance costs, reduced lead times, increased speed of implementation and time to market , that can be significantly be reduced because we have reduced significantly the time of the processing over the 30 some processes. And we have also reduced the labor costs because of the low cleaning requirement or cleaning validation requirement. And, of course, low space requirement and footprint requirement. That is a main reason why disposable technology is more appealing and more and more people want to go towards that.

In this interview Dr. Shrivastava also discusses disposable membrane chromatography systems, it's cost effectiveness, and how it applies to the large scale.  Download the entire interview here.


Dr. Shrivastava will be presenting Disposable Membrane Chromatography Systems as a Platform for mAb Purification on Thursday, April 2 at Biopharmaceutical Development and Production Week.  As a reader of this blog,when you register to join us March 30-April 2, 2015, you can save 20% off standard rates when you mention code XB15155BLOGJP! Have any questions? Reach out to me at jpereira@iirusa.com.


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Wednesday, February 11, 2015

[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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Friday, February 6, 2015

What is the importance of disposable membrane chromatography systems for mAb purification?

Biopharmaceutical Development and Production Week speaker Siddhartha Shrivastava, Ph.D., Senior Scientist, Downstream Process Development, Patheon recently took some time to sit down with us and discuss his upcoming presentation centered around downstream processing.  Today, he shares  the importance of disposable membrane chromatography systems for mAb purification.

What is the importance of this work?
Sid: Well, the topic as name suggest --- the non-chromatographic platform for mAb purification. As we can see, around 70-75% of those therapies in the market are based on antibodies and the classical way in which industry approaches (this is) to purify them is a bed chromatography approach. What that means is that you need to have a good platform of bed chromatography columns with some costly resins. Of course, we need to have a lot more space, a big footprint resulting in high capital expenditure, as well as operational expenditures for those platforms to purify the mAb.
It is good for a product which has got a long shelf-life or is high in demand, but for the products which have got a low demand or are only for therapeutic or clinical use or clinical trials, that kind of expenditure does not make sense. So, we are trying to focus that part, the drugs which have got a low demand and the product that needs fast turnover, we are trying to develop the technology which will be, in essence, a disposable or single-use so that we would have low validation cost, low cleaning cost and low installation cost and of course, what that would mean are low leading times on the material process that would, of course, mean the product cost would be reduced significantly. 
So, this is the essence and the target of the work that we did. Here at Patheon we are trying to generate a uniform platform matter for the mAB purification using this technique. This will be described in my presentation, as well.
In this interview Dr. Shrivastava also discusses disposable technology, it's cost effectiveness, and how it applies to the large scale.  Download the entire interview here.


Dr. Shrivastava will be presenting Disposable Membrane Chromatography Systems as a Platform for mAb Purification on Thursday, April 2 at Biopharmaceutical Development and Production Week.  As a reader of this blog,when you register to join us March 30-April 2, 2015, you can save 20% off standard rates when you mention code XB15155BLOGJP! Have any questions? Reach out to me at jpereira@iirusa.com.


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Thursday, January 29, 2015

BDP Week Football Spectacular: Save 49% when you register to join us at BDP Week!

This Weekend Only: Register for Biopharmaceutical Development & Production Week by Sunday, February 1 with priority code BDP15SB49 and you will save 49% (up to a $1800+ savings) off the standard rates.

Co-Located with:
Single-Use Applications & Flexible Facilities
March 30 - April 2, 2015
Hyatt Regency Resort & Spa 
Huntington Beach, CA
Mention priority code BDP15SB49

BDP Week provides you an immersive 4-day learning and networking experience—that will help you to:
  • • Get focused learning on Single-Use Applications and Flexible Facilities from these two established, co-located events.
  • • Discover technological and scientific advances improving cost, speed and productivity in process development
  • • Gain strategies to avoid pitfalls, maximize efficiencies and alleviate bottlenecks with elaborate coverage of manufacturing technologies for emerging biologics.
  • • Apply new found knowledge gained from 180+ speakers and demo new technologies from 70+ exhibitors for immediate advancements of your process and products.
  • • Connect with 800+ biopharmaceutical development and manufacturing peers to, swap ideas, and generate business outcomes with fun, casual networking functions.

Questions? Email Jennifer Pereira.

*This promotion is only valid January 29- February 1, 2015 only. Offer cannot be applied retroactively to confirmed paying registrants and cannot be combined with any other discounts or promotions. All registrants and guests are subject to IBC approval. 


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Friday, January 23, 2015

Genetic Engineering: It's Not Frankenstein

Today's guest post comes from Genome Editing Applications sponsor Horizon Discovery.

