LOS ANGELES—The 2023 American Society of Gene & Cell Therapy (ASGCT) annual meeting wrapped up last week with a record attendance and membership. It featured a wide array of news and announcements on clinical trials, gene editing and delivery technologies, rising applications of artificial intelligence (AI), and company launches.
This year’s hybrid meeting marked the largest gathering in the meeting’s 26-year run, with nearly 8,000 total registrants and some 6,600 scientists, physicians, patient advocates, and professionals attending the meeting in-person. Gone were the organizational challenges of a few years ago, as the Society’s booming membership numbers outstripped the modest convention facilities. The organizers were clearly having fun—ASGCT executives were greeted with booming walk-on music as they were introduced, and an exhibit hall DJ briefly threatened to give those in the vicinity hearing damage.
When asked about the state of gene therapy in 2023 in a pre-ASGCT interview with GEN, Terry Flotte, MD, provost/dean at the University of Massachusetts Chan Medical School, said, “we’re continuing to have great dissemination of the technology to more patient populations. New therapies are being approved by the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) as clinical trials are advancing.”
Flotte, who also serves as the Editor-in-Chief of GEN’s sister journal, Human Gene Therapy, and was recently announced as ASGCT’s incoming vice president (and future president), specifically looked forward to the talk by head hematologist at Boston Children’s Hospital, David Williams, MD, describing advances on ex vivo hematopoietic stem cell gene therapy. Williams was awarded the ASGCT Founders Award on Day one of the meeting this year, an honor presented to former NIH director Francis Collins, MD, PhD in 2022.
Key talks continued into Day two with a Presidential Symposium opened by Nobel laureate and Innovative Genomics Institute (IGI) co-founder, Jennifer Doudna, PhD who highlighted her group’s recent work applying CRISPR to T-cell therapies. David Liu, PhD, Harvard University and Broad Institute chemist and the developer of base and prime editing, followed up with an impressive array of encouraging preclinical studies applying “CRISPR 2.0” editing technologies to diseases such as sickle cell disease and spinal muscular atrophy.
The same Presidential Symposium announced the selection of eight rare diseases for the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) clinical portfolio. The consortium, hosted under the Foundation for the National Institutes of Health (FNIH), is a public-private partnership aiming to increase efficiency by standardizing manufacturing and streamlining regulatory pathways for these rare disease gene therapies.
News in the industry
The ASGCT show floor also saw new company launches. Adeno-associated virus (AAV) expert Nicole Paulk, PhD, unveiled Siren Biotechnology, a Bay Area start-up that combines AAV gene therapy with cytokine immunotherapy into a new treatment modality to fight cancer. Paulk, formerly on the faculty of University of California San Francisco, orchestrated a well-coordinated launch party with a trio of presentations. Siren will be starting with brain and eye cancer in its efforts to tackle solid tumor cancer therapy.
Form Bio, a provider of computational life sciences technology and an offshoot of George Church’s Colossal Biosciences, announced the launch of FORMsight AI, an AI-based solution for predicting and optimizing manufacturing outputs of cell and gene therapy constructs. Form Bio also announced its designation as a Google Cloud Premier partner, giving organizations the option to incorporate Form Bio’s platform when adopting Google Cloud’s Multiomics Suite.
Mammoth Biosciences highlighted their ultracompact protein (<500 amino acid) NanoCas platform, which fits completely within a single AAV and leaves more than half of the space available for additional cargo such as regulatory elements, fusion proteins, gRNAs, and more. At ASGCT, Janice Chen, PhD, Co-founder and CTO of Mammoth, presented data showing that NanoCas is capable of robust in vivo editing in mice, thereby supporting one and done precision editing approaches to unlock new therapies.
Kelonia Therapeutics, revealed the results of preclinical research demonstrating that its in vivo Gene Placement System (iGPS) efficiently delivered CAR molecules specifically to T cells at therapeutic dose levels in both mice and non-human primates. Kelonia CSO Kevin Friedman, PhD, stated that iGPS lacks the toxicities and chemotherapy associated with standard CAR T and has potentially more persistent and durable clinical benefit without the time-consuming ex vivo manufacturing issues.
Dyno Therapeutics announced the launch of bCap 1TM capsid, a central nervous system (CNS)-targeted AAV gene delivery vector created by generative AI. Dyno states that the vector has been validated in non-human primate (NHP) cells and provides improved pan-brain neuronal transduction and liver detargeting at a low intravenously administered dose.
Tune Therapeutics presented data on the first successful example of epigenetic editing in non-human primates targeting PCSK9, a protein responsible for regulating cholesterol in the bloodstream and a common therapeutic target for the prevention of cardiovascular disease.
Chroma Medicine, another epigenetic editing company, gave oral presentations describing its first proof of concept editor achieving near-complete in vivo silencing efficiency and Chroma’s editors’ potential for genotoxicity-free multiplex editing.
REGENXBIO presented 15 abstracts at ASGCT, many focusing on the advantages and challenges working with AAV. The company has a growing pipeline of several gene therapies, including treatments for rare diseases (Hunter’s Syndrome, Duchenne Muscular Dystrophy) as well as more common afflictions such as wet-aged macular degeneration and diabetic retinopathy.
Touting itself as “the future of programmable RNA medicine,” ShapeTX featured in seven presentations during the week, including new data from the company’s RNAfix®, RNAswapTM, AAVidTM, and TruStableTM platforms. The breadth of data “highlights the arsenal of disruptive technologies we’ve built,” said Francois Vigneault, PhD, co-founder and CEO. “By integrating massive parallel biological screens, advanced bioinformatics, and generative deep learning models with RNA technologies, we are accelerating drug discovery for previously untreatable diseases.”
Ultragenyx, a biopharmaceutical company focused on developing novel therapies for rare diseases, presented clinical, preclinical and manufacturing data from its investigational gene therapy programs. Oral presentations included longer term durability data from DTX301 for the treatment of ornithine transcarbamylase (OTC) deficiency, and DTX401 for the treatment of glycogen storage disease type Ia (GSDla). Both clinical trials are AAV8 gene therapies advancing into Phase III.
Chinese contract development and manufacturing organization (CDMO) uBriGene celebrated its foothold in the US market with the acquisition of a state-of-the-art GMP manufacturing facility from Mustang Bio in Worcester, Mass. The facility offers facilities for production of cell and gene therapies, including cGMP manufacturing of multiple gene-modified cell types. uBriGene will take over clinical manufacturing of Mustang Bio’s MB-106, a CAR-T cell therapy treatment for hematologic malignancies.
A miscellaneous collection of additional exhibitor press releases is provided by ASGCT online.
Looking ahead, ASGCT 2024 will be held May 7–11th in Baltimore, MD. Further developments in the field will be presented at the European Society of Gene & Cell Therapy (ESGCT) 30th annual congress from October 24–27 in Brussels, Belgium.