Cell & Gene

Shoreline Biosciences, Inc.'s Chief Scientific Officer Dr. Robert Hollingsworth, Ph.D. shares his insight on intelligently designed allogeneic off-the-shelf, standardized, and targeted iPSC-derived natural killers (NK) and macrophage cellular immunotherapies on this brought to you in partnership with Applied Biosystems by Thermo Fisher Scientific.

He explains the company's preclinical testing on a potential NK cell therapy for acute myeloid leukemia. We discuss how the field recognizes the importance of off-the-shelf NK cell therapy for cost reduction and improving safety measures. As such, Dr. Hollingsworth discusses why NK cells are necessary to move immunotherapies forward.



https://www.cellandgene.com/doc/inside-nk-and-macrophage-cellular-immunotherapies-with-shoreline-biosciences-cso-dr-robert-hollingsworth-0001?utm_source=editorpost&utm_medium=link&utm_campaign=socialmedia

Anna Rose Welch shares a few of the actionable takeaways she walked away with from the ARM/ASGCT potency assay working session in the hopes they will be a complementary exploration of the analytical challenge impacting every ATMP company.

Overcoming CGT Potency Assay Woes: 4 Best Practices Here, I share a few of the actionable takeaways I walked away with from the ARM/ASGCT potency assay working session in the hopes they will be a complementary exploration of the analytical challenge impacting every ATMP company.

To advance the field of immuno-oncology, we must shift our mindsets and ways of working to embrace agile practices and new levels of knowledge sharing – both within and between organizations.

By Peter Sandor, Astellas Pharma US

Igniting Innovation Through Partnership: A New Model To Accelerate Cancer Cell Therapy For Patients To advance the field of immuno-oncology, we must shift our mindsets and ways of working to embrace agile practices and new levels of knowledge sharing – both within and between organizations.

Fair warning, Cell & Gene readers. While this list includes some of my favorite editorial from 2022, it’s not even remotely close to being as extensive as I’d like. Why is that? Our library of content is so deep and so rich, I couldn’t possibly share and recap all of it with you in one article. Here are some highlights that helped to inform our readers about everything from process development to vendor selection to partnering with the right investment team.

My Top Editorial Picks for 2022 Fair warning, Cell & Gene readers. While this list includes some of my favorite editorial from 2022, it’s not even remotely close to being as extensive as I’d like. Why is that? Our library of content is so deep and so rich, I couldn’t possibly share and recap....

In August, Judge Bernal of the Central District of California ruled that stem cells that are removed from a patient, centrifuged, and reinjected into a patient are not “drugs” and therefore do not require FDA premarket approval. FDA filed a notice of appeal in October, but the appeals process will take time. Until then, the decision may have consequences for stem cell therapy companies.

By Chad Landmon and Gulrukh Haroon, Axinn, Veltrop & Harkrider LLP

The Regulatory Labyrinth Of Stem Cell Treatments In August, Judge Bernal of the Central District of California ruled that stem cells that are removed from a patient, centrifuged, and reinjected into a patient are not “drugs” and therefore do not require FDA premarket approval. FDA filed a notice of appeal in October, but the appeals...

Knowledge of the applicable rules and legal challenges, as well as ways to ensure the enforcement of proprietary rights, requires companies to invest in negotiating and drafting robust and balanced . This article provides practical tips (for both the principal and the CDMO) for drafting these vital legal agreements.

By Mauro Turrini, Jean-Christophe Troussel, Nadia Feola, and Auriane Schockaert, Bird & Bird

Make The Service Agreement Work For You And Your CDMO Knowledge of the applicable rules and legal challenges, as well as ways to ensure the enforcement of proprietary rights, requires companies to invest in negotiating and drafting robust and balanced CDMO service agreements. This article provides practical tips (for both the principal and the CDMO)...

Leveraging human genetics data early in the drug discovery process to inform target prioritization is a powerful approach to mitigate risk and increase the likelihood of advancing safe therapies that transform patient’s lives.

