• 4 Unique Demands Of A Gene Therapy Supply Chain
    4 Unique Demands Of A Gene Therapy Supply Chain

    In Part 2 of my conversation with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, he addresses the unique demands of a gene therapy supply chain.

  • Digital Platforms For Patient Engagement In Clinical Trials — Trends & Outlook
    Digital Platforms For Patient Engagement In Clinical Trials — Trends & Outlook

    Pharmaceutical companies have three targeted users: physicians, pharmacists, and patients. Various digital platforms are used to create a good customer experience for educating these end users. The core focus is on patients driving patient care, education, and adherence.

  • Inside PDA’s Technical Report No. 81
    Inside PDA’s Technical Report No. 81

    Parenteral Drug Association (PDA) recently released Technical Report No. 81, “Cell-Based Therapy Control Strategy,” written by PDA’s Cell and Gene Therapy Task Force. Michael Blackton, Global Head of Quality at adaptimmune and PDA’s Co-Chair of the Cell and Gene Therapy Interest Group co-authored the report. Blackton’s also a member of the Biopharmaceutical Advisory Board and International Board of Directors. Here, he breaks down Technical Report No. 81 and explains the report’s biggest takeaways.

Considerations For Effective International Collaborations
Considerations For Effective International Collaborations

While there are many key aspects of a successful international collaboration across many time zones and great distance, in our experience — a collaboration between organizations based in the U.S. and Japan — there are a few essential elements.

  • Spark’s Bartock On Streamlining The Supply Chain
    Spark’s Bartock On Streamlining The Supply Chain

    I caught up with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, to talk through some of the gene therapy sector’s heftiest supply chain challenges. In Part 1 of a 2 part series, hear what advice he has for pharma, bio executives, and CMOs to successfully manage and even streamline the supply chain.

  • Overcoming The Unique Filling Challenges Of Autologous Therapies
    Overcoming The Unique Filling Challenges Of Autologous Therapies

    Autologous (patient-derived) therapies represent unique challenges to processing. One-patient, single-batch is a radical change from the scale our industry is accustomed to. At this level the filling is more analogous to limited Phase 1 clinical trial materials or even preclinical applications. The logistics and required compliance of manufacturing these personalized therapies is drastically different and presents a combinatorial explosive problem. One aspect of this process where traditional methods fall apart is filling of these therapies.

  • CAR T-Cell Therapies In The EU5: What Can We Expect From Payers?
    CAR T-Cell Therapies In The EU5: What Can We Expect From Payers?

    The difficulty of balancing paying for innovation with budgets has plagued Europe for years, and the emergence of cell and gene therapies has only exacerbated that conundrum. 

  • Sneak Peek: The Unique Challenges Of Cell And Gene Supply Chains
    Sneak Peek: The Unique Challenges Of Cell And Gene Supply Chains

    Two of the field’s most valuable luminaries representing both academia and industry — Dr. Bruce Levine and Simon Ellison, ISCT Commercialization Committee — discuss determining how CMOs can and should successfully manage cell and gene therapy supply chains.

  • Philadelphia: The Cell And Gene Therapy Sector’s New Normal
    Philadelphia: The Cell And Gene Therapy Sector’s New Normal

    Being a Greater Philadelphia Area resident affords me the opportunity to meet and collaborate with scientists, doctors, researchers, and other highly skilled professionals knee-deep in the cell and gene therapy sector. From the University of Pennsylvania to CHOP to Spark Therapeutics to Tmunity and many, many more, Philadelphia not only continues to shine as the geographical standout for innovation and continuous forward movement for cell and gene therapies but also as the smart economical choice for SMEs in the field.

  • Who Will Win The Regenerative Medicine For Diabetes Race?
    Who Will Win The Regenerative Medicine For Diabetes Race?

    Regenerative medicine could be a game-changing approach to meet the needs of insulin-dependent patients. Although the field is still in its infancy, the initial clinical data looks promising. This article takes a closer look at a few innovative companies that are racing to bring the first regenerative medicine for diabetes to the market.

  • Critical Considerations For Packaging And Delivery Of Gene Therapy Products
    Critical Considerations For Packaging And Delivery Of Gene Therapy Products

    The temperature-sensitivity of cell and gene therapies, their personalized nature, and their circular (“vein-to-vein”) supply chain make maintaining a seamless supply chain critical; however, it is not easily achieved.

  • Adaptimmune’s VP QA CMC Talks Quality Management
    Adaptimmune’s VP QA CMC Talks Quality Management

    One conversation with Michael Blackton, VP QA CMC at Adaptimmune, proves why he is known for being a passionate, results-oriented leader focused on QA, analytical development, and more. Validate for yourself as Blackton answers my questions about process development and quality management. And, he provides a fresh outlook on recruiting senior-level executives.  

  • Nontraditional Design Concepts To Accelerate CAR T Processing
    Nontraditional Design Concepts To Accelerate CAR T Processing

    The first round of CAR T therapies, now considered “legacy,” is largely being produced using biosafety cabinets and bench-top incubators with multiple open operational steps. The next generation of production processes will reduce or eliminate the open operations and automate cell-based manipulations.

