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At 12 weeks posttreatment, all 4 of the patients showed improvement in the form of a lower logMAR.

The move was made in relation to the previously-announced death of a US teenager who received the therapy.

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

The chief executive officer of Eledon Pharmaceuticals also discussed other future plans for the company.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The product is marketed under the name Qfitlia.

Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.

Natalie Goedeker, CPNP, the codirector of pediatric neuromuscular clinical research at Washington University in St. Louis, discussed the session she chaired at MDA’s 2025 conference.

Review top news and interview highlights from the week ending March 28, 2025.

The former FDA commissioner discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s investigational monoclonal antibody and its use along with EGenesis porcine kidney transplant product.

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

In pooled data from 156 patients, there were no deaths or study discontinuations.

These findings could support gene therapies targeting CTG18.1 that are currently in development.

According to Secretome, the first-in-human, open-label trial is the first trial to assess an allogeneic stem cell therapy product for HFpEF.

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, discussed his powerful journey as a patient advocate and gene therapy pioneer, shedding light on challenges and progress in LGMD.

In observance of National Colorectal Cancer Awareness Month, held annually in March, we took a look back at news in cell therapy for colorectal cancer indications.

Michael Flanagan, PhD, the chief scientific officer at Avidity, discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

Review top news and interview highlights from the week ending March 21, 2025.

In observance of Multiple Sclerosis Awareness Month, held annually in March, we took a look back at the past year's news in cell therapy for MS.

The product is marketed under the name Amvuttra.

One of the patients, who was aged 3 years at the time of treatment, achieved 122.3% microdystrophin expression compared to control.

Patients treated at Dose 2 in the dose-escalation phase of Part 1 of the study showed a persistent 68% reduction in CK levels over 2 years.

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

OAV101 IT is a version of Zolgensma that is delivered directly to the spine.

The chief scientific officer at Avidity Biosciences discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

Acute liver injury is known to be a possible adverse event associated with AAV vector-based gene therapies such as Elevidys.