Update on Japan's New Cell Therapy Regulations

By: Leah Kinthaert

At the Cell Therapy BioProcessing Pre-Conference Symposia for the BioProcess International Conference and Exposition, VP of Business Development for RepliCel and cell therapy industry thought leader Lee Buckler updated the audience on Japan's new cell therapy regulations. Buckler was a great person to give insight into these new regulations, because RepliCel is partnered with a Japanese company.

The regulatory environment for cell therapies changed greatly in 2012 when Shinzo Abe was elected prime minister. Last year Abe (who has since then been re-elected) set a plan in action to change the existing regulations and provide $1 billion in stem cell research funding over the next 10 years. This political and economic driven regulatory innovation has created a market push into Japan; Buckler described how his company re-prioritized Japan into their strategy due to this seachange.

Japanese companies are actually getting overt recommendations from their government to go into cell therapies; the government has incentivized companies to locate in Kobe, where the Kobe Biomedical Research Center is located. Buckler stated: "One can't underestimate the power of government to influence corporate decisions. CEOs feel the need to respond to government presence."

Buckler went on to give details about his company RepliCel's research in Japan. RepliCel has partnered with one of the world's top hair care and cosmetics companies, Shiseido. Shiseido has built their own manufacturing facility in Japan, making RepliCel one of just two companies with a cell manufacturing footprint in Japan.

Buckler ended his lecture by asking the audience to ponder this question: "Over the long term, will it (changes in Japanese regulations) have real lasting commercial implications?"

The agenda for the PreConference Symposia on Cell Therapy Bioprocessing on Monday, October 26 can be found here.

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State of the Cell Therapy Industry 2015: Approvals, Funding and the Future

By: Leah Kinthaert

At the Cell Therapy BioProcessing Pre-Conference Symposia for the BioProcess International Conference and Exposition, the packed room was treated to two "Where are we now in cell therapy" analyses which gave an excellent examination of the current state of the industry, and provided predictions and suggestions for a successful future.

Kirk Trisler, Principal at Dark Horse Consulting, opened the event with an extensive overview of the current landscape in cell therapies, going through cell therapies that have been approved and examining who the players are for cardiology, oncology, neurology, diabetes, and opthamology.

Tisler presented two helpful maps, one showing "Cell Therapy CMO Options in Europe" the other "Cell Therapy CMO Options in the US". The US map showed that the "only options for Phase III" in the US are WuXi, Lonza, PCT (one in CA, one in NJ), UC Davis, Temple, Stanford, City of Hope, and U of Iowa.

Tisler then went through a list of all cell therapies that have been approved. They are: Provenge by Dendrion; Lavir by Fibrocell; Holoclar by Chiesi; Chondrocelect by TiGenix; Carticel by Genzyme/Vericel; and Apligraf, Dermagraft and Ginutiut all by Organogenisis.

Chris Gemmiti, Business Development Lead, Wyss Institute - whose discussion was titled "State of the Industry - From Fund Raising to Partnering to Adoption and Commercial Success" - continued on the track of giving an overview for the current cell therapy space. He said that there has been over three billion dollars in federal funding for regenerative medicine over the last three years. Financing for cell therapy companies in the first half of this year alone is over seven billion dollars, with an additional eight billion in milestone payments.

Gemmiti then went on to talk about the companies who have been funded this year: including Semma, Voyager, Dimension, Unum, Regenxbio, and Audentes. "Look at Semma," he said "(they got) $44 billion in funding and they're not even at the IMD stage."

He continued with more helpful stats:

• There are 580 regenerative medicine companies worldwide
• There are 72 approved products

Gemmiti closed his presentation by cautioning the audience: "Market caps are great but they have to translate to revenue in the real world...Just having approvals and a large market cap doesn't mean commercial success."

The agenda for the PreConference Symposia on Cell Therapy Bioprocessing on Monday, October 26 can be found here.

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Innovations in Single-Use Technologies: Mario Philips of Pall

By: Leah Kinthaert

BioProcess International Magazine had a great interview with Mario Philips, President, Single-Use Technologies at Pall Life Sciences, earlier this year. Pall is the sponsor for the Cell Therapy Bioprocessing Pre-Conference Symposia at the BioProcess International Conference and Exhibition happening Monday, October 26 in Boston. What follows are some of the highlights of the conversation between Philips and BioProcess International Magazine’s Publisher Brian Caine.

In some ways, you could say Mario Philips’ career came full circle when his company entered the cell therapy market. A chemical engineer by trade with some time spent in the biopharma industry, the 2000s found him working in the semiconductor industry at a company called ATMI. Philips said: “The semiconductor industry was facing the challenge of molecular and particle contamination when cleaning the stainless steel containers used for transportation…So we developed a bag-in-a-bottle and a bag-in-a-container system.” A friend gave him the idea to translate what they were doing to the life sciences. So he set out to become the leader in SUT (single use technology) by asking customers what was not yet addressed by the current technology. The connection with cell therapy comes from the fact that Belgium, where he lives, has a comparatively large biotech industry. Not many equipment manufacturers saw cell therapy as a promising business model - but his company at the time did.

Philips brings that thought leadership and innovation to Pall, where his goal is to bring cost-effective technologies that help “companies get more out of their R&D related technologies and help them either scale out (for autologous therapies) or scale up (for allogeneic therapies).”

Caine asked Philips to describe exactly what making those technologies cost-effective would entail: – “For autologous therapies…the goals are to close up the process and develop industrial scale automation…For allogeneic therapies…the goals are to close up the process, reduce the risk, and bring in some controls.”

