Latest Conference Coverage

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.

Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.

Catch up with some of CGTLive’s top conversations from the annual ASTCT and CIBMTR meeting.

Alison Betof Warner, MD, PhD, assistant attending physician, Memorial Sloan Kettering Cancer Center, discussed the solid tumor session she chaired at the 2023 Tandem Meetings.

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.

The associate professor of neurology at Columbia University spoke about the session he will be chairing at MDA’s 2023 conference.

The associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.

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.

The MD, PhD candidate at Baylor College of Medicine discussed findings from a new study presented at the 2023 Tandem Meetings.

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.

The assistant professor of BMT and cellular therapy discussed updated safety and efficacy data on CD22 CAR T-cell therapy.

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.

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 assistant professor of medicine and pediatrics at Stanford University discussed updated data from 2 studies of Orca-T cell therapy.

The professor medicine and pediatrics at Washington University in St. Louis overviewed data from the SPEARHEAD-1 trial of afami-cel.

By 3 months, 12 of 17 patients treated with a full cycle of EG-70 achieved a complete response.

The assistant professor of medicine at Vanderbilt University Medical Center discussed results from a retrospective analysis presented at the 2023 Tandem Meetings.

It was found that ctDNA levels at 28 days post-treatment were significantly higher for patients who ultimately progressed by 90 days post-treatment.

The assistant professor of medicine, bone marrow transplantation and cellular therapy, Stanford, discussed favorable data from a real-world experience study.