By Abhinav Dhall, Carolyn Wills, Nathan Jorgensen, Tirtha Chakraborty, and John Lydeard, Vor Bio

Leveraging Human Genetics To Advance Cell Therapies For Treatment Of Blood Cancers Leveraging human genetics data early in the drug discovery process to inform target prioritization is a powerful approach to mitigate risk and increase the likelihood of advancing safe therapies that make a transformative impact in patients’ lives.

Here, I offer my biggest takeaways from a recent ARM/ASGCT workshop on one of the most popular topics to gossip about both in and outside the analytical labs: Potency assays. In part one of this two-part article, I start by unpacking what I see as the most prominent knowledge gaps and questions underpinning the industry’s potency assay challenge.

CGT Potency Assays: When Will We Wake Up From The Nightmare? Here, I offer my biggest takeaways from a recent ARM/ASGCT workshop on one of the most popular topics to gossip about both in and outside the analytical labs: Potency assays. In part one of this two-part article, I start by unpacking what I see as the most prominent knowledge gaps and questions u...

In the final part of the series, experts from Kite Pharma, Novartis Gene Therapies, and SwanBio Therapeutics explain how to drive down and expedite commercial-scale manufacturing in the space.

How To Reduce COGS & Expedite Commercial-Scale CGT Manufacturing (Part 2) In the final part of the series, experts from Kite Pharma, Novartis Gene Therapies, and SwanBio explain how to drive down COGS and expedite commercial-scale manufacturing in the CGT space.

In part one of this two-part series, as seen in Life Science Leader magazine, experts from Kite Pharma, Novartis Gene Therapies, and SwanBio Therapeutics weigh in on understanding how to drive down COGS and expedite commercial-scale manufacturing in the CGT space.

How To Reduce COGS & Expedite Commercial-Scale CGT Manufacturing (Part 1) In part one of this two-part series, as seen in Life Science Leader magazine, experts from Kite Pharma, Novartis Gene Therapies, and SwanBio weigh in on the understanding of how to drive down COGS and expedite commercial-scale manufacturing in the CGT space.

To figure out what the future holds for companies in the RNA sector of the ATMP space, I sat down with four executives to discuss their wildest hopes, dreams, and expectations. As you’ll note, there is no shortage of work facing the RNA therapeutics industry in the year(s) ahead. But there’s also a lot for which we can hope.

Featuring Michelle Lynn Hall, Eli Lilly and Company; Jacob Becraft, Strand Therapeutics; Thomas McCauley, Omega Therapeutics; and Christopher Anzalone, Arrowhead Pharmaceuticals

mRNA & RNA Therapeutics In 2023: Where Do We Go From Here? To figure out what the future holds for companies in the RNA sector of the ATMP space, I sat down with four executives to discuss their wildest hopes, dreams, and expectations. As you’ll note, there is no shortage of work facing the RNA therapeutics industry in the year(s) ahead. But there&...

To ensure patient access to life-saving therapies, resilient business processes are a critical priority for cell and gene therapy companies, and resilience against cyber-attacks is a critical component of this overall strategy.

By Accenture

Building Cyber Resiliency In Cell And Gene Therapy Manufacturing To ensure patient access to life-saving therapies, resilient business processes are a critical priority for cell and gene therapy companies, and resilience against cyber-attacks is a critical component of this overall strategy.

To better understand 2023’s most pressing issues in the cell and gene therapy space, I sought expert commentary from Cartesian Therapeutics’ Murat Kalayoglu, Poseida Therapeutics, Inc.’s Mark Gergen, REGENXBIO Inc.'s Nina Hunter, and Spark Therapeutics, Inc.’s Ron Philip.

