Cell Therapy
Clinical Trials
Gene Editing
Gene Therapy
Viral/Non-Viral Vectors

Key Takeaways from ASGCT 2022

Ryan Leahy
25 May 2022
Ryan Leahy, our Vice President of Research, recently attended his first Annual Meeting of the American Society for Gene and Cell Therapy.

The meeting was a hive of palpable positivity across three floors, with a driving undercurrent across all sessions highlighting exactly how far the cell and gene therapy field has come, with significant emphasis on the potential of cell and gene therapies to treat a variety of novel indications.

Francis Collins was awarded ASGCT’s Founder’s Award, an inaugural accolade for the 25th ASGCT meeting. Collins, who is President Biden’s acting scientific advisor, spoke at length about the strides and successes over the past quarter of a century for advanced therapies, while also highlighting the need for greater progress, particularly in scaling in vivo genome editing and expanding the repertoire of treatable indications. A major emphasis was placed on delivery as the “real challenge” and according to Collins, a “zip code for human tissues” is needed.

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Also recognized was AskBio President Katherine High, who received the Jerry Mendell Award for Translational Science. Dr. High is internationally recognized for her pioneering research in bench-to-bedside and clinical translation of genetic therapies for inherited disorders, and her team achieved the first FDA approval for a gene therapy for genetic disease. Meanwhile, Dr. Donald Kohn received the Outstanding Achievement Award – the organization’s highest honour – for his trailblazing work developing stem cell gene therapies over the past 35 years.

Gene Therapy and Genetic Medicine in Focus

One thing that was staggering was the sheer amount of gene-based therapeutics and gene editing programming across the agenda.

Gene therapy developers turned out in force for the ASGCT meeting, with many touting their developments in AAV – Voyager highlighted their discovery platform for identifying novel capsids targeting desired cells and tissues with improved specificity, as Codiak BioSciences showcased preclinical data for exosome-based enhancements to AAV gene therapy, Sangamo contributed eight abstracts on it’s preclinical-programs emerging from its diverse and versatile genomic engineering platform, and Spark Therapeutics hosted a symposium highlighting the exciting programmes they have in play. Phacilitate favourite, Nicole Paulk (UCSF), chaired a number of sessions focussing on the challenges of developing AAVs for gene therapy, having attended every meeting since 2006.

Particularly exciting top-line data from Rocket Pharmaceuticals showcased positive results in Severe Leukocyte Adhesion Deficiency-I (LAD-I), with all patients showing clinical reversal of disease course. Rocket is looking to file with health authorities and anticipates regulatory filing for the first half of 2023. As reported by CEO Gaurav Shah, “At 3–24 months after RP-L201 infusion, all nine patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events. We are also pleased to report 100% overall survival at 12-months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with 3–24 months of available follow-up. Data also shows evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities.”

Codexis delved in to the preclinical data that supports their gene therapy programmes with improved efficacy for Hemophilia A, Fabry Disease and Pompe Disease with engineered enzyme variants, potentially inspiring a new generation of gene therapies with improved expression profiles. The data was made available across two posters at the meeting.

AvaidoBio presented their pionieering, preclinical data that demonstrates their one-time, AAV gene therapy represents a novel and promising approach for the treatment of frontotemporal dementia. The compelling data in the preclinical study suggests a demonstrated reduction in disease pathology. Taking advantage of existing neural networks to achieve maximal biodistribution with low doses of viral vector.

Further data also supported the use of exon-skipping gene therapy to preserve muscle function for Duchenne Muscular Dystrophy, if delivered early enough. The clinical trials showed production of full-length dystrophin for the first time for patients with DMD who are lacking the protein. While the trials take place with a small study population, the landmark Nationwide Children’s Hospital study supports the therapies potential to safely and effectively increase full-length Dystrophin levels.

Caribou Biosciences presented supporting data for the superior specificity of their genome-editing technology in primary human T cells, and Genenta Science showcased a proprietary stem cell gene therapy for a variety of solid tumor cancers.

FDA Weighs In On the Progress and Future of Cell and Gene Therapy

The USA Food and Drug Administration was present in several sessions across the meeting, most notably a session on interaction with regulatory bodies. Peter Marks highlighted the need for a playbook or platform approach to developing multiple cell or gene therapy products, and that a globally harmonized template would further facilitate the development of these therapies. Also noted by Dr. Marks and Ana Hidalgo-Simon (European Medicines Agency) was the manufacturing and reimbursement challenges that stand in the way of progress.

Is RNA the Way?

Shape Therapeutics advanced programmable medicine with an announcement around the advances to it’s AI-Driven RNA technology platforms. Orna Therapeutics presented their novel, first-in-class circular RNA data for their platform in cancer, genetic disorders and infections diseases, describing Orna’s pipeline for the first time and revealing key data for it’s in-situ CAR program.

Cell Therapy Improvement

While you’d be forgiven for thinking this was a primarily a gene therapy meeting, there have been some significant developments for cell therapy developers and researchers across the sessions. Positie and encouraging data delivered by Poseida for their Allogeneic HSC programmes highlighted the potential in AML, highlighting the broad capabilities of their platform technologies and their potential to improve patient outcomes through off-the-shelf adoptive cell therapy.

Autolus weighed in with their developments in cell programming capabilities to improve precision and control of their highly active products, showcasing three novel approaches. Umoja Biopharma made strides for in vivo cell therapy, and were able to showcase for the first time both their UB-VV100 product candidate with a low risk of off-target transduction and a favourable safety profile as well as their next-generation scalable manufacturing of their lentiviral vector drug product.


A particular highlight for me was a session titled, ‘My Letter to Santa: Wishes and Reality Impacting Cell and Gene Therapy’, hosted by Stefano Baila of Anemocyte and chaired by Anthony Davies, Dark Horse Consulting. The session featured input from Federico Mingozzi (Spark Therapeutics), Jim Faulkner (Ascidian), Alessandro Aiuti (San Raffaele), Miguel Forte (Bone Therapeutics), Nathalie Belmonte (Quell Therapeutics) and Stefan Braam (Ncardia/Cellistic). The session truly explored the depth of how far CGT has come and the breadth of development and progress in this space, as well as what this could look like for Europe and the US. Particular interest was raised in the closing questions: will cell and gene therapies ever become mainstream? The first to answer was Dr. Mingozzi: “I’m a believer that gene and cell therapy will become mainstream, but there’s still work to do”. “The answer is absolutely yes,” followed Dr. Forte.

The major take-away from the ASGCT annual meeting was that the future of gene and cell therapy is particularly young, enthusiastic, diverse and vibrant, with many brilliant scientists showcasing their research via posters and presentation sessions across the meeting.

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