At the Forefront of Cell Therapy in Boston: Oren Levy of Karp Lab

Oren Levy from Karp Lab gave an excellent talk called “MSCs on Steriods” last month at Biotech Week Boston. Oren's research focus is "investigating the roles of signal transduction pathways in hMSC physiological processes, specifically, the involvement of the JAK/STAT cascade in hMSC proliferation and osteogenic differentiation". His research also focuses on "hMSC homing and engraftment to various sites in the body". Karp Lab is located in the Cambridge/Boston Biotech Hub and works closely with Brigham and Womens' Hospital, MIT, Harvard Medical School and Harvard-MIT Health Sciences and Technology.

Levy started his discussion with this unfortunate fact - so common with so many potential biopharma cures being researched right now – that “MSCs clinical endpoints have not been met and there’s not a single approved FDA product”. He then also shared the impressive stat that “MSCs are used in 600 clinical trials worldwide”; with so much research happening right now, the industry is hopeful that some breakthroughs are on the horizon.

What are MSCs and Why Are They on Steroids? 

The NIH gives a good definition of MSCs on their website: “Mesenchymal stem cells (MSCs) are adult stem cells which can be isolated from human and animal sources”. MSCs are being studied for bone, cartilage, heart and blood vessel repair, as well as inflammatory and autoimmune diseases. Why are MSCs on steroids? If you check out their website, Karp Lab has a great sense of humor (and it’s an understatement to say the Lab’s work is “creative”) which makes sense that the title of the talk was playful. But in all seriousness, MSCs have the potential to be very powerful; researchers such as Oren Levy and Karp Lab have a real sense of urgency about getting MSC therapies to work. So much so that they embarked on a very ambitious project: with the help of Sanofi, Levy screened over 10,000 small molecules to improve cell targeting.

Our Goal is to Improve Control Over Cell Fate 

Oren Levy mentioned two disease areas where his team was studying the use of MSCs: prostate cancer and multiple sclerosis. In fact, Levy mentioned that their multiple sclerosis study was about to be submitted for publication, so we have that to look forward to soon. For prostate cancer, the screening of the small molecules Levy’s team did with Sanofi comes into play. Levy discussed how they use “drug loaded MSCs to kill prostate cancer cells”. How they’re trying to make this happen is by “small molecule pretreatment to give MSCs the homing mechanisms they lack”. Essentially, they’re “using engineering strategies to improve MSCs targeting to tumor sites”. The combination of engineering and life sciences – bioengineering – is a major trademark of what Karp Lab does. Their website relates one of their mission statements: “Our lab firmly believes that innovation occurs at the interface of disciplines”. You can read an in-depth interview of Jeff Karp of Karp Lab produced by Biotech Week Boston and written by journalist Nick Paul Taylor here.

Don't forget to follow Biotech Week Boston on Twitter for news on innovation in biotech and medicine. Each year passionate scientists and innovators converge on Boston to share ground breaking data, research and ideas - don't miss our next event in September 2017!

Learn more about #BiotechWeekBoston

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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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Plenary Sessions Pave the Way for "Cure Not Treatment"

By: Brian Caine

Cell Therapy Bioprocessing & Commercialization got off to an exciting start as plenary speakers Marc Better PhD, Julie Alllickson PhD and Bruce Levine described the cell therapy landscape and their outlined progress.

More than 200 cell therapy professionals heard Dark Horse Consulting president and event chairperson Anthony Davies set the stage by reviewing the real progress and strides the cell therapy market has made while acknowledging the hard work that the industry is facing.

Marc Better, Phd, Vice President, Product Services at Kite Pharma kicked off the first of three presentations describing the market as "exciting and challenging". He outlined the general challenges faced by Kite Pharma and focused on the solutions it implemented to effectively and successfully engineer autologous T cell therapies.

Julie Allickson, PhD, Director, Regenerative Medicine Clinical Center, Wake Forest Institute for Regenerative Medicine said real commercialization would come as research moved to "cure, not treatment".

She provided attendees with Wake Forest’s approach to determine what process design, development, manufacturing, and technologies will best support their initiatives.

"Academic and industry must come together in order to be successful," she said to create "a manufacturing roadmap for TERM Technologies".

Allickson reinforced the need to create a consortium to develop infrastructure and resources to advance manufacturing and set a standards. She also noted that bio-printing is showing greta results and has a bright future.