Caine then asked why Pall was entering the cell therapy market when so many other companies are waiting for a return and “a bit more clarity in the market”.– Philips’ responded: “At Pall we believe it is a risk, but it is a more calculated risk, and we want to be first-to-market with this platform.”

Caine: Where do you see yourself as a leader in the cell therapy market and what products does Pall have to support that? Philips answers: “I think we have a strong position in what I call the expansion step of the cells. We have more product development in what I call the volume reduction step. Then we want to leverage expertise from our current business – one such area is process development....The second area that we want to leverage is our internal biopharma automation and process group. We build large chromatography skids. We set up complete single-use suites with the bioreactor in the middle and the mixers around it. We are building the expertise to connect these great products into a real platform solution. So I would say our focus is currently on industrial manufacturing, helping customers out of a relative crunch that they are in.”

The agenda for the PreConference Symposia on Cell Therapy Bioprocessing on Monday, October 26 can be found here. You can find the complete interview with Philips here.

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"Think Beyond a Single Batch" says Nick Timmins' of CCRM

By: Leah Kinthaert

On October 26, Nick Timmins, Director of Product and Process Development at CCRM returns as a speaker at the BioProcess International conference in Boston. For a preview, here's a brief recap of the highlights from his half hour discussion at the recent Cell Therapy Bioprocessing & Commercialization event held just a few weeks ago in Alexandria, Virginia. The title of Timmins' lecture was: "Solutions to Process and Technology Bottlenecks."

Process Bottlenecks and Technology Gaps – Timmins started his talk off with the pronouncement that he should call his lecture "Process Bottlenecks and Technology Gaps".  He explained: "We tend to throw the term bottleneck out there without really using it in the right context, at least not the context that is formally taught, an engineering-type context." If you have a technology gap "you need to create a new solution. If you have a bottleneck you can always add more capacity, up to certain limits."

Bottlenecks can cause an accumulation of inventory – The beginning of Timmins' talk focused on defining bottlenecks in cell therapy. He describes one of the major pitfalls of a bottleneck: "If you spend a whole lot of effort running at maximum capacity, you're going to get an accumulation of inventory. Inventory costs money. In our case (cell therapy) inventory is often labile. Cells sitting in a hold step...is doing bad things to your cells."

Think beyond a single batch –  Timmins cautioned his audience "to think beyond a single batch" because "as we move beyond commercialization and industrialization, plant and equipment utilization becomes very important" and a "major component of costs."

About Nick Timmins – Nick received his PhD in Chemical Engineering from The University of Queensland, Australia, specializing in novel methods and applications for 3D multicellular spheroids. Nick is the Director of Product and Process development at CCRM. Nick’s previous work experience includes Team Leader, Cell Therapy for the Australian Institute for Bioengineering and Nanotechnology (AIBN) at The University of Queensland. In this role Nick was responsible for the bioprocess development for clinical scale manufacture of blood products from haematopoietic stem cells and process scale-up and automation of mesenchymal stem cell manufacture. Prior to this, Nick completed his Postdoctoral Fellowship at the Tissue Engineering Laboratory, ICFS at the University Hospital Basel in Switzerland where he designed and developed a bioreactor for 3D cell culture and tissue engineering, a joint industry/university project.

Timmins will be speaking at the PreConference Symposia on Cell Therapy Bioprocessing on Monday, October 26. More information can be found here.

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Unique Challenges of Cell Therapy Bioprocessing

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Lee Buckler, Cell Therapy Group. Lee discusses the state of the industry and unique challenges that this event is helping tackle. Below is a brief excerpt from the podcast, follow the links below to access the complete podcast and transcript.

Listen to the podcast | Read the transcript

What are some of the unique challenges of cell therapy bioprocessing that this event is helping the industry to tackle?

Well, I think that one is just the cross flow of expertise that I mentioned before. So, one of the reasons why I think this Event has been so successful is because it is so closely associated with the bioprocessing journal, as well, in which we’ve really been trying to raise the level of publications related to cell therapy bioprocessing to try and encourage that cross flow of expertise and exchange of information between those in traditional biologics bioprocessing and those who are still cutting their teeth in cell therapy bioprocessing. So, the lack of that cross flow has been one of the challenges that we face. Bringing people in who have experience with larger-scale systems. Cell therapy is still being produced in relatively small scales. Even when we think it’s big scale, it’s still relatively small scale. So, bringing people with large-scale experience and thinking ahead about how these cell products are going to be manufactured on a large scale if we get great clinical efficacy is a tremendous asset.

Other than that the unique challenges differ significantly depending on whether you’re talking about autologous vs. allogeneic. One of the challenges with autologous is that it doesn’t really scale-up very well because a batch is a lot. So, there is a lot of cost of goods embedded in the human processing and in the testing. So, innovation with autologous cell therapies is around closing systems up. Potentially they don’t have to be inside playroom environments and/or in bringing lower cost batch testing solutions to cell therapy products.

When you’re looking at allogeneic, of course, depending on whether you’re looking at an adherent or non-adherent cell population, you’re really looking at how can you take the cell expansion process from key flasks or roller bottles or vats into tanks where we can do scale-up at a much larger scale and hopefully, potentially, reduce the media consumption because media consumption is the largest – as has been pointed out in past sessions of this conference – media consumption is the largest cost driver.

Some of the really important data that has been presented here and then also published in Bioprocessing International is some of these metrics around what is the exact cost of the processing technology that we are using today vs. what we are going to have to get to in the future.