  • Cardiac Stem Cell Controversy — 3 Important Lessons Learned
    Cardiac Stem Cell Controversy — 3 Important Lessons Learned

    Over the past 20 years, hope has grown around the use of stem cells to enhance regeneration following a heart attack, wherein stem cells would facilitate tissue repair and help restore heart muscle function. However, the legitimacy of cardiac stem cell research is now being called into question.

  • The Status of Regulatory Framework For Cell And Gene Therapies
    The Status of Regulatory Framework For Cell And Gene Therapies

    Jiwen Zhang, Executive Director, Regulatory Affairs at Tmunity Therapeutics Incorporated and Cell & Gene Editorial Advisory Board member explains the status of the cell and gene sector’s domestic and international regulatory framework. As President of the International Standards Coordinating Body (SCB), she also provides a synopsis of the most recent Stakeholder meeting.

  • What’s In A Name? Understanding Unmet Medical Need May Help Align Prioritization Strategies
    What’s In A Name? Understanding Unmet Medical Need May Help Align Prioritization Strategies

    Unmet medical need (UMN) is not a new concept, but it is an increasingly important one. Regulators and payers are nudging the industry to steer R&D investments toward areas with higher unmet need and less crowded pipelines.

  • Paying For CAR-T: Reimbursement Insights For Cell & Gene Therapy Space
    Paying For CAR-T: Reimbursement Insights For Cell & Gene Therapy Space

    While recent scientific breakthroughs for regenerative cell therapies are bringing great hope to patients and providers in desperate need of treatments and cures, how do you determine payment for something of enormously high value that has literally never existed before?

  • Beyond The IND: Regulatory And Clinical Planning For Cell And Gene Therapies
    Beyond The IND: Regulatory And Clinical Planning For Cell And Gene Therapies

    A quick and nimble approach is often sought after in the cell and gene therapy product space due to the likely one-time or limited patient dosing and/or rare disease conditions limiting the number of available patients, which reduces the number of clinical studies.

  • Establishing A Critical Supply Chain For Autologous Cell And Gene Therapies
    Establishing A Critical Supply Chain For Autologous Cell And Gene Therapies

    With the recent breakthroughs in cell and gene therapy, there is increased emphasis on the design and implementation of different supply chain models to support the movement of materials and drug product across the chain of care. Unlike more traditional supply chains, many of these therapies have unpredictable sources and manufacturing and infusion locations. The most extreme example is loosely referred to as the “vein-to-vein” supply chain — particularly for autologous therapies.

  • A Patient’s Journey To Join A CAR-T Cell Therapy Trial
    A Patient’s Journey To Join A CAR-T Cell Therapy Trial

    Nicole Gularte is currently battling cancer for the 8th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.

More From Cell & Gene

C&G EDITOR IN CHIEF, ERIN HARRIS

  • 4 Unique Demands Of A Gene Therapy Supply Chain
    4 Unique Demands Of A Gene Therapy Supply Chain

    In Part 2 of my conversation with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, he addresses the unique demands of a gene therapy supply chain.

  • Inside PDA’s Technical Report No. 81
    Inside PDA’s Technical Report No. 81

    Parenteral Drug Association (PDA) recently released Technical Report No. 81, “Cell-Based Therapy Control Strategy,” written by PDA’s Cell and Gene Therapy Task Force. Michael Blackton, Global Head of Quality at adaptimmune and PDA’s Co-Chair of the Cell and Gene Therapy Interest Group co-authored the report. Blackton’s also a member of the Biopharmaceutical Advisory Board and International Board of Directors. Here, he breaks down Technical Report No. 81 and explains the report’s biggest takeaways.

  • Spark’s Bartock On Streamlining The Supply Chain
    Spark’s Bartock On Streamlining The Supply Chain

    I caught up with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, to talk through some of the gene therapy sector’s heftiest supply chain challenges. In Part 1 of a 2 part series, hear what advice he has for pharma, bio executives, and CMOs to successfully manage and even streamline the supply chain.

  • Sneak Peek: The Unique Challenges Of Cell And Gene Supply Chains
    Sneak Peek: The Unique Challenges Of Cell And Gene Supply Chains

    Two of the field’s most valuable luminaries representing both academia and industry — Dr. Bruce Levine and Simon Ellison, ISCT Commercialization Committee — discuss determining how CMOs can and should successfully manage cell and gene therapy supply chains.

  • Philadelphia: The Cell And Gene Therapy Sector’s New Normal
    Philadelphia: The Cell And Gene Therapy Sector’s New Normal

    Being a Greater Philadelphia Area resident affords me the opportunity to meet and collaborate with scientists, doctors, researchers, and other highly skilled professionals knee-deep in the cell and gene therapy sector. From the University of Pennsylvania to CHOP to Spark Therapeutics to Tmunity and many, many more, Philadelphia not only continues to shine as the geographical standout for innovation and continuous forward movement for cell and gene therapies but also as the smart economical choice for SMEs in the field.

