2024 American Society of Gene and Cell Therapy Annual Meeting

Tuesday, May 7—Saturday, May 11, 2024
The Baltimore Convention Center, Baltimore, Maryland, USA

We had a wonderful time attending the largest international gathering of gene and cell therapy scientists.

The American Society of Gene and Cell Therapy’s (ASGCT) annual meeting is the premier event for professionals in gene and cell therapy. The meeting is the best place for people in the field to learn from the latest scientific research, stay up to date on new technologies and make career-advancing connections with peers. Originally designed as a venue for academic researchers to share their work, the annual meeting has grown to serve a wide community including clinicians, bio-industry development, regulatory agencies, equipment manufacturers and patient advocates.

MaxCyte showcased our cGMP-compliant nonviral cell engineering platform and how we can accelerate therapeutic development from concept to clinical utility.

Featured Presentation

Overcoming Challenges in Translating Research Development to a Scalable Clinical Manufacturing Process

Thursday, May 9, 2024, from 8:30 to 9:30 a.m. ET in Room 309-310

From bench to bedside. We will explore the complexities of translating research into scalable manufacturing processes for cell and gene therapies. Executives from the industry will share their insights and innovative strategies for navigating obstacles, the importance of collaboration and technological innovation as well as regulatory compliance to help advance transformative therapies. Join us as we delve into the critical intersection of research development and clinical manufacturing to accelerate the development of life-saving treatments. This session will include a roundtable discussion followed by a presentation from KSQ Therapeutics on developing a scalable cGMP process for their engineered tumor-infiltrating lymphocyte (eTIL™) cell therapy.

Speakers during our presentation

Maher Masoud Maxcyte

Maher Masoud

President and Chief Executive Officer at MaxCyte

Maher Masoud is the President and Chief Executive Officer of MaxCyte and has more than 25 years of experience in the biopharmaceutical industry. Maher previously served as Executive Vice President, Head of Global Business Development and Chief Counsel, at MaxCyte. Maher started his biopharmaceutical career as a research associate with Glen Research, before joining Human Genome Sciences as Director and Corporate Counsel, overseeing legal activities for the company’s global clinical trials. Prior to joining MaxCyte, he oversaw the operations of six business subsidiaries at Wellstat. During his tenure at Human Genome Sciences and Wellstat, Maher supported the launch of three FDA-approved therapies—Benlysta®, Vistogard® and Xuriden®. Maher earned his Juris Doctor degree from Michigan State University College of Law after completing his degree in cell and molecular biology genetics from the University of Maryland.

Picture of Jason Bock

Jason Bock

Chief Executive Officer at CTMC

Jason Bock, PhD, is Chief Executive Officer of CTMC, a joint venture between Resilience and MD Anderson Cancer Center. CTMC was formed in May of 2022 to accelerate patient access to impactful cell therapies by bridging cell therapy development and manufacturing with MD Anderson’s clinical trial capabilities. In 2019, Jason was recruited by MD Anderson from Teva Pharmaceuticals to build the Biologics Development group. The group purchased a 60,000-square-foot facility in the Texas Medical Center and has since formed multiple partnerships with both MD Anderson faculty and early-stage biotech firms to bring their products through the investigational new drug process. Previously, Jason was Site Head and Vice President of Global CMC Biologics in the Specialty R&D Division of Teva Pharmaceuticals. He joined Teva through the acquisition of CoGenesys, a private biotech firm as a spinoff from Human Genome Sciences, where he worked after completing a PhD at Stanford University in molecular and cellular physiology.

Picture of Tom Leitch

Tom Leitch

Chief Technology Officer at KSQ Therapeutics

Tom Leitch, Chief Technology Officer at KSQ Therapeutics, has nearly 25 years of leadership experience in cell and gene therapy, biologics, and vaccines and earned engineering degrees from Virginia Tech. His experiences span manufacturing sciences, CMC strategy development, internal and external manufacturing operations, quality, engineering, technology transfer, and supply chain at multiple leading biopharmaceutical companies.

