• A Technology Roadmap For Today’s Gene Therapy Manufacturing Challenges
    A Technology Roadmap For Today’s Gene Therapy Manufacturing Challenges

    In Nov. 2018, The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) published technology roadmaps addressing needs and gaps in three key product areas: gene therapy, antibody-dug conjugates, and vaccines. The roadmaps were developed with the collaborative input of industry, academic, and government experts. This piece highlights technology opportunities for gene therapy manufacturing.

  • Toward A More Robust Regenerative Medicine Regulatory Pathway
    Toward A More Robust Regenerative Medicine Regulatory Pathway

    In recent months, the U.S. Food and Drug Administration has increased its compliance focus on the regenerative medicine industry, vowing to crack down on illicit stem cell clinics and others advertising the untested use of human cell and tissue-based products (HCT/P), many of which are used in orthopedics.

  • What Investors Want: 7 Key Components To Raising Capital
    What Investors Want: 7 Key Components To Raising Capital

    ARM’s 2019 Cell & Gene Therapy Investor Day featured expert-led panels to help the audience wrap their minds around the investment outlook for the cell and gene therapy space. One such panel, aptly titled, “The Investment Outlook for the Cell and Gene Space,” was comprised of investors from DEFTA Partners, Roivant Sciences, Aisling Capital, and Aquilo Capital Management. Dr. Reni Benjamin, Managing Director, Biotechnology Equity Research at Raymond James & Associates moderated the panel.

  • CDMO Selection: 3 Questions To Save Time And Reduce Stress When Reviewing An MSA
    CDMO Selection: 3 Questions To Save Time And Reduce Stress When Reviewing An MSA

    Whether because of unfamiliarity or lack of resources, master service or supply agreement (MSA) and Supply Agreement execution can be time consuming and difficult. At any given pharma company in the development or clinical stage, the leadership team and investors prioritize patients, therapies, and trial results.

  • Refrigerated Transport For Gene Therapy Products — Exploring The Pros And Cons
    Refrigerated Transport For Gene Therapy Products — Exploring The Pros And Cons

    The viability and cell count of immunotherapies are extremely sensitive to temperature. Additionally, these products demonstrate reactivity to cooling and thawing rates between room temperature and refrigerated/frozen temperatures.1 The same sensitivity applies to apheresis material, which is susceptible to failure if not fully protected against temperature fluctuations.

  • ARM’s Lambert Recaps 2019 Cell And Gene Investor Day
    ARM’s Lambert Recaps 2019 Cell And Gene Investor Day

    ARM’s 2019 Cell and Gene Investor Day took place on March 21st in New York, NY. Now in its seventh year, Investor Day provides insight into financing opportunities for cell and gene therapy-based treatment and tools. I caught up with ARM’s CEO and Cell & Gene Editorial Advisory Board member, Janet Lambert, after the event to discuss top takeaways, near-term financing opportunities, and more.

  • 4 Strategies For Success In The CAR-T 2.0 Marketplace
    4 Strategies For Success In The CAR-T 2.0 Marketplace

    As the second wave of CAR-T therapies begins coming to market in the next five years, their manufacturers will need to anticipate and prepare to address many of the same commercial challenges as their predecessors, as well as several additional ones.

  • Cryogenic Transport For Gene Therapy Products — The Pros And Cons
    Cryogenic Transport For Gene Therapy Products — The Pros And Cons

    Temperature-controlled packages used for the transport of gene therapy products use a combination of thermal insulators or thermoregulators to decrease the heat transfer rate from outside of the shipper to the inside. The overall design of the package is pivotal in achieving the minimal heat transfer rate.

  • ARM’s Cell & Gene Investor Day 2019
    ARM’s Cell & Gene Investor Day 2019

    Today, March 21st, is ARM’s 7th Annual Cell & Gene Investor Day. The New York City-based one-day event provides institutional, strategic, and venture investors with unique insight into financing opportunities for cell and gene therapy-based treatment and tools.

  • 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.

  • Big Data And AI In Pharma Development And Manufacturing: An Inside Look
    Big Data And AI In Pharma Development And Manufacturing: An Inside Look

    Artificial intelligence and Big Data have the potential to lower the cost and time of drug trials, to better determine patient outcomes with established drugs, and to better design new drugs.

  • 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.

More From Cell & Gene

C&G EDITOR IN CHIEF, ERIN HARRIS

  • What Investors Want: 7 Key Components To Raising Capital
    What Investors Want: 7 Key Components To Raising Capital

    ARM’s 2019 Cell & Gene Therapy Investor Day featured expert-led panels to help the audience wrap their minds around the investment outlook for the cell and gene therapy space. One such panel, aptly titled, “The Investment Outlook for the Cell and Gene Space,” was comprised of investors from DEFTA Partners, Roivant Sciences, Aisling Capital, and Aquilo Capital Management. Dr. Reni Benjamin, Managing Director, Biotechnology Equity Research at Raymond James & Associates moderated the panel.

  • ARM’s Lambert Recaps 2019 Cell And Gene Investor Day
    ARM’s Lambert Recaps 2019 Cell And Gene Investor Day

    ARM’s 2019 Cell and Gene Investor Day took place on March 21st in New York, NY. Now in its seventh year, Investor Day provides insight into financing opportunities for cell and gene therapy-based treatment and tools. I caught up with ARM’s CEO and Cell & Gene Editorial Advisory Board member, Janet Lambert, after the event to discuss top takeaways, near-term financing opportunities, and more.

  • 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.  

CELL AND GENE INDUSTRY EVENTS

Stability Programs – Key Factors in Meeting FDA/ICH Expectations April 22 - 22, 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
Essentials of Disinfectant Efficacy Testing – Ensuring Microbial Control April 25 - 25, 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
Critical Thinking In Clinical Research – A Better Way May 7, 2019
11am-12:00pm EDT, Online Training
DIA 2019 Global Annual Meeting June 23 - 27, 2019
San Diego, CA
More Industry Events

INDUSTRY INSIGHTS

  • Cell Therapy Manufacturing: The Supply Chain Challenge
    Cell Therapy Manufacturing: The Supply Chain Challenge

    Overcoming the challenges of manufacturing and administering a complex product to any patient is critical for delivering cell therapies. Here two industry experts provide practical advice on strategies to meet supply chain challenges.

  • 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.

More Industry Insights