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Tuesday, April 15, 2014

An Interview with BioCision. Why Innovating for Reproducibility is Good Business.



BioCision is one of those companies that easily catches your attention (they use lots of bright colors) but might take awhile to be a regular part of your consciousness.  After all, laboratory and bioprocessing tools are not the sexiest product category.  Because of that, I tried to ignore BioCision for some time as a company that looked interesting but was too 'laboratory-focused' for my tastes.

What eventually forced me to take a closer look at this company was their clear commitment to two things:  (1) making standardization and simplicity major driving forces for their innovation, and (2) online audience engagement not just for marketing but as an incredibly useful tool in driving the innovation feedback loop.

Also, I'll admit I was completely wrong about them only offering research laboratory tools. It turns out their applications may indeed offer even more value to those involved in clinical production.

They are a private company quietly but busily innovating around some of the most basic biologic handling issues in the lab or manufacturing suite -- often solving problems you didn't know you had until you are presented with the solution.  They are an interesting mix of conservative yet innovative - bold but basic.

Long-story short:  this company has really grown on me as the 'read deal' such that theirs is the first corporate advisory board I recently agreed to join (there's my disclosure).  

A company is, of course, about a lot of people doing a lot of important things (big and small) that collectively make it a success but there's no doubt that Rolf Ehrhardt, the company's President and CEO, has stamped the company with his imprint.  Because it is so clear to me that the BioCision culture and modus operandi is very much an imprint of Rolf's personality (as is often the case with companies a la Apple, Miltenyi, etc), I had to sit him down for an interview.  That is what follows and I hope you find it interesting if not informative.


CTB: Tell us about BioCision and the sector(s) you serve? 

The reproducibility of scientific studies has become a major issue in life science research from drug discovery and development through to clinical trials as researchers around the world continue to use different protocols for processes associated with sample preparation, cryopreservation and cold chain management. The scientific community is now becoming more aware of factors that affect sample integrity and experimental variability. By standardizing handling, storage, and transportation protocols we can vastly improve the quality and reproducibility of preclinical and clinical data, helping to accelerate the transition from lab research to drug development and market launch.  

That’s where BioCision comes into play. In 2007 a group of scientists, biomedical engineers, and drug discovery experts founded BioCision to develop laboratory tools with standardization in mind in order to set a new bar in temperature control for biomedical samples. Today our products are used by researchers around the world in pharmaceuticals, biotechnology, academics and healthcare. We are positioned for continued growth in multiple sectors including cell therapy, regenerative medicine, biobanking, bioprocessing and cold chain management. 

CTB: What would you say is BioCision’s underlying philosophy or business approach?

Our goal is to enable researchers to achieve the highest attainable levels of pre-analytical sample reproducibility and consistency – experiment to experiment, lab to lab and site to site. In order to accomplish this, we are very focused on our role as a leading advocate for the standardization of biomedical samples for basic, pre-clinical and clinical research.

In addition to our novel products, our business strategy is based on our ability to engage and mobilize the scientific community around this effort to improve reproducibility and develop new sample handling standards. We just announced the creation of a new scientific advisory board that includes internationally recognized researchers who are uniquely qualified to help BioCision develop additional products and support solutions that are able to address standardization challenges for our customers. 

We look forward to working closely with the members of our advisory board, especially as we continue to expand our position as a global partner to support research and product development, in cell therapy in the years ahead. 

We have also been able to leverage social media platforms not only to share and support our message of preserving sample integrity but engaging our audience on their innovation needs. For example, we have worked hard to develop and maintain a blog called “Sampling Science®” to keep our readers informed about standardization efforts in cell therapy research and other fields of interest.  Most recently, we created a discussion group called “Scientists Against Sample Abuse™” - or “SASA™” - to continue this important conversation about improved sample handling with customers and fellow advocates.

In the end, these efforts have resulted in a growing community of laboratory researchers and clinicians who understand the significant impact that sample/drug handling and storage techniques can have on study results and even the efficacy of new biological therapies.