CGT In 2023 With Cartesian Therapeutics, Poseida Therapeutics, REGENXBIO, And Spark Therapeutics To better understand 2023’s most pressing issues in the cell and gene therapy space, I sought expert commentary from Cartesian Therapeutics’ Murat Kalayoglu, Poseida Therapeutics’ Mark Gergen, REGENXBIO’s Nina Hunter, and Spark Therapeutics’ Ron Philip.

To learn more about the near-term future of , the advantages of short interfering RNA ( ) over other therapeutics, and more, our own Erin Harris caught up with DTx Pharma's CEO Arthur Suckow, Ph.D.

DTx Pharma And The Near-Term Future Of RNA Therapeutics To learn more about the near-term future of RNA therapeutics, the advantages of short interfering RNA (siRNA) over other RNA therapeutics and more, I caught up with DTx Pharma's CEO Arthur Suckow.

I chatted with Tom Payne, Laverock Therapeutics' Chief Operating Officer, about the company’s mission to utilize its Gene Editing induced Gene Silencing (GEiGS) technology and its associated computational platform to engineer induced pluripotent stem cells (iPSC)-derived cell therapies with improved efficacy, safety, and accessibility.

A Growing Emphasis On Gene Silencing I chatted with Tom Payne, Laverock Therepeutics' Chief Operating Officer, about the company’s mission to utilize its Gene Editing induced Gene Silencing (GEiGS) technology, and its associated computational platform, to engineer induced pluripotent stem cells (iPSC)-derived cell therapie...

Translational research tools, including biological assays (bioassays) and biological markers (biomarkers), are needed to bridge the gap from understanding the disease process to identifying therapeutic targets and testing potential therapies in preparation for clinical research in humans.

On November 15, Gregory J Opiteck, Ph.D., VP Head of Translational Sciences at Allogene Therapeutics and Douglas Sanders, Ph.D., VP Head of Translational Medicine at AVROBIO joined Cell & Gene Chief Editor Erin Harris to cover assay and vendor selection in support of allogenic CAR T therapy as well as the various modalities of AAV gene therapy.

Evaluating Translational Research Tools For CGTs Translational research tools, including biological assays (bioassays) and biological markers (biomarkers), are needed to bridge the gap from understanding the disease process to identifying therapeutic targets and testing potential therapies in preparation for clinical research in humans. ...

Here's a breakdown of our most recent Evaluating Translational Research Tools For CGTs, featuring industry experts Gregory Opiteck of Allogene Therapeutics and Douglas Sanders of AVROBIO.

ICYMI: Cell & Gene Live, Evaluating Translational Research Tools For CGTs Here's a breakdown of our most recent Cell & Gene Live Evaluating Translational Research Tools For CGTs, featuring industry experts Gregory Opiteck, Ph.D., VP, Head of Tran...

Alliance for Regenerative Medicine (ARM)’s Meeting on the Mesa (MOTM) 2022 was held October 11-13, 2022, in Carlsbad, CA. The meeting was packed with good information, and I wanted to share just a few of the most important soundbites I gleaned from the event. So, whether you attended the event or want to know what you may have missed, here are some highlights from MOTM 2022.

Meeting On The Mesa 2022: Big Pictures & Takeaways Alliance for Regenerative Medicine (ARM)’s Meeting on the Mesa (MOTM) 2022 was held October 11-13, 2022 in Carlsbad, CA. The meeting was packed with good information, and I wanted to share with you just a few of the most important soundbites I gleaned from the event. So, whether you attende...

While have been previously developed against the Ebola virus, the COVID-19 vaccines were the first widespread global application of this vaccine platform. Adenovirus vectored vaccines have unique that must be tested to ensure quality and safety.

By Diane McCarthy, U.S. Pharmacopeia

Assessing Quality Of Viral Vectored Vaccines While viral vectored vaccines have been previously developed against Ebola virus, the COVID-19 vaccines were the first widespread global application of this vaccine platform. Adenovirus vectored vaccines have unique critical quality attributes that must be tested to ensure quality and safety...