Be sure to join us at Cell Therapy Bioprocessing & Commercialization in Alexandria, VA - September 30th to October 2nd, 2015. Register before 9/4 and save $300 with the code XB15188BLOG.

Register here | Download the agenda

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BioProcess International 2014 Symposiums Highlights

In addition to all the programming the BioProcess International conference offers you, IBC has even more programming choices for you by making the five pre-conference symposia available as either an add-on to your 3 or 4 day conference registration, as a stand-alone learning experience, or you can combine with an exhibit hall & keynote pass.

Isn't It Great to Have Choices?  Here are your symposium options for this year:

1. Antibody-Drug Conjugates - Developing ADC Technologies to Increase Therapeutic Windows: Understand how to apply novel linkers, conjugation methods and payloads to develop, scale up and manufacture next generation ADCs.
2. Innovation through Integrated Process Development Sponsored by: 3M: : Learn how Innovation through Integrated Process Development provides step change solutions to bioprocesses. Delivering purity to process fluidity leading to process efficiencies and better process economics.
3. Cell Therapy Bioprocessing: Discover how to successfully develop and manufacture cell therapies and achieve commercial success through technical and process innovations.
4. Optimize Processes and Improve Raw Materials Continuity and Transparency: Improve your supply chain with the latest techniques to trace your raw materials, better collaborate with suppliers, and troubleshoot the performance of your processes.
5. Knowledge Management across the Product and Process Lifecycle: Learn how to apply data from your process development and manufacturing operations on an ongoing basis to your next project/run to improve efficiency, quality and control.

Plus, register for the knowledge management symposia and be entered to win a free copy PAT Applied Biopharmaceutical Process Development Manufacturing courtesy of CRC Press.

See the symposium agendas here.

BioProcess International Conference and Exhibition will take place October 20-23 in Boston.  As a reader of this blog, you can register to join us with priority code BPI14BLOG and save 20% off the standard registration rate.  Have any questions about the symposiums or want to get involved?  Reach out to Jennifer Pereira.

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Could Gene Therapy Replace the Need for a Heart transplant?

Could gene therapy help avoid a heart transplant?  We’ll soon be one step closer to knowing the answer thanks to a groundbreaking trial in the UK.  Lee Adams, 37, is the first of an eventual 24 patients who will take part in a trial to see if Mydicar, a treatment from U.S. biotech firm Celladon, can help overcome advanced heart failure. 

The treatment is designed to deliver a spike in SERCA2a protein in the heart muscle.  The SERCA2a protein is responsible for making heart muscle contract and low levels have been known to make the heart pump weakly.  The protein will be delivered to the heart via harmless virus.  Researchers plan to take a samples after an initial six month period to measure the presence of the gene.  For those that undergo a subsequent transplant, the researchers will be able to examine the actual heart as well.

Of the 24 patients the study plans to involve, 16 will receive the actual treatment while the other eight are given placebos.  Like Adams, these patients have advanced heart failure and rely on Left Ventricle Assist Devices (LVAD) to keep them alive while they await a transplant. 

The trial, led by Imperial College London and funded by British Heart Foundation as well as Celladon, claims to be the first in the world to investigate the use of gene therapy to correct heart failure. 

Says Professor Sian Harding, who helped develop the treatment, “It's important to remember that the therapy is not correcting a gene defect. We are working much more downstream, which means that no matter what the cause of the heart failure, the therapy should be equally beneficial for patients whether their heart problems stem from genes, lifestyle or the environment or a mixture of all of these."

Adams has a reserved optimism towards the study.  "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients. But I'm not building up my hopes too much because, for all I know, I might have had the placebo.”

Here’s to hoping it is the “miracle cure”.

What else is new in the field of cell therapy?  Join us for the Cell Therapy Bioprocessing conference, September 15-16, Boston, MA.  Download the agenda to see what’s on tap.

SAVE 20%* off the standard rate as a reader of this blog. Register here and use code XB14188BLOG.

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BioProcess International Podcast Series: Michael Keenan and Barry Walsh, Conference Producers

BioProcess International Magazine is releasing a series of podcasts over the summer to prepare for the BPI Conference and Exhibition taking place this October in Boston.  To kick things off, we're going to start by highlighting the interview with conference producers Michael Keenan and Barry Walsh.

In their interview, they discuss the new additions to the BioProcess International Conference Conference and Exhibition 2014 including cell therapy commercialization, raw materials, BPI Theater, early stage molecules, BPOG, the standardization of the single use industry and more. Other new topics that Michael and Barry are excited about includethe new focus on cell therapies and antibody drug conjugates, quality risk management strategy, and knowledge management.

They also share details on the new networking opportunities at this year's event including a visit to Pall Lifesciences, the Poster Hall and opportunities to meet the top bioprocessing companies in our Exhibit Hall.

Download the podcast here.

Would you like to join us this October 20-23 in Boston at the BioProcess International Conference and Exhibition?  As a reader of this blog, when you register to join us and mention code BPI14BLOG, you can save 20% off the standard rate.

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Researchers Regenerate Cornea, Successfully Grow Tissue from Stem Cells

A team of Boston researchers has identified a way to leverage limbal stem cells in order to regenerate human corneal tissue—One of the first examples of developing tissue from an adult-derived human stem cell.  The research, a collaboration between Massachusetts Eye and Ear, Boston Children’s Hospital, the VA Boston Healthcare System and Brigham Women’s Hospital, provides hope for those with eye-damaging injuries or diseases. 

The loss of limbal stem cells, which help maintain and restore corneal tissue, is one of the leading causes of blindness. 