CTB: Your products seem to have a very strong visual design concept that’s reminiscent of Apple or Miltenyi.  How important is that to the company?

The design of our products is positioned as a significant value-added benefit for our customers. On the surface, the fluorescent colors we use for our products bring a spot of fun to the laboratory and help us stand out at exhibitions and medical conferences. But there’s much more behind our design strategy. 

The use of color-coding also helps laboratories work smarter and reduce the risk of errors. Most labs still use buckets of ice, refrigerators, water baths and mostly white, grey or glass containers for biomedical sample handling and storage. This makes it difficult to organize samples in an intuitive way, leading to delays or errors in processing that can affect sample quality and results. 

We try to design products based on how scientists actually interact with samples. We make it possible for researchers to use color to streamline operations and improve the quality and efficiency of lab procedures. The use of color makes it easier to store and organize similar samples. Labs can develop storage protocols based on color so any researcher is able to easily find a sample. We envision a future where products can be custom-designed and color-coded to meet the needs of major laboratory systems around the world. 

CTB: Has the company done much in the way of partnerships with larger companies?

Last year, we announced an agreement with the American Type Culture Collection (ATCC), the world's leading provider of authenticated biological material, that enables the development a custom line of products including the CoolCell LX cell cryopreservation device.  As a result, ATCC cell-freezing protocols recommend the use of CoolCell LX containers to maintain an optimal controlled and standardized freezing rate. Their protocols are widely used as the industry standard in global biomaterials research. 

This year we are working on a couple of exciting projects. The first is with TxCell, a French company that is developing cell-based immunotherapies for the treatment of severe chronic inflammatory diseases. Several of our products are being used to support temperature control and the processing and handling of cell therapies in their Phase IIb clinical trial investigating the use of an antigen-specific regulatory T (Ag-Treg) autologous cell-based immunotherapy for the treatment of patients with moderate to severe refractory Crohn’s disease. In this effort our CoolCell alcohol-free cell freezing container is being used to freeze Type 1 regulatory T (Tr1) cells in Aseptic Technology ampules. 

It’s an interesting case study, because in previous trials, TxCell used electronic programmable freezers to manage the cell freezing temperature. However, the Phase IIb trial will involve collection of cells from multiple sites, prompting  TxCell to choose the CoolCell containers for cell freezing, because they provide the same freezing outcomes as programmable freezers, while being much easier and much less expensive to deploy to multiple clinical sites due to their portability, lack of necessary maintenance and ease of use. TxCell is also using our CoolRack and CoolBox ice-free cooling systems in the cell handling stage to standardize the temperature of the cell preparation, media, and cryopreservatives to 4°C without the use of ice and reduce the risk of drug-product contamination. Because the efficacy of regulatory T cells can change without proper and reproducible handling methods, the standardization of pre-analytical drug sample handling, cryopreservation and storage are key to ensuring consistent trial results. 

TxCell will present additional details on the use of CoolCell containers for cell freezing during a poster session at the 20th Annual Meeting of the International Society of Cellular Therapy in Paris this April. 

A related paper has also been published in the March issue of BioProcess International titled “Effective Cryopreservation and Recovery of Human Regulatory T Cells.”

Earlier this year, we received a $4 million equity investment from Brooks Automation, a global provider of automated sample storage systems for compound management and biorepositories. The investment initiates a strategic partnership that will support the development of innovative sample handling solutions for cold chain management and the handling of drug products. 

In just the past few years, numerous independent publications have endorsed and recommended the use of BioCision products in their methods and protocols sections. We look forward to partnering with more companies in the years ahead by offering innovative solutions to temperature control and sample handling and storage needs. 

CTB: What product offerings do you have in the cell therapy space?

We are perhaps best known for our CoolCell cell freezing containers, which provide a reproducible  freezing rate of 1°C/minute when placed in a -80°C freezer and have become the new industry standard for cell cryopreservation in research and clinical applications, including cell therapy. The rate at which stem cells are frozen is crucial to the overall success of cryopreservation process and the freezing rate should be measurable in a way that does not rely on the freezer size or model and should also be equal across all samples in a freezer.  Our CoolCell SV series is specially designed to fit closed-system, injectable glass ampoules that are popular in the cell therapy space where aseptic vials are critical for minimizing contamination. 

