Cell & Gene

23/12/2022

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

22/12/2022

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.

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.

21/12/2022

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

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.

21/12/2022

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 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.

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....

21/12/2022

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

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...

15/12/2022

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

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)...

14/12/2022

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 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.

14/12/2022

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.

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...

12/12/2022

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.

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.

09/12/2022

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.

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.

09/12/2022

BlueRock Therapeutics' President and CEO Seth Ettenberg talks to listeners about the company's Phase 1 Clinical Trial for Advanced Parkinson’s Disease. Ettenberg also covers the most promising therapeutic applications for iPSCs currently in development and the major regulatory challenges that the field faces for their clinical use. We also cover how far iPSCs have come, and what future progress may entail.

In partnership with Applied Biosystems by Thermo Fisher Scientific.

08/12/2022

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

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&...

02/12/2022

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

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.

30/11/2022

On this , Orgenesis Inc. CEO Vered Caplan explains the business model behind providing cell and gene therapies that may be manufactured at the point of care.

She explains how working with hospitals and healthcare providers to enable them to participate in CGT development on their sites and providing these therapies in-house by adapting them to closed-system manufacturing rather than relying on the typical biotech model is an important step forward in CGT manufacturing.

In partnership with Applied Biosystems by Thermo Fisher Scientific.

30/11/2022

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.

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.

23/11/2022

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.

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.

18/11/2022

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.

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...

18/11/2022

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.

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. ...

18/11/2022

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.

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...

17/11/2022

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.

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...

14/11/2022

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

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...

11/11/2022

Capstan Therapeutics' CEO, Laura Shawver, Ph.D., talks us through the company's in vivo CAR therapies and what led to the in vivo cell engineering breakthrough that has been decades in the making. Shawver explains the promise of harnessing and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo. She also details best practices to garner funding and investment partnership. In partnership with Applied Biosystems by Thermo Fisher Scientific.

10/11/2022

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.

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. 

07/11/2022

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.

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...

04/11/2022

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

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.

03/11/2022

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 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.

02/11/2022

"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."

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...

28/10/2022

PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases has focused on delivery.

SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differs from other techniques in genetic medicine, and much more.

is brought to you in partnership with Applied Biosystems by Thermo Fisher Scientific.

26/10/2022

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

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.

24/10/2022

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

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...