It’s long been known that limbal stem cells have powerful regenerative potential, but the issue has been locating these stem cells.  The breakthrough came when Markus Frank, M.D. and Natasha Frank, M.D., co-senior investigators on the study, discovered the existence of ABCB5 molecules in limbal stem cells as well as their role in the maintenance and survival of the cell.  Scientists were then able to leverage the ABCB5 molecule as a marker for these cells and more easily identify them.  

The Frank team then put this theory to the test on two groups of mice--A group with fully functioning limbal cells and those without.  Using the presence of the ABCB5 molecule, researchers were able to locate and then extract these cells from donor tissue and transplant them into the corneal tissue of one of the groups.  They found that this group of mice was able to re-generate fully functioning corneas.  

The control group was divided in two with half the mice being given limbal cells that were ABCB5 negative and half receiving no limbal cells.  Both of these groups failed to repair the damaged tissue.  This helped to confirm ABCB5 molecule status as being responsible for the regenerative properties of these stem cells.  

“ABCB5 allows limbal cells to survive, protecting them from apoptosis [programmed cell death],” said Markus Frank.  “The mouse model allowed us for the first time to understand the role of ABCB5 in normal development, and should be very important to the field in general,” added Natash Frank. 

What’s next for the team?  Said Frank in a Fox News interview, “For the first step, we’re really working towards an autologous graft in patients who are blind in one eye.  And then the second step, we’d really work towards using donor derived cells to transplant in a similar manner that may require immune suppression – but it may not.” 

What else is new in the field of cell therapy?  Join us for the Cell Therapy Bioprocessing conference, September 15-16, Boston, MA.  Download the agenda to see what’s on tap.

SAVE 20%* off the standard rate as a reader of this blog. Register here and use code XB14188BLOG.

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Heart Attack Vaccination Could Be on the Way

This post was contributed by @MikeMadarasz

Heart attacks are the leading natural killer worldwide and claim approximately one million lives every year.  A staggering figure, researchers with the Harvard Stem Cell Institute and University of Pennsylvania claim to have discovered a treatment that could potentially reduce the risk of heart attack by up to 90%. 

Scientists at the Harvard Stem Cell Institute and University of Pennsylvania have collaborated on a “genome editing” approach that has dramatically reduced cholesterol levels in mice with a single injection.  Kiran Musunuru MD, PhD, leader of the HSCI team working on this project, called the success of the first iteration of the experiment “pretty remarkable”.

The research was focused on altering a gene known as PCSK9 which is found in the liver and recognized as being a regulator of cholesterol.  In 2003, a group of researchers in France discovered that a rare mutation in this gene was positively correlated with high cholesterol and early heart attacks. 

More recently, a research group in Texas has discovered that a separate mutation in this gene also has the opposite effect.  The “good” mutation is present in only 3% of the population, but those carrying this specific gene have bad cholesterol levels ranging from 15-28% lower.  Consequently, their risk of heart attack is 47-88% lower. 

This lead Musunuru and his team to deduce that if they could replicate the effect, they’d be able to protect people from heart attack.   

The research reached a turning point in 2013 with an improvement to a technology called Cas9.  Said Musunuru, “Cas9 is a protein that will create a break in DNA, and the CRISPR is an RNA component that will bind to a matching sequence and directs the Cas9 to that sequence in the DNA in which you are interested. This creates a break where you want it. The cell can then repair itself, though often with errors, which is useful if you want to disrupt a gene to create a ‘knockout’ of the gene.”

“What we were thinking was that with this genome editing technology we can do something we couldn’t do before—make permanent changes in the genome at the level of the DNA; we can actually go to the source. So the question was whether we can use genome editing to make normal people like people born with the ‘good’ mutations.” Based on the results with mice, the answer is a resounding “yes”.

The group saw a 35-40% reduction in cholesterol levels in these mice which they say could translate into about reduction in heart attacks as high as 90% in humans.  Says Musunuru, “It could be a one-time treatment, a permanent alteration. If you used this in a population, you could reduce the occurrence of heart attack by 30 percent, 50 percent, 90 percent.”

Get the full article from HSCI here.  

Get more on the latest in the field of cell therapy at this year’s Cell Therapy Bioprocessing conference.  Join us September 15-16 in Arlington, VA.  Save $100 as a reader of this blog when you register to join us and mention code XB14189BLOG.

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BioProcess International 2014 takes the Industry’s Largest Event to New Heights

BPI 2014 to feature the new “BPI Theater” showcasing the industry’s hottest new products and innovation, interactive idea exchange sessions and access to partnering360® to create and build relationships

IBC Life Sciences, division of Informa, today announced that the 11th annual BioProcess International (BPI) conference & exhibition will be held from October 20-23 at the Hynes Convention Center in Boston, MA. BPI is the largest annual event where all segments involved in the development and manufacture of biologics gather to network, collaborate and learn about the most advanced bio-processing methods. This year’s BPI event promises to be the largest and most dynamic edition yet with more than 1500 delegates from around the world, five keynotes presentations, 160 speakers, 80+ posters and 150+ exhibitors.

“At GE Healthcare we are looking for an understanding of bioprocessing from start to finish, from cells to formulated drug substance. Going to BPI we see all of these aspects represented in the program and meet all those people working across that value chain,” said Guenter Jagschies, Ph.D. Senior Director, Strategic Customer Relations, GE Healthcare Life Sciences, Sweden. “BPI is an ideal event where our ideas meet those of our customers and peers.”

The conference program covers the full BioProcess spectrum including upstream processing, downstream processing, formulation and delivery, manufacturing strategy, analytical and quality and a new early stage company session on moving quickly from IND to the clinic.

