• 3 Business Challenges from the CDMO’s Perspective
    3 Business Challenges from the CDMO’s Perspective

    Partnering with a CDMO that marries innovation with technical, regulatory, and manufacturing experience can be cell therapy and gene therapy companies’ best opportunity for scalability and yet their biggest hurdle. From process development through commercial supply and all the steps in between, cell and gene therapy companies expect CDMOs to meet an understandably high bar.

  • News Round-Up: Gottlieb Steps Down, Roche-Spark Tx
    News Round-Up: Gottlieb Steps Down, Roche-Spark Tx

    What a week. As you know, The Washington Post broke the news of Scott Gottlieb’s resignation as FDA Commissioner — the news, while not entirely unexpected, shot disappointment and concern through the entire healthcare industry. Nancy Bradish Myers, JD, president of Catalyst Healthcare Consulting penned an article for Cell & Gene that explains the silver lining of Gottlieb’s exit.

  • Raising The Bar: The Silver Lining Of FDA Commissioner Gottlieb’s Exit
    Raising The Bar: The Silver Lining Of FDA Commissioner Gottlieb’s Exit

    It was the announcement many have been dreading — but expecting — for several months now. Dr. Scott Gottlieb, 23rd Commissioner of the FDA, announced his decision to step down after 663 days (22 months) on the job. The news is sure to raise concerns for many, from investors to parents of teens tempted to vape. But I think the biggest surprise is how equally sad thought-leaders within the agency will be to say goodbye.

  • Designing Gene Therapy Trials For Access: What Payers & Health Systems Want To See
    Designing Gene Therapy Trials For Access: What Payers & Health Systems Want To See

    Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.

  • Recalibrating The Supply Chain For Allogeneic Cell Therapies
    Recalibrating The Supply Chain For Allogeneic Cell Therapies

    In November 2018, I wrote an article about establishing a supply chain for autologous cell therapies — those formulated using a patient’s own cells. This partner piece highlights considerations that need to be taken into account when developing a commercialization and supply chain strategy for allogeneic cell therapies.

  • 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.

  • 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.

  • Patient-Centricity: Lessons Learned From A Career Inside (And Outside) Big Pharma
    Patient-Centricity: Lessons Learned From A Career Inside (And Outside) Big Pharma

    Today’s clinical trial partner is tomorrow’s post-approval patient. They are the experts who can shape a program over time, be a voice during the regulatory approval process, and provide insight on what other patients hope to find in a therapy.

  • 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.

More From Cell & Gene

C&G EDITOR IN CHIEF, ERIN HARRIS

  • 3 Business Challenges from the CDMO’s Perspective
    3 Business Challenges from the CDMO’s Perspective

    Partnering with a CDMO that marries innovation with technical, regulatory, and manufacturing experience can be cell therapy and gene therapy companies’ best opportunity for scalability and yet their biggest hurdle. From process development through commercial supply and all the steps in between, cell and gene therapy companies expect CDMOs to meet an understandably high bar.

  • News Round-Up: Gottlieb Steps Down, Roche-Spark Tx
    News Round-Up: Gottlieb Steps Down, Roche-Spark Tx

    What a week. As you know, The Washington Post broke the news of Scott Gottlieb’s resignation as FDA Commissioner — the news, while not entirely unexpected, shot disappointment and concern through the entire healthcare industry. Nancy Bradish Myers, JD, president of Catalyst Healthcare Consulting penned an article for Cell & Gene that explains the silver lining of Gottlieb’s exit.

  • 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.

CELL AND GENE INDUSTRY EVENTS

Car-T Congress USA March 20 - 22, 2019
Boston, MA
Responding to Clinical Observations: Developing Robust Audit & Inspection CAPAs April 11 - 11, 2019
Online Training; 1:00 - 2:30 PM EDT US
Aseptic Process Validation: Top-Tips for Compliance and Success April 17 - 17, 2019
1pm-2:30pm EDT, Online Training
ARM Meeting on the Mediterranean April 23 - 24, 2019
Barcelona
Instructional Design for GMP Training – Improve Effectiveness and Measurability April 23 - 23, 2019
1pm-2:30pm EDT, Online Training
American Society of Gene & Cell Therapy (AGCST) April 29, 2019 - May 2, 2019
Washington, DC
Preparing eCTD Submissions: A Step-By-Step Guide April 29 - 29, 2019
1pm-2:30pm EST, Online Training
DIA 2019 Global Annual Meeting June 23 - 27, 2019
San Diego, CA
More Industry Events

INDUSTRY INSIGHTS

  • Freezing Cellular Raw Materials For Cell Therapy Production
    Freezing Cellular Raw Materials For Cell Therapy Production

    Key Biologics is harnessing new freezing technology to help deliver the future of medicine. Digitally enabled cryogenic cold chain technology can now deliver the raw materials needed to manufacture cellular therapies.

  • 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.

More Industry Insights