Life Science Leader’s annual Industry Outlook issue hits mailboxes the first week of December. In it, I contributed an article about the future of commercial manufacturing featuring commentary from Chris Fox, Novartis Gene Therapies; Karen Kozarsky, SwanBio Therapeutics; and Vaishali Shukla, Kite Pharma. We didn’t have room in the issue for all the valuable information they shared, so here’s what didn’t make it to print.

Life Science Leader's Annual Industry Outlook Issue: What Didn't Make It To Print Cell & Gene’s sister publication and fellow Life Science Connect resource, Life Science Leader magazine, is the essential business journal for life science executives who work for everything from emerging biotechs to Big Pharmas, aka you. 

Oligonucleotide therapeutics have the potential to improve the lives of millions of people and alter morbidity and mortality outcomes in multiple diseases and disorders that were previously uninsurable, particularly neurodegenerative diseases.

The Promising Future Of Oligonucleotides Episode 35 of Cell & Gene: The Podcast features Dr. Michelle Mellion, PepGen’s SVP Head of Clinical Development at Cambridge, MA-based biotech PepGen. In it, Dr. Mellion talks all about the company’s oligonucleotide therapeutics that were developed to treat severe neuromuscular an...

This article compares fundamentals of current and upcoming lentiviral production processes and highlights areas of improvement that would increase manufacturing cost-effectiveness. Strategies discussed are production methods, cellular platforms, and bioprocessing options.

By Aziza Manceur, Martin L., and Renald Gilbert, National Research Council Canada

Cost-Effective Biomanufacturing Of Lentiviral Vectors This article compares fundamentals of current and upcoming lentiviral production processes and highlight areas of improvement that would allow an increase in manufacturing cost-effectiveness. Strategies discussed are production methods, cellular platforms, and bioprocessing options.

The principal obstacle currently limiting the widespread application of lifesaving gene therapy technology is the safe and efficient in vivo transfer of the editing constructs into the right cells.

By Jonathan Thon, STRM.BIO

The Future Of Gene Therapy Depends On New Delivery Vehicles The principal obstacle currently limiting the widespread application of lifesaving gene therapy technology is the safe and efficient in vivo transfer of the editing constructs into the right cells.

"While I cannot define precisely what (or which) shape(s) risk-sharing will take for the multitude of unique CGT products in the works," writes Anna Rose Welch, "I’ve been privy to several conversations that go a long way toward defining the current state of these evolving ATMP outsourcing partnerships and business models."

"Lean On Me": Defining Risk-Sharing In Cell & Gene Therapy Manufacturing Partnerships While I cannot define precisely what (or which) shape(s) risk-sharing will take for the multitude of unique CGT products in the works, I’ve been privy to several conversations that go a long way toward defining the current state of these evolving ATMP outsourcing partnerships and business m...

This article is the final installment of a three-part series examining how advanced therapy scientific and quality expectations are impacting outsourcing business practices/models today and, hopefully, creating a foundation for greater risk-sharing in th

"Risky Business:" How Cell & Gene Therapies Are Upending Partnership Models This article is the final installment of a three-part series examining how advanced therapy scientific and quality expectations are impacting outsourcing business practices/models today and, hopefully, creating a foundation for greater risk-sharing in the future.

Fortunately, the cell and gene therapy ( ) revolution coincides with advanced digital technologies. At the forefront are digital twins, which combine at-scale computing, modeling methods, and IoT connectivity to create full-scale digital replicas of physical assets or real-world processes, such as factories, value chains, and even humans. CGT can harness this digital twin technology.

By Kerim Ozbilge and Frank Traina, Ernst & Young

Digital Twins & The CGT Value Chain: A Universe Of Possibilities Fortunately, the cell and gene therapy (CGT) revolution is coinciding with advanced digital technologies. At the forefront are digital twins, which combine at-scale computing, modeling methods, and IoT connectivity to create full-scale digital replicas of physical assets or...