Prior to KSQ Therapeutics, Tom was the head of manufacturing at bluebird bio and led the development and execution of the company’s manufacturing strategy during a period of rapid growth that expanded the network to include more than ten internal and external manufacturing sites around the world. At bluebird, Tom was instrumental in the regulatory approval of multiple cell therapy products.

Earlier in his career, Tom was responsible for all commercial and clinical manufacturing activities at Alexion’s manufacturing plant in Smithfield, Rhode Island. Tom also spent fourteen years at Merck in positions of increasing responsibility in technical operations and quality.

Photograph of Ben Askin

Ben Askin

Scientist, Cellular Process Development, at KSQ Therapeutics

Ben Askin is a seasoned scientist with over nine years of experience in pharmaceuticals and biotechnology. Currently serving as a key member of the Cellular Process Development Team at KSQ Therapeutics, Ben brings a wealth of expertise spanning the scientific, manufacturing and quality domains.

During his tenure at Kite Gilead EU in Hoofddorp, Netherlands, Ben played a pivotal role in the successful launch of the European manufacturing site for the groundbreaking CAR-T therapy, Yescarta®. His contributions were instrumental in facilitating a seamless technical transfer and ensuring compliance during the European Medicines Agency's site inspections.

Ben holds a Bachelor of Science degree in Chemistry and Psychology from The College of New Jersey.

Networking Event

Wrap the day with fellow researchers while meeting the MaxCyte team

Wednesday, May 8, 2024, from 7:00 to 9:30 p.m. ET
Blackwall Hitch at 700 E Pratt St, Baltimore, MD 21202

We are inviting all attendees of the ASGCT annual meeting to an evening of networking. Join us at this exclusive event where you will have the opportunity to meet the MaxCyte team. Enjoy complimentary hors d’oeuvres and beverages while mingling with fellow scientists and researchers.

Registration for this event has now closed.

Brick building displaying the Blackwall Hitch sign above the restaurant doors

Oral presentation

Non-Viral Engineering of Primary Human Macrophages Using a cGMP-Compliant Electroporation Platform

Presented by Ashley Strickland-Dietz, PhD, Immunology Scientist I at MaxCyte
Friday, May 10, 2024, in Ballroom 2 from 5:15 to 5:30 p.m. ET

Macrophages are highly plastic innate immune cells that can exhibit pro- or anti-inflammatory effector functions depending on their activation state. For this reason, macrophages can be used for various therapeutic applications ranging from cancer treatment to regenerative medicine. To engineer macrophages for these purposes, biomolecules or other genome editing tools must be delivered into these cells. Electroporation, also known as electropermeabilization, is an effective nonviral transfection method that utilizes an electric field to facilitate the passage of molecules across biological membranes. Here, it is demonstrated that highly efficient delivery of mRNA, DNA and CRISPR ribonucleoproteins (RNPs) into primary human macrophages can be achieved using the MaxCyte ExPERT GTx system - a cGMP-compliant electroporation platform. In fact, use of the ExPERT GTx to transfect macrophages with mRNA or SIRPα-targeting CRISPR RNPs resulted in efficiencies greater than 90% with minimal impact on cell viability. This system also enabled delivery of DNA, ranging in size from 2.5-kb to 9.2-kb, into macrophages with transfection efficiencies between 60% and 90%. These efficiencies were inversely related to plasmid size and were accompanied by a dose-dependent decrease in cell viability. Despite this, electroporation was determined not to directly impair migration, activation/polarization, or phagocytosis in viable macrophages. For further proof-of-concept, the ExPERT GTx was also used to generate clinically relevant chimeric antigen receptor (CAR)-expressing macrophages targeting the tumor antigen CD19. These CAR macrophages were found to be functional and highly effective at eradicating CD19+ target cells. Together, these demonstrate that the MaxCyte ExPERT GTx electroporation platform can be used to engineer macrophages for use in cell therapy applications.