In addition, BPI features four one-day pre-conference symposiums and four two-day intensive training courses on ADCs, cell therapy bioprocessing, knowledge management, process development, quality risk management, CMC analytical, comparability and stability studies, technology transfer and introduction to biopharmaceutical manufacturing. Download the agenda to see the full program.

This year’s BPI exhibit hall will feature the BPI Theater, designed to showcase the industry’s latest products and innovations. The theater will host live product demonstrations, panel discussions, rebroadcast keynote presentations and host live interviews with leading thought-leaders.

Featured in the BPI Theater will be companies who are in the forefront enabling the development of new biological products with new product launches, including Pall Life Sciences, Meissner Filtration and Sartorius Stedim Biotech.

Another powerful feature of this year’s event is the addition of partnering360®, the largest online network of life science dealmakers. Delegates will be granted exclusive access to a BPI specific partnering360® networking tool that will enable them to identify and schedule meetings with potential partners, and foster deeper engagement before, during and following the event.

Significant partnering opportunities exist for biopharmaceutical firms developing new and improved biologics including mAbs, vaccines, ADCs, recombinant proteins, bispecifics, enzymes, biosimilars and cell/gene therapy.

About IBC Life Sciences
IBC Life Sciences, a division of Informa, is your connection to the life sciences industry. Through conferences, webinars, podcasts and its suite of integrated marketing solutions, IBC Life Sciences delivers expert knowledge, valuable connections and customized offerings so customers can meet their goals. www.IBCLifeSciences.com 

IBC Life Sciences hosts the annual BioProcess International™ (BPI) Conference and Exhibition. The BioProcess International Conference & Exhibition is where the biologics value chain meets to do business. Each year BPI attracts over 1500 biologics industry leaders for four days of power networking and learning. No other industry event delivers the high-level connections and insight needed to accelerate your business. Use this preeminent industry platform to showcase your company, products and thought leadership. To get involved, visit www.IBCLifeSciences.com/BPI

Contact: Dawn O’Connor; Senior Marketing Manager; IBC Life Sciences

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See What's "Long Overdue" in the Field of Cell Therapy

In cell therapy, the Target Product Profile (TPP) is the preferred document used to record the aspirational attributes of a therapeutic.  Anthony Davies, Ph.D., President of Dark Horse Consulting, is a leading expert on TTPs and will be presenting on the specifics of authoring a TPP at this year’s Cell Therapy and Bioprocessing Conference.  He was able to shed some light for us on the current state of TTPs in the field:  

Are Target Product Profiles being increasingly adopted as development and as a mode of regulatory communications?

Anthony: Yes, they are and this is also long overdue. The TPP was introduced by the FDA quite some time ago as a way of capturing your expiration or profile of your product. It is, at last, being adopted more widely by industry. This is much appreciated by the regulatory agencies. It is a very good tool for two purposes.

First of all, from the first interaction the product ever has with a regulatory agency, this enabled the agency to frame the complete trajectory of product development all the way through to commercialization and gives them a tremendous perspective on what they are trying to do in developing these drugs. It’s repeated provision for the agency as the drug moves out of development product and keeps everything on track and keeps everybody realistic about where the drug is meant to be at each stage of development.

Interestingly, it’s also being adopted by biotech companies and pharmaceutical companies as an internal method of tracking the progression of the drug development. The use in both the external, regulatory view of the drug and the internal corporate view of the drug development is a really nice harmonizing tool. I’m very happy at the Conference to be giving a presentation on the nitty gritty of how to fill out a TTP for cell therapy. I hope it will be very useful for the attendees.

Anthony’s full interview can be found in this year’s brochure.  

You can hear more from Anthony and other leaders in the space at this year’s Cell Therapy Bioprocessing Conference.  Join us September 15-16 in Arlington, VA.  Save $100 as a reader of this blog when you register to join us and mention code XB14189BLOG.

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Changes Ahead in Cell Therapy

The field of cell therapy has traditionally resided more within the academic sphere.  However, over the last two to three years, we’ve seen a shift where big pharma has become much more involved.  This change has left many speculating about the implications on the industry going forward.  Marty Giedlin, VP of Development with Sangamo BioSciences, will be discussing some of these implications at the Cell Therapy and Bioprocessing conference.  He recently shared a few thoughts with us on this shift in cell therapy going forward:

Overall, how do you see cell therapy changing within the next five years?

Marty: Well, I think it’s going to go to more solid tumor targets and more epithelial tumors because that’s the hardest nut to crack. I think that’s where people want to go – the breast cancers, the colon cancers and their related metastases because that provides a big challenge in how you get --- I mean, you could maybe find T-cells or TILs within those tumors, but how do you get T-cells into those tumors because of just the physiological make-up of solid tumors, their ability to regulate the immune response and other factors that sort of limit adoptive cell therapy effects? I think we have some of the antibodies out there like anti-CTLA-4 and PD-1 and others that can sort of overcome some of the immunosuppressive activities of tumors. So, I think it’s going to be more of a matrix of immunotherapies that’s going to have to attack solid tumors. Besides the cells, there is cytokine support or antibody support. So, I think it’s going to be more academic and big pharma coming together. How can we put these individual therapeutics together to exact a clinical response?