Bruce Levine, PhD, Associate Professor in Cancer Gene Therapy, University of Pennsylvania detailed their approach to targeting tumors with CAR-modified T-Cells and reported on the positive short and long term results.

Join the conversation on Twitter by following #IBC_CTB15

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Exclusive Interview with Larry Couture, VP, Center for Applied Technology Development, City of Hope

Dr. Larry Couture, Vice President of Center for Applied Technology Development, City of Hope - a Keynote Speaker at this years Cell Therapy Bioprocessing & Commercialization Event, sat down to discuss the biggest challenges facing regenerative medicine developers, how pharma companies benefit from learning the academic model for translation, regulatory hurdles, and this years meeting. Below you will find a brief teaser from the interview, to access the full interview, follow the links below...

Podcast | Transcript

What are the biggest challenges facing regenerative medicine developers?
Well, the field is really beginning to take off. There are now four or five or six clinical trials and probably at least as many academic trials – some of those are academic – that are about to enter the clinic or will be entering the clinic in the next couple of years. 

One of the problems and challenges that are starting to emerge about where this is going to go and what we are going to have to face and some of the issues we are dealing with right now that I think are going to become big are cost of goods, the ability to scale-up some of the manufacturing processes that we are using purity to sell product. Not necessarily trying to achieve 100% per se, but trying to optimize purity and identify how pure these products should be...[Click here to read more]


Want to hear more from Dr. Couture? Join him in Alexandria, VA for Cell Therapy Bioprocessing & Commercialization, September 30 - October 2, 2015 - where he will have a keynote address titled "Development of a Well Characterized Regenerative Medicine Cell Product". Register now with the code XB15188BLOG to save $100 off the current rate.

See you in Alexandria!

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The 3 Biggest Challenges Facing Regenerative Medicine Developers

 
Dr. Julie Allickson, Director of the Regenerative Medicine Clinical Center, Wake Forest Institute for Regenerative Medicine and Wake Forest School of Medicine - also a keynote speaker at this years Cell Therapy Bioprocessing & Commercialization event - sat down with us for a podcast interview. During the interview, Dr. Allickson discusses the future challenges for regenerative medicine developers, how pharma companies that are developing regenerative medicine therapies can benefit from the academic model for translation, the regulatory hurdles that regenerative medicine developers face, the key differences between the industry in North America, Europe and Asia, as well as her talk at this years meeting. Below you will find a teaser from the interview...Interested in hearing more? Access the complete interview now...

Podcast | Transcript

Where do you see the biggest challenges facing regenerative medicine developers over the next five years?

I think there are several different challenges, but when I really try to add those up I feel that there is a lot that we need to develop yet to get a robust manufacturing process. I think there is a lot that goes into making sure that the product is safe and meeting all the requirements for the regulators. But also, as we are looking at scaling up, scaling out the products, I think that there are a lot of efforts that we still need to put into that.

The other piece would be looking at automation and really pushing the automation to decrease what I’m going to say is the “human factor”, which can bring error and, actually, it would be much more timely. So, I think that the field really needs to push for the automation to be able to get more of a robust manufacturing process.

We actually just published a paper in February in stem cells translational medicine with Josh Hunsberger as the primary author, but really looking at the roadmap for tissue engineering and regenerative medicine in the manufacturing space. I believe that that’s definitely one of the challenges, but I think, also, we have challenges in clinical trial design.

There is a lot of failure – I would say – in clinical trials as we are learning, but I think it is getting better. As we nail down the appropriate patient populations, we’re not so broad and incorporate control groups and also really being thoughtful as we are looking at the end points in clinical trials. It is very critical as we’re selecting what matches up with the potency of the product. I think that’s definitely key.

Then, I would say third would be regulatory considerations. As we move forward, we know it’s really a new paradigm – regenerative medicine – in healthcare and I think that gives us the opportunity to be able to voice our concerns, educate the regulators so that they really understand what we need.

So, I think it is definitely communication, educating the regulators and I think that the more opportunity that we have to facilitate those discussions, the better place that we’ll be in because there is such an opportunity for healthcare if we can accelerate the commercialization of some of these products.


Want to hear more from Dr. Allickson? Join her in Alexandria, VA for Cell Therapy Bioprocessing & Commercialization, September 30 - October 2, 2015 - where Dr. Allickson will have a keynote address titled "Academic Model for Translation of Regenerative Medicine Including Tissue-Engineered Products in the 21st Century: What are the Pathways? What are the Barriers?"