A recent independent example of our product in use in the cell therapy space was published in the Journal of Autoimmunity. Researchers cited the use of a CoolCell freezing container for studying the role of T cells in myasthenia gravis. The CoolCell freezing container was used to gradually freeze cells isolated from the blood of both patients and healthy donors for long-term storage while sample collection was underway.

Our CoolRack® and CoolBox™ ice-free cooling systems are also commonly used to standardize the temperature of cells, cell media, and cryopreservatives prior to the cryopreservation stage. 

More recently, we have developed a product called CoolStation™ which is a mobile ultra-low temperature workstation.  The CoolStation systems provide an ultra-low temperature open-top work space through the use of dry ice or LN2 where a variety of operations such as cryogenic packaging, sample sorting or transport, or sample freezing can be performed.  For example, by placing a CoolStation next to a biosafety cabinet during cell preparations, cells could be placed into a CoolCell freezing container and the CoolCell could be directly placed in the CoolStation for immediate freezing, limiting the non-frozen exposure to DMSO or other potentially harmful cryoprotectants. 

CTB: Why have you decided to expand into the field of cell therapy?

Cell therapy is a rapidly expanding field that holds great promise for future clinical applications and early trials continue to yield positive results. But in the grand scheme of things this is still a very new field of research. Just a few years ago it was often assumed that small variations in cell temperature or cryopreservation would not dramatically affect the outcome of an experiment.  As cell therapy research has advanced we now have a much better understanding of how stem cells behave in different environments, and it’s clear that even slight changes in temperature management can have major implications for the efficacy of the final therapeutic product. Today, we regularly find and share reports on the importance of proper sample handling, particularly related to temperature control, to ensure the reproducibility of cell therapy research.

For example, following workshops organized by the U.S. National Institutes of Health (NIH), the journal Nature recently launched an initiative to more systematically ensure key methodologies are reported by researchers, including the creation of a checklist prompting authors to disclose certain technical and statistical information.

The movement to minimize irreproducibility in cell therapy research has led to growing interest in BioCision’s innovative solutions as researchers discuss different tools and protocols for handling and storing samples. 

CTB: How do you decide on new products to develop?

We remain focused on sample standardization in all areas of life science and clinical research and are continuously developing and improving tools that address common problems related to biomedical sample handling and storage. 

Currently we are working on three main areas of product development. First, we continue to advance our cell freezing and related workflow solutions, with a special focus on stem cells and primary cells for research. Second, we are developing new solutions for sample handling before and after long-term storage (i.e. Biobanking). And third, we are studying clinical and diagnostic applications where maintaining consistent temperatures - either cold or warm - is critical, for example in home-based assays and cold chain management for short transport. 

In these efforts we are always actively seeking partners and collaborators who have an application for our innovative technology. 

CTB: Are you working on any new technologies that the cell therapy sector should be excited about?

Yes.  We have a number of products related to the cell cryopreservation workflow in various stages of development, some of which will be released at the ISSCR annual meeting in Vancouver in June, and these products will directly relate to cell therapy and aid in further standardizing cell handling.  And, as mentioned above, we are working with Brooks Automation to develop new products that will also apply to the cell therapy workflows.  

CTB: Will you be at the upcoming ISCT conference?

Yes, we’ve been looking forward to this year’s conference for quite some time. Working closely with TXCell, we are excited to present an abstract during the poster session that will analyze the benefits of using our CoolCell freezing containers in the manufacturing of an autologous antigen-specific Treg (Ag-Treg) cell-based immunotherapy for the treatment of patients with severe refractory Crohn’s disease. Researchers investigated CoolCell freezing containers as an alternative to the classical programmable freezer and developed a new standardized method of cell therapy product cryopreservation. It’s a major first step towards the use of cell therapy in the clinic. 

