Neurology

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

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

Review top news and interview highlights from the week ending February 16, 2024.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the importance of rigorous clinical trial design for determining whether CAR-T will truly be of benefit in autoimmune disease.

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

The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.

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.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the current standard of care for B-cell driven autoimmune diseases and how CAR-T could address unmet needs.

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

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

Review top news and interview highlights from the week ending February 9, 2024.

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.

The chief scientific officer of CureDuchenne discussed how nonviral approaches may address issues including gene size and redosability.

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

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

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

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.

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

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.

Review top news and interview highlights from the week ending February 2, 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.

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

Efficacy and safety results from the RESKUE clinical trial will be presented at at the WORLDSymposium 2024 on February 9.

SRP-5051 is an investigational peptide-conjugated phosphorodiamidate morpholino oligomer.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed the organization’s research imperatives of 2024.

The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy currently being evaluated in MTLE.