Poster presentations

Non-Viral Cell Engineering of Peripheral Blood Natural Killer Cells Using a GMP-Compliant, Scalable Electroporation Platform

Photo of Lauren Unsworth

Poster 861 presented by Lauren Unsworth, Research Associate II at MaxCyte
Wednesday, May 8, 2024, in the Exhibit Hall from 12:00 to 7:00 p.m. ET

Natural killer (NK) cells are an integral part of the innate immune system as they recognize an abundance of surface receptors on target cells without the need for antigen specificity and recruit and activate other immune cells by secreting a myriad of cytokines. The hallmark of NK cells is their cytotoxic function which enables them to recognize and attack tumor or infected cells by releasing granules that induce apoptosis of target cells. Electroporation (EP) is a nonviral method for cellular engineering that has been used with virtually all cell types and has been specifically shown to be an effective cell engineering method for peripheral blood NK cells. Advantages of electroporation include highly efficient transfection with a variety of molecules (e.g., DNA, RNA, and ribonucleoprotein (RNP) complexes), regulatory acceptance, and cGMP compatibility. In this study, we optimized conditions for electroporating primary NK cells with the GMP-compliant MaxCyte ExPERT GTx electroporation platform, with specific focus on maximizing cell viability, cell recovery, transfection efficiency, cell expansion and functionality. Previous studies showed that expansion of NK cells before electroporation was necessary for effective transfection, and therefore larger scale platforms were required, leading to the use of more costly reagents. Here, we showed that the MaxCyte ExPERT GTx could genetically modify naive or expanded primary NK cells using mRNA, RNP complexes or plasmid DNA at both a small (1x106 cells in 25µl) and larger scale (1x108 cells in 5ml). We observed over 90% gene expression and viability following mRNA electroporation and over 60% expression and viability after minicircle DNA (mcDNA) loading at both small and larger scales. We also evaluated the effect of commercially available DNA sensing pathway inhibitors to determine if transfection efficiency and cell viability could be increased when loading small and large plasmid DNA. Furthermore, we knocked-out the NKG2A inhibitory receptor at different timepoints during expansion by loading a sgRNA-Cas9 RNP complex and looked at post-electroporation effects. With our optimized transfection conditions specific for NK cells, we determined that electroporation had minimal impact on cell viability, cell expansion, surface marker expression and killing capacity.

Efficient Scalable Manufacturing of Virus-Like Particles for the Delivery of CRISPR-Cas9 Ribonucleoproteins Using a cGMP-Compliant Electroporation Platform

Picture of Peter Gee

Poster 1733 presented by Peter Gee, PhD, Senior Field Application Scientist at MaxCyte
Friday, May 10, 2024, in the Exhibit Hall from 12:00 to 7:00 p.m. ET

Genome editing tools such as CRISPR-Cas9 nucleases, base editors, and prime editors hold tremendous promise for treating human diseases by being able to specifically modify a targeted region of the DNA genome. However, there are limited in vivo delivery options that are safe, efficient and transient. Moreover, viral vectors such as AAV are hampered by limited cargo size capacity. Virus-like particles (VLPs) offer a solution to these problems. VLPs are derived from retroviral structural proteins, which can be engineered to specifically package a cargo of interest. They are safer than traditional viral vectors because they lack a viral genome but can utilize the traditional virus delivery machinery to target and enter cells. Recently, VLPs have been reported to efficiently package base editors and prime editors for delivery into mice at therapeutically relevant levels. To realize the potential of VLPs for in vivo delivery, alternative methods to scale up their manufacturing will be critical for future clinical applications. Here, we utilized the MaxCyte ExPERT GTx, a cGMP-compliant electroporation instrument, to manufacture VLPs— packaged with CRISPR-Cas9 RNPs for genome editing in target cells — in adherent and suspension HEK293 cells. We found that electroporation consistently produced significantly higher yields of functional VLPs compared to a commercially available transfection reagent, with up to a 20-fold improvement when using an optimized electroporation protocol. Furthermore, the production of VLPs using electroporation exhibited favorable production kinetics compared to other transfection methods, enabling a shorter VLP manufacturing process. Finally, we demonstrated the scalability of VLP production across a 15-fold volume range with minimal re-optimization. In summary, our results show that electroporation is a viable means for consistent, efficient and scalable manufacturing of VLPs for gene-editing applications and has high promise to address needs for future clinical and commercial VLP manufacturing.