We also plan to present an abstract in collaboration with researchers from Dr. Jeff Bluestone’s lab at the University of California, San Francisco who are investigating the cryopreservation of peripheral blood mononuclear cells (PBMCs) with the intention of isolating regulatory T lymphocytes (Tregs) for autologous cell-based immunotherapy treatment in patients with autoimmune diseases or transplantation recipients. Similar to the TxCell study, the UCSF team investigated CoolCell freezing containers as an alternative to programmable freezers to cryopreserve PBMCs. They concluded CoolCell containers can help overcome challenges associated with multiple clinical site collections in cell therapy research because our device is less expensive, portable, lacks necessary maintenance, and is easy to use. 

CTB:  Thanks for the taking this time with us.  We look forward to hearing more about what the company has in store for the cell therapy sector soon!

Oh ya.  And remember that bit about social media?  Check them out:

https://www.facebook.com/BioCisionLLC                                                                          https://twitter.com/biocision











                                        


                                            http://www.labtube.tv/labtubemeets.aspx?vid=159051

      




Tuesday, February 4, 2014

US vs Regenerative Sciences Comes to End


 

A court case we blogged a fair amount about even before it started (see the first post in 2008) appears to have finally come to an end in the DC Circuit Court's decision to uphold the ruling in favor of the FDA.

United States Court of Appeals 
FOR THE DISTRICT OF COLUMBIA CIRCUIT 

Decided February 4, 2014 

No. 12-5254 

UNITED STATES OF AMERICA, 
APPELLEE 

v. 

REGENERATIVE SCIENCES, LLC, A CORPORATION, ET AL., 
APPELLANTS 

Appeal from the United States District Court 
for the District of Columbia 
(No. 1:10-cv-01327) 

It has been an interesting ride.  Chris Centeno and his partners (and investors) have been the lightning rod for a lobby and debate around the right way to regulate the use of cells therapeutically - particularly one's one cells.  The arguments became more sophisticated as the case wore on and, as so often happens, the ground shifted considerably while the case was ongoing.  The case and Chris Centeno's unparalleled commitment to the cause has contributed significantly to a debate that is important to have and I suspect is not over simply because the case is done.

Dr. Centeno and I have had our differences but now is the time to recognize his contributions to the debate and, more importantly, to the field. We look forward to his future contributions to developing robust, cell-based treatments for patients.



Thursday, January 2, 2014

Regenerative Medicine / Cell Therapy at Biotech Showcase 2014


 

As many of you know, during the week of January 13, healthcare and life science executives meet investors in San Francisco for a flurry of conferences, meetings, parties, and general mayhem.  

The EBD Biotech Showcase is one of several events taking place that week and it the one of particular interest to us because it has the most concentrated focus on regenerative medicine and cell therapy companies.  The full list of presenting companies is here. For those of you on Twitter, follow #BTS14.

We've gone through and picked out the regenerative medicine and cell therapy content.

Sponsored by the Alliance for Regenerative Medicine, the 4th annual Regenerative Medicine Industry Briefing kicks off the meeting at 8 am on Monday morning (Jan 13th) .  This year it is in the big plenary room and it promises to be the best yet.  Invite your friends, investors, partners, and media contacts.

We spotted the following 29 companies scheduled to present at various times as shown on the schedule spanning Monday through Wednesday:

Adaptimmune
Advanced Cell Technology
AlloCure
Bone Therapeutics
Cell Medica
Cell2B
CTI Clinical Trial and Consulting Services
Cytori Therapeutics
DiscGenics
Fibralign
Harvard Apparatus Regenerative Technology
International Stem Cell
Kiadis Pharma
MaxCyte
MolMed
NeoStem
Neuralstem
Northwest Biotherapeutics
NovaRx
PharmaCell
Pocastem
Q Therapeutics
RepliCel Life Sciences
RhinoCyte
Sernova
TiGenix
TVAX Biomedical
TxCell
ViaCyte

Because it is a full schedule with 4 concurrent tracks, we made our own schedule of regenerative medicine and cell therapy presentations. In case it is useful, we provide it here for you to download and use.  


