The recently concluded American Society of Gene and Cell Therapy (ASGCT) 2024 annual meeting showcased the cutting-edge advances in gene and cell therapy.
At least 2,000 abstracts were presented including a number of clinical trials offering insights into the latest scientific endeavors. Our team has been analyzing the data and brings you a powerful search tool. Get access to more functions and features to help you dig deeper into the ASGCT abstracts and authors, and find more leads faster. See how to get instant access below! 👇
These clinical trials pave the way for potential breakthroughs in human health and paint a promising picture for the future of gene and cell therapy. Take a look at some of our interesting findings:
Clinical Trials Data
66 abstracts focus on clinical trials
10 CAR-T
35 Gene therapy
4 Gene editing
Phase 1, 2, 3 breakdown (see graph)
Breakdown:*
Phase 1:
47 abstracts
9 CAR-T
23 Gene therapy
4 Gene editing
Phase 2:
7 abstracts
1 CAR-T
4 Gene therapy
None for gene editing
Phase 3:
5 abstracts
1 CAR-T
3 Gene therapy
None for gene editing
^Footnote:
*Some data overlap
*9 studies are post-hoc analysis, observational or pilot study
FIG. 1: ASGCT 2024 Clinical Trial Abstracts Breakdown
Durability of Treatment
Great progress has been made on cell and gene therapies over the past decade. But are these treatments durable?
Some noteworthy findings:
Abstract 243: (CAR-T for neuroblastoma) presented the longest duration data
13.9 yr median follow-up in 19 pts followed up (1 out to 18+ yrs)
2 of 3 CRs lasted at least 8 yrs (1 lasting 18+ yrs)
5 of 8 high risk pts are disease free at 13.9 yrs
Abstract 1: Lenmeldy, the first approved treatment for MLD from Orchard Therapeutics
6.76 yr median follow-up (out to 12 years in 1 pt)
Replaced gene (ARSA) function maintained throughout follow-up
95% of pre-symptomatic pts at time of admin retained ability to walk at last follow-up
Abstract 246: RP-L201 gene therapy in pediatric patients with severe LAD-I
Presented up to 3.9 yrs follow up data from RP-L201 Ph1/2 trial
No RP-L201 serious adverse events were reports throughout duration
Durable correction in LAD-I in all 8 patients with at least 2 yrs follow-up
Abstract 8: University of Pennsylvania researchers analyzed data from CAR-T with 1,323 patient-years of follow-up
Patients treated with Gamma- and Lenti- vector-modified T-cell gene addition analyzed.
No transformation or clinically concerning clonal skewing and pathogenic insertional mutagenesis found.
FIG. 2: Clinical Trials Treatment Durability
Gene Editing Abstracts
We found 4 gene editing - clinical trial abstracts:
Abstract 147 - Nuclease-Free Genome Editing (GeneRide) in Pediatric Methylmalonic Acidemia*
Technology: Proprietary (GeneRide [synthetic viral vector])
Disease: Methylmalonic Acidemia in Pediatric Patients
Phase: I/II
Failed to reach outcome levels needed to demonstrate therapeutic benefit. But successfully demonstrated in vivo homologous recombination genome editing without the use of nucleases or promoters in pediatric population.
Abstract 244 - BRL-101 - CRISPR-Cas9 Editing of BCL11A Enhancer in Beta-Thalassemia (TDT)*
Technology: CRISPR
Disease: Transfusion-Dependent β-Thalassemia
Phase: I/II
Demonstrated clinically meaningful increases in efficacy outcomes and safety profile consistent with prior reports - supports continued investigation of BRL-101 for TDT.
Abstract 381 - Guide RNA Site Consistency Across Ancestries and Potential for Off-Target Editing of VERVE-101*
Technology: Proprietary (VERVE-101 [in vivo base editing medicine])
Disease: Heterozygous Familial Hypercholesterolemia
Phase: I
1st in vivo base editing medicine to demonstrate proof-of-concept in humans. Off-target assessment in different cellular contexts suggests highly specific with a low risk for clinically relevant off-target edits.
Abstract 1632 - Beyond AAV, Gene Editing of HSV in Herpetic Stromal Keratitis*
Technology: CRISPR
Disease: Herpetic Stromal Keratitis
Phase: N/A (investigator-initiated)
Intrastromal injection of the virus-like particle-delivered CRISPR was well tolerated with safety and efficacy data suggesting in vivo CRISPR delivery with VLP holds great potential in treating ocular diseases.
*Titles shortened by BiopharmIQ team
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Article History:
AV, MV, JD, DG (05/13/24)
This article is not investment or legal advice.
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