Download the agenda | Register for the event 

We hope to see you in a few weeks!

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Accelerate Your Cell, Gene and Cancer Immunotherapies to Market - Best Rate Ends Friday

Save $400 with the code XB15188BLOG by Friday, July 31 | Register here.

IBC's Cell Therapy Bioprocessing & Commercialization is the most focused conference for cost-effective and efficient cell, gene and immunotherapy approaches that accelerate cell-based product development and production towards commercial success >> Download the full agenda.

Key Topics Include: 
• Applying Genome Editing and Other Disruptive Technologies
• Developing Scalable and Transferrable Manufacturing Processes
• Biopreservation, Cold Chain Management and Patient Delivery
• Developing Successful Business Models
• Fostering Partnerships and Outsourcing Strategies for R&D and Manufacturing
• Pricing, Reimbursement, and Adoption
• Negotiating the Regulatory Pathways to Approval

_______________________________________________________________

Save $400 with the code IMMUNO15BL by Friday, July 31 | Register here.

IBC's Immuno-Oncology is the industry's leading resource for diving into the current issues surrounding efficacy and response rates with first generation immunotherapies to propel the next wave of cancer immunotherapies >> Download the full agenda.

Key Topics Include: 
• Strategies for Combination Therapeutics
• Advances with Checkpoint Inhibitors
• Agnostic Antibody Agents
• Advances in Cancer Vaccines
• Advancing the Next Wave of Cancer Immunotherapies

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Today's Challenges in Cell Therapy

 

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Marty Giedlin, VP of Development at Sangamo BioSciences, Inc. Marty discusses key industry challenges, the evolution of the cell therapy field and where it is headed in the next five years, advancements in technology and much more. Below is a brief excerpt from the podcast, follow the links below to access the complete podcast and transcript.

Listen to the podcast | Access the complete transcript

What are the challenges that you’re experiencing in your industry?

Well, I guess it really depends on what kind of cell therapy you’re talking about. We at Sangamo are processing both autologous T-cells and autologous stem cells. We have the most experience in our T-cell program where we knock out the CCR-5 receptor for HIV in HIV-positive patients. So, the challenges are trying to find that patient population that’s most amenable to this type of therapy, both in viral load, how many years they’ve been in retroviral therapy and that sort of thing. And also finding out which of the CD4 cells – which sub-population – is most important in controlling HIV. So, those are some of the challenges that are facing us there.

With our stem cell program, we have a partnership with CIRM (California Institute for Regenerative Medicine) in California with the City of Hope where we are looking at using CD34 stem cells either from peripheral blood or from bone marrow. What sub-population of those cells are really those that have the capacity for self-renewal over time? Those are the cells that we really want to target to do our gene modification technology.

Then, there are culture conditions to minimize replication, retain stemness and maximizing engraftment with respect to CD34s. And then we’re looking at ways of mobilizing peripheral blood CD34s and how does that affect stemness with respect to CD34s? And then also looking at different ways of bone marrow harvest. So, are there better ways of getting maximal yield to make sure we get enough cells to give back to the patient?

So, for us in the adoptive cell therapy, those are some of the challenges that we’re looking at over the next three to five years.

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How will cell therapy change in the next five years?

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Anthony Davies, President of Dark Horse Consulting. Anthony discusses key industry challenges he is seeing, the evolution of the field in the next five years, some exciting new initiatives he is working on, regulatory issues and much more. Below is a brief excerpt from the podcast, be sure to click on the links below to access the complete podcast.

Listen to the Podcast | Access the complete transcript

How do you see the field of cell therapy changing in the next five years? 

I believe strongly that the next five years will bring the first significant drug approval. This will be a transformative moment for the field. A lot of players who are sitting on the wings will move in and there will be a big acceleration. Who will benefit from that the most are the organizations and the entities which have spent the difficult, recent years preparing and positioning themselves the best. 

I think we would do well to look around at this point because it will – in a sense – be the calm before the storm. The companies which are working fervently now preparing themselves to be able to take their drugs all the way to commercialization are the ones who are going to step in to the fast lane when the first big approval comes. And as I said, I do think that the next five years is a very realistic timeframe for this to occur.

[The above was a brief excerpt from the podcast]

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