2014 EBD Biotech Showcase Regenerative Medicine and Cell Therapy Presentation Schedule (PDF)*
    This schedule is sponsored by






* We can't promise we didn't miss a company, screw up the schedule somehow, or that EBD might not change things between now and the event.  You should really check this against the EBD program and please... rely on this resource in your discretion








Monday, December 16, 2013

An interview with GlaxoSmithKline's Dr. Jan Thirkettle

 

An interview with:

Jan Thirkettle, BSc, PhD
Head, Advanced Therapy Delivery
GlaxoSmithKline Medicines Research Centre


In anticipation of Phacilitate's 2014 Cell and Gene Therapy Forum to be held at the end of January in Washington, DC, I invited the Phacilitate team to provide blog readers with an interview of one of their upcoming faculty. The result is the very interesting Q&A thread you see below about GSK's call and gene therapy program combined with some of his thoughts on the sector.  

Phacilitate: You head the Advanced Therapy Delivery group at GSK – please could you outline your specific goals and activities as an organisation for us? Which projects/technologies are you prioritising currently, and why?

Dr. Thirkettle: Our immediate priority is to deliver the Chemistry, Manufacturing and Controls (CMC) aspects of a collaboration we already have for an investigational ex vivo gene therapy currently in late stage development with TIGET, the San Raffaele Institute for Gene Therapy in Milan. 

Through this collaboration we believe that we have a responsibility to complete the process for development of gene therapy, get it to file and make it available to more patients. We are really excited by the clinical data which TIGET have generated on the current programs thus far, but this is also laying really important groundwork for how we will work on other products in that collaboration and giving us real insight into the issues which we need to tackle in order to be able to progress following opportunities in this collaboration and make these autologous therapies more scalable.

This feeds into our second priority, which is to develop more efficient manufacturing technologies that can allow us to reduce the cost of goods, reduce the complexity, and increase the scalability of ex vivo gene therapy products. This is important within our existing collaboration but also it is going to be critical to allow us to engage with other opportunities which might have larger patient numbers.

Thirdly, whilst the immediate focus of our group is on the CMC aspects (process development, vectorology, cell characterisation, QC), we are also very focussed on working across the GSK platform organisation to leverage the expertise we have from our pharma and vaccine background and which is relevant to this area. This engagement and strategic planning is also critical to make sure that we build expertise in all of the other really key functions; for instance, this is an area where really strong understanding of the regulatory and the quality aspects is absolutely critical.

Phacilitate: This January, you will be joining a panel of big pharma representatives tasked with answering the questions of whether 2014 is the year pharma get serious about Cell & Gene Therapy, and whether it has been a lack of innovation or a lack of proven products that has held them back in the past - what’s your initial response to these questions?

Dr Thirkettle: I’m not sure it’s fair to say that big pharma has yet to get serious, if you look at the investments and deals of the past 3 years. I certainly don’t see any lack of innovation out there, it is almost the opposite. We talk about cell and gene therapy as a modality, but it is a huge, broad field; as broad as ‘biopharmaceuticals’ which is a field with many approaches and technologies. 

Within the cell and gene therapy field we have a very large number of modalities, different cell types, different approaches, and a huge diversity of technologies. And they are all evolving at a really blistering pace. So one of the challenges for anybody who is outside of the groups who are really driving that core science is to know which is the right bit to grab hold of and it’s not the sort of area that you can just dabble in if you want to be really be effective and add value.

Rather than lack of innovation, then, the challenge is maybe more of knowing how to pick the winners at the right time and understand the potential utility of that technology/product at this early stage. I think the key thing is that it requires a number of elements to come together; you need the biology and the underpinning science to be strong; you need a therapy with good clinical data to really evidence that and discharge some of the risk; and then you need an understanding of how broadly applicable that technology might be - is there some sort of platform that sits behind it that spreads your risk? And finally, you need an understanding if whether you can actually make that product, can you scale it up?

