Noah Stansfield

Among 11 treated patients, 8 achieved a complete response.

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Cellectis noted that the allogeneic product was manufactured at its new in-house facility in Paris.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.

Verismo Therapeutics’ SynKIR-110 previously received orphan drug designation from the FDA.

The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Investigator Matthew Charman, PhD, told CGTLive™ that the new knowledge of the viral replication process could potentially inform the development of future gene therapies.

CB-011 and CB-010 are being evaluated for the treatment of r/r multiple myeloma and r/r B-cell non-Hodgkin lymphoma, respectively.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Direct Biologics’ ExoFlo is also being evaluated for the treatment of COVID-19 acute respiratory distress syndrome.

Patients in the UK with r/r multiple myeloma may still be able to access the CAR-T through ongoing clinical trials, which are not impacted by the decision.

Intergalactic Therapeutics’ IG-002 utilizes a proprietary non-viral delivery method.

Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.

GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.

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.

The planned clinical trial of EB103 has received clearance to include patients with HIV-associated lymphoma and primary and secondary central nervous system lymphoma.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news in gene therapies for hemophilia—one of the most common of these disorders.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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.

Three patients treated with Genethon’s GNT-003 were able to cease treatment with standard of care phototherapy for at least 1 year.

The company has halted phase 3 plans for BIVV003 in favor of therapies for Fabry and immune rejection in kidney transplantation.

AskBio’s AB-1003 was previously granted fast track designation by the FDA.

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

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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