Genetic Engineering: It's Not Frankenstein
Author: Hannah Murfet (PCQI, BSc). Quality and Regulatory Manager

Genetic engineering often faces unjustified bad press with regard to genetically modified crops, however genetic engineering continues to provide great promise for drug discovery, assessment and production. We must apply care and regulation to allow continued advances to focus of human treatment and not enhancement, science should not allow us to create a monster.

Genetic engineering put simply is the adjustment of the instructions of our cells, the smallest functional unit of life. The genome is the sequence of information required to code for an organism. this consists of a long code represented by 4 letters (G, A, T and C). The sequence of these letters codes for the proteins and how often they are produced. Changes in the coding of these 4 letters take a range of forms from single letter changes, insertions and duplications.

Genetic engineering is routinely applied in medical treatment, particularly with the mass production of human insulin in bacteria since 1982 (1). In this example the gene for insulin was inserted into bacteria, the bacteria then produce insulin protein which can then be purified for medicinal use. The future of this technology has continued with antibody therapy, where immune proteins are engineered and produced for targeted treatment of cancer and autoimmune disease.

Scientists are able to make use of changes in coding to further our understanding of disease models such as cancer, where changes in the 4 letters can lead to changes in the way our cells behave (2). Cell and animal models can be used in early stage drug trials to determine effectiveness and resistance.

The future of genetic engineering takes this a stage further to target the cells in our bodies. This technology makes use of a virus to insert a gene to treat disease where the coding defective, Glybera is the first drug of this kind to be recommended for approval (3). The cost of these therapies is currently high, but the field of genetic engineering is continuing to evolve and new technologies such as CRISPR are being adopted (4).

Right now all the work in this field is creating some positive enhancements for medicine. With careful control and regulation the prospects for drug development and treatment have massive potential, however we must take care of the ethical implications of genetic alteration, particularly anything that may lead to human enhancement - we certainly don't want to create any monsters!

(1) http://www.brighthub.com/science/genetics/articles/21983.aspx

(2) http://www.oapublishinglondon.com/article/607

(3) http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.html

(4) http://www.bio-itworld.com/2014/5/22/advances-genetic-engineering-american-society-microbiology-meeting.html



Genome Editing Applications will take place will take place March 18-19, 2015 in Boston.  Find out more about the program.  As a reader of this blog, you can save 20% off the standard rate when you register to join us and mention code D15223BLG.  

This is co-posted on LinkedIn


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Thursday, January 22, 2015

Right Drug, Right Time

Today's guest post comes from Genome Editing Applications sponsor Horizon Discovery.

Right Drug, Right Time
Author: Hannah Murfet, Vice-Chair CQI Next Generation Network

“Right first time” is a concept familiar to the quality profession, but it is taking on a whole new meaning since the emergence of personalised medicine. Personalised medicine aims to eradicate an often random and reactive approach to patient care by employing techniques such as genetic testing to ensure treatment is more likely to be effective and safe in a specific patient population.

Traditional approaches to drug treatment generally take a population-wide trial and error approach. That is to say that the drugs used for treatment are known to be generally effective, but their specific effect on an individual patient is often unknown. Drugs are typically tested on large patient populations in order to test for safety and efficacy. As the patient population is seldom uniform, the efficacy is reduced as the drug works well for some and not others. Personalised medicine has the opportunity to change this by allowing us to use the right drug at the right time – a prime example of root cause analysis.

The successes of personalised medicine are staking up, and include genetic testing for breast cancer, chronic myeloid leukaemia and colorectal cancer. One very well-known example is screening for BRCA1 and BRCA2 gene mutations to predict whether a patient needs preventative breast cancer treatment. Other examples include targeted therapy based on chromosome alterations in patients with chronic myeloid leukaemia and KRAS gene mutations used as biomarkers to predict the success of specific drug treatments for colorectal cancer. Seemingly, the possibilities for personalised medicine are endless.

While the upfront costs of personalised medicine are initially higher, they will be reduced in the long term as the cost of quality is reduced by removing the expense of ineffective and repeated treatments. It’s a tremendously exciting time for the pharmaceutical and medical device fields, but I also feel those in the quality profession can take something from this. The application of the right drug, at the right time, has parallels with the work of the quality profession. We aim to use the right tool, the right approach and the right improvement at the right time. So let’s take a ‘personalised medicine’ approach to business improvement. I’m sure the results will be just as powerful for improving quality.

Genome Editing Applications will take place will take place March 18-19, 2015 in Boston.  Find out more about the program.  As a reader of this blog, you can save 20% off the standard rate when you register to join us and mention code D15223BLG.  

This post is co-posted with the CQI Blog.