Phacilitate: You are also kindly chairing a session which explores the long-standing but still critical challenges in developing cost effective and robust manufacturing models for cell & gene therapies at industrial scale. What progress do you see being made in this regard? To what extent will it be a case of these problems being solved relatively quickly and easily as big pharma come on board more and more, as some have suggested?

Dr Thirkettle: There are some massive challenges, but I suppose I would never underestimate the ability of really committed scientists, whether from academia or biotechs or big pharma, to solve those problems given enough focus and time.
I think the key is creating a focus because in a way it goes back to the previous point - this is a hugely broad field. When you stand back and look at this mass of really exciting science and opportunity, the challenge actually is to understand where to focus that investment in time and money. It becomes an awful lot easier to create that focus and energy to invest where you have got really compelling clinical data - you know you have got something that can potentially be a product. Equally if you can have confidence that a technology has multi product utility, your ability to invest is that much greater. If you can make a strong case on either of those points then you’re in a good position to create the focus needed to drive investment in the manufacturing and commericalisation aspects.

Phacilitate: Finally, what are you hoping to get out your participation in the meeting this January?

Dr Thirkettle: I’m really hoping first of all to meet lots of other people who are facing the same challenges and learning the same things. I think it is really exciting to see the journey over the past few year with increasing discussions around “How do we make these therapies? ” rather than “Will these therapies ever work?” This is a field which would potentially be greatly enabled if there players could find a way to share learnings as much as possible about what works and what doesn’t. There is a huge opportunity to work in a pre-competitive mode. It is in everybody’s interest because there are so many challenges, so many things to learn. I think this is a forum which makes it really easy to have those conversations, and start to build those relationships that, ultimately, should help everyone.

Friday, June 28, 2013

Cell Therapy Industry Infographic

 

Here is an infographic of the data in one of my recent presentations.  

Enjoy. Consume. Share. Critique.



Sunday, April 21, 2013

Commercialization of Regenerative Medicine: Learning from Spin-Outs

 


The meeting “Commercialization of Your Regenerative Medicine Research: Lessons from Spin Out Successes” was hosted by the Oxbridge Biotech Roundtable (OBR) (Oxford, UK) at the University of Oxford in February, 2013, and attracted a multi-stakeholder audience spanning academia and industry. 

The event featured case studies from Gregg Sando, CEO, Cell Medica (London, UK), John Sinden, CSO, Reneuron (Guilford, UK), and Paul Kemp, CEO and CSO, Intercytex (Manchester, UK). 

OBR is a student-led initiative with over 7000 members across eight different UK and US locations with a mission to foster a conversation about the healthcare and life sciences industry. 

Anna French and David A. Brindley, along with some of my assistance, captured and have now published the main themes of the meeting and the major questions facing the regenerative medicine industry and its rapidly emerging subsets of cellular and gene therapies. 

Notably, we discuss the compatibility of regenerative therapies to the existing healthcare infrastructure, biomanufacturing challenges (including scalability and comparability), and the amenability of regenerative therapies to existing reimbursement and investment models. Furthermore, we reiterate key words of advice from seasoned industry leaders intended to accelerate the translation path from lab bench to the marketplace.

To read the review see: Commercialization of Regenerative Medicine: Learning from Spin-Outs

Anna French, R. Lee Buckler, and David A. Brindley. Rejuvenation Research. April 2013, 16(2): 164-170. doi:10.1089/rej.2013.1423.

Wednesday, April 17, 2013

2013 Annual Regenerative Medicine Industry Report

 

The Alliance for Regenerative Medicine announced today the release of the 2013 annual regenerative medicine industry report.  Here is the announcement in the Wall Street Journal online.

I'm proud to have been a part of putting it together and hope people find it useful.  It is available for download on the ARM website here.  


In addition to the complete download, ARM will make many of the figures, charts,  tables and sections available for members to download and use in their own publications and presentations. Watch for these resources to be announced soon.