WORLDSymposium

Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.

Hematological recovery was achieved and maintained in all 4 patients in the study.

Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial.

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed the limitations of the current standard of care for Fabry disease.

Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.

4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.

All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.

Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.

Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.

Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.

Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.

Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.

Following treatment with AT845 gene therapy, 3 of 4 patients withdrew from their previous enzyme replacement therapy.

Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.

The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.

Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.

FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.

The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.