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Wednesday, January 21, 2015

ADC emerge as leading cancer treatment option

Antibody Drug Conjugates (ADCs) are an ideal way to target cancer cells while missing the cancer cells.  Two ADCs have jumped to the top of the treamtent list -brentuximab vedotin which treats lymphomia and ado-trastuzumab emtansine which treats breast cancer.

As their success grows, and more therapies using this technology are being developed, finding safe manufacturing methods for ADCs has posed to be the next big challenge. According to BioProcess International, there are four key factors of an ADC that must be in tact to produce a working ADC: the antigen, the targeting antibody, the linker, and the payload. In order to create more drugs at a larger scale, researchers are focusing development methods that reduce heterogeneity and ways to create site-specific conjugations.  Not only that, but finding these two things are some of the next key challenges for scientists:
  • -How to increase clinical impact for tumors that display heterogeneous expression of a target antigen
  • -How to improve outcomes for those ADCs that show promising early efficacy before tumors develop resistance.

What do you see as the next big breakthrough in antibody drug conjugate therapy?

This year at BDP Week, we'll have an afternoon dedicated to New Modalities and Next Generation ADCs – Challenges in Development and Production with presentations from companies including MedImmune, Sutro Biopharma Inc. and CytomX Therapeutics, Inc.  For more information on 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 XB15155BLOGJP to save 20% off the standard rate.


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Thursday, January 15, 2015

Continuous Processing The Good, The Bad and the Unexpected

Continuous processing is easier said, than done. It promises to make biomanufacturing more viable,
but will it delver?

At BDP Week, industry experts will share the good, the bad and the unexpected to help you reduce manufacturing costs, improve product quality and increase flexibility while reducing risks.

Continuous Processing Programming Highlights Include:
  • • Media Development Strategies for Platform and Late Phase Cell Culture Process
    • -Wenge Wang, Ph.D., Senior Principal Scientist, Bioprocess R&D, Pfizer
  • • Upstream Disposable Technology Supports the Implementation of Continuous Processing
    • -Shaun P. Eckerle, Principal Scientist, Cell Culture Development, Patheon Biologics
  • • Downstream Processing and New Technology for Continuous Chromatography
    • -Maria Ekblom, Senior Project Manager, Chromatography Systems, GE Healthcare
  • • Ultra Scale-Down Characterization Of Bioprocessing Materials for the Early Prediction of the Impacts of Industrial Scale Continuous Centrifugation on the Recovery and Purification of New Therapeutic Candidates
    • -Alex Chatel, Ph.D., Bioprocess Enterprise Fellow, Biochemical Engineering, University College London, United Kingdom
  • • Continuous Chromatography: The Good, the Bad, and the Unexpected
    • -Oliver Kaltenbrunner, Ph.D., Scientific Director, Chemical Process R&D, Amgen
  • • How to Optimize the Perfusion Rate in High Cell Density Perfusion of Chinese Hamster Ovary Cells Culture in Stirred Tank
    • -Veronique Chotteau, Ph.D., Prinicpal Investigator, Researcher, Cell Technology Group, School of Biotechnology, KTH, Royal Institute of Technology, Sweden
  • • Integrated and Fully Continuous Processing of Recombinant Therapeutic Proteins – From Cell Culture Media to Purified Drug Substance
    • -Veena Warikoo, Ph.D., Director, Purification Development, Genzyme
  • • Exploring Options for Achieving Diafiltration in a Continuous Process
    • -Alex Brinkmann, Engineer III, Biogen Idec
  • • ASAP (Automated Seamless Antibodies Purification): Toward a Fully-Disposable Process
    • -Benoit Mothes, Pharm D, Senior DSP Scientist, Sanofi, France
  • • Continuous Downstream Processing: Where Does It Fit?
    • -Moderator: Marc Bisschops, Ph.D., Scientific Director, Tarpon Biosystems
  • • Single-Use Chromatography Platform For Monoclonal Antibody Purification
    • -Renaud Jacquemart, Ph.D., Senior Scientist, Process Sciences, Natrix Separations
  • • Exploring Options for Achieving Diafiltration in a Continuous Process
    • -Alex Brinkmann, Engineer III, Biogen Idec
  • • Processes of the Future : Single Use, Closed and Continuous for Faster, Cheaper and Safer Manufacturing
    • -S├ębastien Ribault, Ph.D., Director Biotechnology/Life Science, Head of BioDevelopment Center, EMD Millipore

Plus, do you have new research to share with your industry colleagues? We’re searching for the newest, innovative findings to be presented by our attendees in poster displays at BDP Week. Submit your abstract today.

