Noah Stansfield

The data comes from 7 patients treated in the phase 1/2 IGNITE-DMD study (NCT03368742).

The real-world data, which includes patients who received Zolgensma before and after tracheostomy, comes from the noninterventional RESTORE registry (NCT04174157).

The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a closer look at the clinical evaluation of hearing loss gene therapy DB-OTO.

In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.

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

The long-term data comes from 2 cohorts in the phase 2 CARTITUDE-2 clinical trial.

The data comes from an expanded access protocol (EAP; JCAR017-EAP-001) that included 96 patients in total who were treated with the nonconforming product.

Juliane Gust, MD, PhD, an assistant professor of neurology at University of Washington, Seattle Children's, discussed a study that is currently underway via the NIH’s CARnation Consortium that will seek to address this gap in knowledge.

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

Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, discussed clinical trial design considerations for this emerging field.

The Prescription Drug User Fee Act date for the FDA’s decision on the supplement has been set for June 21, 2024.

David Porter, MD, the director of cell therapy and transplant at Penn Medicine, discussed the importance of sharing knowledge in the emerging field of cell therapy for autoimmune disease.

The first 3 participants in the MUSIC-HFpEF trial have now been dosed with the AAV vector-based gene therapy.

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

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what he views as the next horizon in the field of gene therapy.

Hemogenyx noted that the agency confirmed all issues pointed out in the initial clinical hold letter from June 2023 had been sufficiently addressed by the company.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed his research on a new application of efgartigimod alfa (Vyvgart).

At the 24-week analysis, patients who received the high dose achieved an 89% decrease in annualized antiVEGF injection rates.

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

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.

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

ETX101 consists of a transgene encoding a GABAergic regulatory element and an engineered transcription factor that is delivered via a nonreplicating, recombinant AAV9 vector.

The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed MDA Care Centers and his excitement for the organization’s the Upcoming Clinical & Scientific Conference.

AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed future plans for the expanded applications of the allogeneic regenerative neural cell therapy.

The Prescription Drug User Fee Act (PDUFA) target action date has been set for August 4, 2024.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed the ongoing first-in-human clinical trial evaluating the cell therapy for drug-resistant mesial temporal lobe epilepsy.