Top News from WORLDSymposium 2023

WORLDSymposium 2023, held February 22-26, in Orlando, Florida, showcased data from clinical trials across rare, lysosomal diseases including Krabbe disease, Gaucher disease, metachromatic leukodystrophy, Pompe disease, mucopolysaccharidosis, Fabry disease, and others. CGTLive rounded up some top coverage of WORLDSymposium across diseases to keep readers up to date on the biggest conference news.

Increased Motor Development Seen in First 2 Patients With Infantile Krabbe Treated With FBX-101

FBX-101 (Forge Biologics) gene therapy restored galactocerebrosidase (GALC) activity and supported normal white matter development in the first 2 patients with infantile Krabbe disease (IKD) dosed in the phase 1/2 RESKUE clinical trial (NCT04693598).

Gaucher Disease HSC Gene Therapy Demonstrates Efficacy and Safety in Phase 1/2 Trial

AVROBIO’s AVR-RD-02, an investigational hematopoietic stem cell (HSC) gene therapy being evaluated for the treatment of Gaucher disease type 1 (GD1), has demonstrated efficacy and safety in interim data from the phase 1/2 Guard1 clinical trial (NCT04145037).

Arsa-cel Shows Favorable Risk/Benefit Profile in up to 11 Years of Follow-up Data

Long-term data of up to 11 years of follow-up on the gene-edited cell therapy atidarsagene autotemcel (arsa-cel; Orchard Therapeutics) has demonstrated a continued favorable risk/benefit profile inpatients with pre-symptomatic (PS) late-infantile and early-juvenile (EJ), and early-symptomatic (ES) EJ metachromatic leukodystrophy (MLD).

Late-Onset Pompe Disease Gene Therapy Shows Promise in Phase 1/2 Study

Astellas Pharma’s AT845, an investigational adeno-associated virus (AAV) vector-based gene replacement therapy intended to treat late-onset Pompe disease (LOPD) has demonstrated encouraging efficacy in interim data from the phase 1/2 FORTIS clinical trial (NCT04174105).

MPSII Gene Therapy Shows Some Signs of Durable Efficacy

REGENXBIO’s gene therapy RGX-121 has demonstrated safety and efficacy in improving development in children with mucopolysaccharidosis type 2 (MPSII), according to interim data from the phase 1/2 CAMPSIITE trial (NCT03566043).

Fabry Disease Gene Therapy Demonstrates Favorable Impact in Phase 1/2 Trial

Treatment with Sangamo Therapeutics' isaralgagene civaparvovec (ST-920), an investigational AAV2/6 human a-Gal-A gene therapy for Fabry disease being evaluated in the phase 1/2 STAAR clinical trial (NCT04046224), showed a favorable safety profile and resulted in elevated alpha-galactosidase A (a-Gal A) activity, which suggests a favorable impact on progression of Fabry neuropathy.