  • Adaptimmune’s VP QA CMC Talks Quality Management
    Adaptimmune’s VP QA CMC Talks Quality Management

    One conversation with Michael Blackton, VP QA CMC at Adaptimmune, proves why he is known for being a passionate, results-oriented leader focused on QA, analytical development, and more. Validate for yourself as Blackton answers my questions about process development and quality management. And, he provides a fresh outlook on recruiting senior-level executives.  

  • The Status of Regulatory Framework For Cell And Gene Therapies
    The Status of Regulatory Framework For Cell And Gene Therapies

    Jiwen Zhang, Executive Director, Regulatory Affairs at Tmunity Therapeutics Incorporated and Cell & Gene Editorial Advisory Board member explains the status of the cell and gene sector’s domestic and international regulatory framework. As President of the International Standards Coordinating Body (SCB), she also provides a synopsis of the most recent Stakeholder meeting.

  • ARM’s CEO Recaps 2018 Meeting On The Mesa
    ARM’s CEO Recaps 2018 Meeting On The Mesa

    Cell & Gene Meeting on the Mesa took place last month in La Jolla, CA, and it’s no surprise that ARM delivered meaningful data and addressed cutting-edge research to more than 1,200 attendees. I caught up with ARM’s CEO Janet Lambert after the Meeting to get her take on top highlights, the most informative panel discussions, as well as a look ahead to next year’s event. Here’s what she had to say.

  • Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals
    Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals

    In part two of my discussion with Miguel Forte, CEO at Zelluna Immunotherapy and ISCT’s Chief Commercialization Officer, he answers my questions about the Society’s near-term goals and objectives as well as his top areas of focus as CCO.

  • ISCT’s Miguel Forte On Academic-To-Industry Translation
    ISCT’s Miguel Forte On Academic-To-Industry Translation

    In part one of my discussion with Miguel Forte, CEO at Zelluna Immunotherapy and Chair of Commercialization Committee at ISCT, he answers my questions about academic-to-industry translation, the root cause of unproven therapies, how the industry is combatting them, and more.

CELL AND GENE INDUSTRY EVENTS

Patients As Partners March 11 - 12, 2019
Philadelphia, PA
Car-T Congress USA March 20 - 22, 2019
Boston, MA
ARM Meeting on the Mediterranean April 23 - 24, 2019
Barcelona
American Society of Gene & Cell Therapy (AGCST) April 29, 2019 - May 2, 2019
Washington, DC
DIA 2019 Global Annual Meeting June 23 - 27, 2019
San Diego, CA
More Industry Events

INDUSTRY INSIGHTS

  • Dos & Don’ts Of Bioprocess Facility Construction
    Dos & Don’ts Of Bioprocess Facility Construction

    Expansions and renovations to existing biological facilities, and construction of new facilities, provide a unique opportunity to rethink basic design strategies and use new technologies to build a better facility that will improve compliance. This article is the sixth in a six-part series on how single-use systems (SUS) are changing the modern biotechnology facility design and construction paradigm.

  • Cell Therapy: Process Design Considerations To Support Commercialization
    Cell Therapy: Process Design Considerations To Support Commercialization

    Cell therapies have the potential to revolutionize the biopharmaceutical world, but today’s processes, logistics, and delivery make for a challenging entry into the sector’s growth curve. As the industry evolves, we have to answer (at least) three important questions when bringing these exciting new therapies to market.

  • Designing The Cell Therapy Facility Of The Future
    Designing The Cell Therapy Facility Of The Future

    In 1931, Swiss doctor Paul Niehans injected a preparation of live cells from a parathyroid gland into a dying patient. The patient subsequently recovered, and Dr. Niehans had a eureka moment — that injections of living cells can have tremendous therapeutic value.

  • Biomarkers For The Brain
    Biomarkers For The Brain

    Frost & Sullivan recently invited academic and industry leaders in neuroscience research to participate in a unique thought leadership forum, our Virtual Think Tank series, titled Biomarkers for the Brain. This forum brought together leading minds involved in the study of biomarkers of central nervous system (CNS) diseases and disorders. The Virtual Think Tank stirred discussion about technological advancements in the field, current progress, future implications of the research and development occurring today, plus unmet needs in tools, key challenges that still remain, and expectations for future usage of biomarkers in CNS diseases.

  • Moving Forward With Immunotherapies For Cancer
    Moving Forward With Immunotherapies For Cancer

    Industry experts joined a panel at Frost & Sullivan's Virtual Think Tank recently to discuss the current state of immunotherapies, implications of research and development occurring today, key challenges, and future approaches to the targeted use of the immune system in cancer treatment. Read their thoughts on immunotherapies and the critical role for the immune system to play, especially in the advanced stages of cancer, since traditional standard-of-care therapies do often work fairly well.

More Industry Insights