I think the other thing – particularly with respect to cell therapies – is more automation. People tend to think of this as a culture flask business sort of thing. But, I think with the innovations by Miltenyi and their Prodigy platform and other companies coming in with better plastics and bags and just cell handling. I think it’s going to be much more get-it-out-of-the-lab and more of a --- I don’t want to say industrial, but more of a rapid hands-off sort of way of producing these cells. The goal is like what you want to do, depending on your source. You want to basically hand the bag of the raw cells from either the apherisis product or a bone marrow, tap a button and come back a couple of hours later and get the bag of your – in our case – gene modified selected cells to then route to the patient. So, I think those types of things are going to make it much more accessible to a lot of people, as well.

The last thing is, what more can we actually do to characterize the product, particularly with adoptive cell therapy? More people are going for a population approach to taking cells, modifying them and giving them back. But some sub-populations of cells really have activities that you want to give back to the patient to get the best results. So, I think more interest is going into respect to being able to phenotype these cells, do proteome analysis, look at RNA expression of cell populations and so forth. I think that’s really going to make things a little easier as far as targeting the cell population that’s going through the most difference going forth. That could be easier for us because if it’s a smaller population of cells, it has expansion capability – whatever – then its few cells that we have to process and it makes things easier on our end, as well.

Download the brochure for the Cell Therapy and Bioprocessing conference to check out Marty's full interview.

Get the latest from Dr. Barron and other industry experts at this year’s Cell Therapy Bioprocessing conference, September 15-16, Arlington, VA.  Now, SAVE 20% off the standard rate*.  Register here and use code XB14188BLOG.

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Cell Therapy: The Field’s Greatest Challenges Today

The last two decades have brought on a tremendous amount of innovation in living cell-based products.  That innovation has come with a new set of challenges in the field as well.  Jeff Karp, Associate Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School, will be speaking at this year’s Cell Therapy Bioprocessing conference.  We were able to pick his brain on what he feels some of these challenges in the field are today:

What are some of the greatest challenges in cell therapy today?

Jeff: I think that we’re getting to a point in time where we can obtain almost any cell type in unlimited quantities with a few exceptions. This is using reprogramming, programming, different differentiation, protocol that have recently been worked out. So, I really think we are getting to that point where we can obtain nearly any cell type that can then be delivered to patients for treatment.

While I think we’re there, I think one of the greatest challenges that remains is that once we transplant cells, we lose control over the cells. So, when we’re working with cells in a Petri dish, for example, we can pattern the media, we can put cells on all different types of textured substrates, we can control the media and the environment exquisitely. But, when we transplant cells, they are entirely at the mercy of the biological. So, if they end up in different tissues in the body, they are going to behave completely differently. So, we lose control of those cells following transplantation.

So, I think one of the greatest challenges is how can we now take the cells that we worked so hard to derive in vitro and then transplant those cells into patients and exhibit control so that the cells get to the right location and can perform their function when they get there?

Download our brochure to check out the rest of Jeff’s interview.

Get the latest from Jeff and other industry experts at this year’s Cell Therapy Bioprocessing conference, September 15-16, Arlington, VA.  Now, SAVE 20% off the standard rate.  Register here and use code XB14188BLOG.

See also- Cell Therapy Manufacturing: The Rapid Pace of Advancement

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Stem Cells Loaded with Herpes Being Used to Kill Brain Tumors

This post was contributed by @MikeMadarasz of the Institute of International Research

In a breakthrough discovery, researchers at the Harvard Stem Cell Institute have found that stem cells loaded in the herpes virus can effectively kill cancerous cells in brain tumors.  The study showed a marked improvement in the survival rate of mice with Glioblastoma Multiforme, one of the most common forms of cancer, when treated with these virus-loaded stem cells. 

Similar approaches using cancer-killing viruses have been utilized before in phase 1 and 2 clinical trials on brain tumors, but have experienced minimal success.  The biggest hurdle?  Keeping the herpes virus at the tumor site long enough before leaving the brain through cerebrospinal fluid.  To combat this, the Harvard team looked to mesenchymal stem cells, a type of stem cell that according to Harvard, is popular in drug delivery vehicles for the minimal immune responses it triggers.  The virus was loaded into these stem cells and injected into Glioblastoma tumors developed in mice.

Using multiple imaging markers, the team was able to watch the virus make its way through first layer of brain tumor cells and ultimately into all of the cells. 

Said Khalid Shah, MS, PhD, lead author of the study, “We know that 70-75 percent of glioblastoma patients undergo surgery for tumor debulking, and we have previously shown that MSCs encapsulated in biocompatible gels can be used as therapeutic agents in a mouse model that mimics this debulking.”  He added, “So, we loaded MSCs with oncolytic herpes virus and encapsulated these cells in biocompatible gels and applied the gels directly onto the adjacent tissue after debulking. We then compared the efficacy of virus-loaded, encapsulated MSCs versus direct injection of the virus into the cavity of the debulked tumors.”

Shah’s team was able to observe that the gel kept the stem cells alive longer and allowed them kill any cells not removed during the debulking.  This ultimately resulted in a higher survival rate for the mice. 

You can find the Harvard report here.

We’ll have more on the latest in cell therapy at the Cell Therapy Bioprocessing conference, September 15-16, Arlington, VA.  Now, SAVE 20%*. Register here and use discount code XB14188BLOG.

You can download the agenda here.

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Cell Therapy Manufacturing: The Rapid Pace of Advancement

According to Jon Rowley, CEO of RoosterBio and Conference Chair of IBC's Cell Therapy Bioprocessing conference, this coming decade will see the incorporation of living cells into platforms such as engineered tissues, bio-robotics and implantable devices as well as others not yet imagined.  Rowley, a leading expert in this field, will be discussing the integration of biologics into a series of products at this year’s Cell Therapy Bioprocessing conference, September 15-16.  Jon has more to offer on the subject in the meantime and was recently able to weigh in on some of these innovations:

What are some of the most important cell therapy manufacturing innovations that you’ve seen over the last few years?