Now is the time to make plans not attend BDP Week for end-to-end bioprocessing solutions that will help you guarantee reproducible results and manufacturing success.  Register to join us March 30-April 2, 2015 in Huntington Beach, California.  As a reader of this blog, when you register to join us and mention code XB15155BLOGJP, you save 20% off standard rates.


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Tuesday, January 13, 2015

Single-Use Applications 2015

Join us for Single-Use Applications 2015 to learn how to optimize time-to-market, overcome complex drug product and manufacturing challenges, and ensure successful implementation and integrity of single-use adoption and deployment.

The event features 10 Case Studies and 2 Interactive Q&A Sessions on Today's Most Pressing Single-Use Challenges including:
  • • Ensuring Successful Single-Use Implementation and Integrity: Case Studies from: Amgen and Merck & Co.
  • • Single-Use Implementation and Transfer: Case Studies from: Genentech, Merck & Co. and Public Health England
  • • BPSA Roundtable: Interactive Q&A: The Road Ahead: Issues Impacting the Accelerated Adoption of Single-Use Technologies
  • • Single-Use Considerations for Cell Culture Applications: Case Studies from: NIH and Shire
  • • Single-Use Applications in Upstream Processing: Case Studies from: Shire HGT and KPI Biopharma
  • • Production and Regulatory Considerations for Single-Use: Case Study from: Protein Sciences Corp.
  • • Overcoming Challenges Associate with Particles and Extractables & Leachables: Interactive Q&A: Extractables & Leachables
>> View the Single-Use Agenda
>> View the Full BDP Week Agenda
>> View the Agenda-a-Glance (PDF)

Single-Use Applications 2015 will take place from March 30-31, 2015 in Huntington Beach, CA.  It Flexible Facilities & Biopharmacuetical Development & Production Week is co-located with taking place on March 30 - April 2, 2015.


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Thursday, January 8, 2015

The Top 10 Ways to Improve Speed, Quality, Efficiency and Compliance | Register this week and present your poster for free!

IBC's Biopharmaceutical Development & Production Week (BDP Week) will take place March 30 - April 2, 2015 at the Hyatt Regency Resort & Spa in Huntington Beach, CA. This year, we’re pleased to announce that it is co-located with Single-Use Applications & Flexible Facilities.

The event offers the most in-depth coverage of bioprocessing topics on the market with 10 Focused Conference Tracks to help you improve speed, quality, efficiency and compliance.  What are our top 10 topics?



1. ADC Development & Production: Explore revolutionary approaches to development, scale up, formulate, and produce next generation ADCs that are more potent, stable, and have dramatically increased therapeutic windows.



2. Analytical Methods & Technologies: Maximize process and product quality, from comparability and characterization strategies to QbD concepts to HCP quantitation and control to aggregation and subvisible particle analysis.



3. Upstream Processing: Increase cell culture productivity, product quality, and efficiency through innovative technologies, platforms, and approaches to streamline development and reduce time to large scale production.



4. Single-Use Applications: Optimize time-to-market, overcome complex drug product and manufacturing challenges, and ensure successful implementation and integrity of single-use adoption and deployment.



5. Downstream Processing: Optimize efficiencies during harvest and purification of high cell density processes, continuous platforms and emerging modalities through the integration of disruptive technologies and methodologies.



6. Flexible Facilities: Novel and adaptable production strategies and solutions for the multi-product-centric biomanufacturing landscape.



7. Manufacturing Efficiencies: Achieve and maintain operational excellence with solutions to the emerging challenges of increasingly diversified portfolios in a global marketplace.



8. Quality and Control: Develop and benchmark protocols with a focused look at the latest challenges, best practices, and future trends for process validation and viral safety.



9. Bispecific Antibody Development & Production: Utilize new molecule design approaches, development strategies, and platform production processes to overcome the unique challenges of this transformative class of therapeutics.



10. Manufacturing Quality & Control: Develop and benchmark quality assurance and quality control protocols to meet compliance expectations for environmental, contamination, and manufacturing controls.


Plus, at BDP Week you have access to meet with the leaders in the industry with more than 165 speakers, 80+ Case Studies, 85+ New Data Presentations, 70+ exhibitors, 80 scientific posters and 800 attendees. This is the only event that can provide you this much in-depth biopharmaceutical development and manufacturing coverage in just 4-days!

Free Poster Offer 
Present your poster for free! Register for BDP Week with code BDP15LIA16 and present your poster for free! This code also includes the 20% discount off the standard rate you receive as a reader of this blog. This offer expires Friday, January 9.

Have any questions? Email Jennifer Pereira.

You can also enter to win a free pass to this year's event.  Head to Twitter and retweet this tweet to enter!


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