There have been many important advancements that have been highlighted and presented right here at the Cell Therapy Bioprocessing conference over the last few years. We are in a very interesting time in the field of cell therapy where, over the last 10-15 years, we’ve developed some very interesting product innovations that have shown a very good signal in the clinic.

Now, our field has kind of transitioned into an era of manufacturing process innovation. We are seeing a very rapid pace of advancements in manufacturing technologies. I think the advancements fit into two different classes. The scale-up of the cell culture and then the downstream processing, which are both equally important. I think that hands down the conversion to scalable bioreactor expansion methods for allogeneic therapies will be the most impactful on the field as these technologies allow for companies to both scale their bioprocess to commercially relevant lot sizes, as well as help to reduce overall cost of goods.

Also, the field has learned from the traditional protein biologic field and is attempting to proactively address downstream processing bottlenecks before they occur. We’ve seen advancements in continuous centrifugation, like the Ksep technology that will enable the volume reduction, washing of extremely large volumes of primary cells. This is critical as the field moves to these large-scale bioreactor processes.

Download the brochure to check out the rest of Jon Rowley’s interview.

Get the latest from Jon and other industry experts at this year’s Cell Therapy Bioprocessing conference, September 15-16, Arlington, VA.  Now, SAVE 20% off the standard rate.  Register here and use code XB14188BLOG.

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12 New Things to look forward to at BioProcess International 2014

IBC Life Sciences is thrilled to be bringing the 11th Annual BioProcess International Conference & Exhibition back to Boston for 2014. As the #1 event in the bioprocessing industry, we are continually working to evolve the event with the needs of the industry and make sure that you have highly enjoyable, productive and rewarding experience.

The event will take place October 20-23, 2014 at the Hynes Convention Center in Boston, MA.  It is the #1 BioProcessing Event in the Industry with over 1500 attendees, 200 speakers and over 150+ exhibitors.

What are the 12 new things that you can look forward to for this event?

1. 1. More emphasis on enhanced antibodies, novel and next-generation molecules
   2.  Early stage company session: Moving quickly to IND
   3.  Added sessions on  cell therapy bioprocessing, continuous bioprocessing,  and  Process validation and CPV
   4.  BPOG working groups
   5.  More quality and regulatory discussions
   6.  Improved partnering system to schedule meetings
   7. BioProcess International Theater
   8. Point-Counterpoint Discussion: Challenges and Considerations around Relaxing Environmental Controls in Biopharmaceutical Facilities
   9. Pre-Conference Symposium: Knowledge Management across the Process and Process Lifecycle
   10. Town Hall Forum: Standardization of Single-Use Systems: What is the Next Step for the Industry? (Featuring updates from ASME BPE, ASTM, BPOG, BPSA, PDA and USP)
   11. Site tour to Pall Life Sciences in Westborough, MA
   12. Lounges to kick back, relax and partake in open dialogue in the exhibit hall

What are you waiting for?  Register to join us in Boston by by May 23 and Save $600 off the Standard Rate!  Be sure to mention priority code  BP14BLOG.  Have any questions or want to get involved?  Reach out to Jennifer Pereira.

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Managing Contamination Risk While Maintaining Quality in Cell-Therapy Manufacturing

Today, we feature an excerpt from BioProcess International. Dominic Rachel Kilian's article Managing Contamination Risk
While Maintaining Quality in Cell-Therapy Manufacturing was featured in the March 2013 Supplement of BioProcess International.

Managing Contamination Risk While Maintaining Quality in Cell-Therapy Manufacturing

With an increasing number of cell therapies becoming available for patient use, the need for controlled and consistent manufacturing and delivery of cell products is increasingly important. A closed cell culture process not only offers control and consistency, but may also relieve labor demands. Single-use components within a closed process also can reduce contamination risk.

Closed systems with single-use platforms may reduce the risk of biological contamination and cross-contamination that could inadvertently be introduced into cell-culture processes. Such contaminants use culture nutrients to produce unwanted proteins and limit the growth of (or destroy) a cell culture. Slow-growing adventitious agents can be subtle and may become apparent only when unwanted proteins are detected. Detection of contamination will result in unusable product, and depending on the circumstances, entire lots may have to be discarded. This can be incredibly costly in both materials and time. 

Manual cell culture processes can be labor intensive and include numerous open events. They have limited process control, which can lead to difficulties in achieving high reproducibility. Some systems are closed during the stages in which cells are grown. It is equally important, however, to close cell seeding and harvested processes. That is more difficult to achieve, so not as many equipment vendors currently address those additional needs.

Read the rest of the article here. 

If you'd like to find out more about single use technologies and cell therapy bioprocessing, Richard Eglen, Ph.D., Vice President and General Manager, Corning Incorporated, will be on hand at the Cell Therapy Bioprocessing Event to present the technology workshop Optimization of Single-Use Technologies in Cell Therapy.  For more information on this session and the rest of the program, download the agenda.  Would you like to join us in Bathesda, Maryland this coming October 21-22? As a reader of this blog, when you register to join us and mention promo code BX13188JP20, you're eligible to save 20% off the standard rate! 

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Single-Use Technologies in Cell Therapy

Today, we feature an excerpt from BioProcess International. Dominic Clarke's article Single-Use Technologies in Cell Therapy: Lessons Learned was featured in the March 2013 Supplement of BioProcess International.

 Single-use (also referred to as disposable) technologies and systems are those intended for one-time use. They generally consist of plastic polymers that include but are not limited to polyethylene (PE), ethylene vinyl acetate (EVA), polyvinyl chloride (PVC), polypropylene (PP), and polycarbonate (PC). Disposables originated in the early 1950s by Fenwal Laboratories (now Fenwal Blood Technologies, a Fresenius Kabi Company) in the form of plastic blood bags. Since that time, SUTs have grown into an extensive range of

products: from standard Petri dishes and culture flasks routinely used in cell culture laboratories to large-scale cell stacks and rocker bags (Figure 1) and now even immense 3,000-L culture bags for commercial-scale production levels (1). Although early uses were dedicated to regulated (medical device) blood-transfusion applications, the latest advances can be attributed to wide-scale use and adoption into biopharmaceutical production (2). Such increased use and adoption by the industry has been a driving force for improving overall quality standards for SUTs.

Using disposable technology for cell therapy development, manufacturing, and clinical application is standard practice. Cell therapy experts are very familiar with SUTs. But the hybrid of disciplines and applications making up the industry has in many ways created some confusion when it comes to the next generation of considerably different cell therapies currently in clinical development.

Consider, for instance, a group working on an exciting new therapy originating from a hospital/clinical setting. Such personnel are well versed in working within a clinical environment mainly using regulated (medical device) SUTs. Now consider a completely different therapy being developed by a research laboratory at a university level, which in turn eventually spins off into a new startup cell therapy company. Those personnel are adept at working in a nonregulated environment with standard R&D labware. As such, the
knowledge and perceived requirements for SUTs for clinical scale-up and product manufacturing is likely to be noticeably different. In both cases, small-scale development using established single-use technologies and systems introduced initially will be acceptable. But it’s likely that neither will have appropriate technology and necessary documentation to support manufacturing requirements in later
clinical phases.

With more cell therapy products entering phase 3 clinical trials and commercialization, it is imperative that the biopharmaceutical industry recognize not only the opportunities presented with SUTs, but more important, the knowledge gaps and potential pitfalls. It can be devastating to reach a point when a product is ready to enter a phase 3 clinical trial (or, worse, commercialization) and have to make a change to a single-use cell growth/expansion bag or storage container because another technology was discontinued or altered. Given the vast availability of alternative (similar) SUTs, making a change doesn’t seem too daunting a task until you notice,
for example, that your cells don’t behave the same way with the new film, or the bags don’t meet the same storage requirements. That situation could significantly delay timelines or even destroy potential product.

Fortunately, manufacturers already have learned from encounters with such issues. Some have incorporated single-use platforms for producing new commercialized drugs. Along with this, the SUT industry suppliers and service providers of the BioProcess Systems Alliance (BPSA, www.BPSA.org) help establish guidance and best practices for implementing single-use in biomanufacturing processes (2). The cell therapy industry can benefit from using current resources and developing a better understanding of the impacts of disposable systems on manufacturing.

Read the rest of the article here. 

If you'd like to find out more about single use technologies and cell therapy bioprocessing, Richard Eglen, Ph.D., Vice President and General Manager, Corning Incorporated, will be on hand at the Cell Therapy Bioprocessing Event to present the technology workshop Optimization of Single-Use Technologies in Cell Therapy.  For more information on this session and the rest of the program, download the agenda.  Would you like to join us in Bathesda, Maryland this coming October 21-22? As a reader of this blog, when you register to join us and mention promo code BX13188JP20, you're eligible to save 20% off the standard rate! 

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What are some of the sessions that highlight this year's Cell Therapy Bioprocessing Event?

The Cell Therapy Bioprocsesing event taking place October 21-22, 2013 in Bethesda, Maryland.  For more information on the event, download the agenda.  If you'd like to join us, as a reader of this blog, you're can save 20% off the standard rate when you register to join us and mention code B13188JP20!

Leading up to the Cell Therapy BioProcessing event, we've interviewed several of the key stakeholders who helped shape this year's event.  We'll start by looking at the recent interview we conduced with Event Chair Lee Buckler, the Founder and Managing Director of the Cell Therapy Group.

Today, Buckler answers the question:

What are some of the sessions you are particularly looking forward to in this year’s program? And who are you looking forward to meeting while there?

Lee: Well, I always look forward to meeting my friends, of course, and I’ve been in this sector for a lot of years. So, it’s always good to see people I know and hang out with them. But, inevitably, you meet new, fresh faces as well and I think there’s any of a number of them this year that I’m looking forward to meeting and hearing from at the different companies. There have been a couple of important additions this year, which is going to result in some interesting sessions and be the infusion of new people who I look forward to meeting. One of those is around the production of iPS cells, which is not an area that I have a tremendous amount of experience or exposure to. So, I’m always keen to hear about what is happening on that front with the potential clinical production of human-induced pluripotent stem cells. Then, also, there is a fair amount of focus this year on the production of immunotherapies. I’m anxious to hear what Novartis is doing, for instance, with a program that they picked up at the University of Pennsylvania. They bought the old Dendreon Facility in New Jersey and are looking to replicate and do some process improvements on that process. I’m keen to hear about that. 

And then the tissue in bioengineering track, as well, is a new addition to this year’s Conference and is always interesting to hear about the intersection between cells and scaffolds and constructs because I think that’s where a lot of innovation is going to take place where we can combine cells either with genetic manipulation or with biologically inerts or non-biological material.

You can download Lee's full podcast here. Next week, Lee will look at what is unique about this conference.

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