Latest Conference Coverage

Beleaguered company AlloVir has shut down the program before moving on to phase 2.

The medical director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the pros and cons of potentially curative treatment options for SCD.

Differences between IEC and non-IEC post-relapse outcomes showed some significance in PFS but not in ORR.

The researchers are continuing to investigate in vivo efficacy to pave the way to IND-enabling studies.

Patients with high bone marrow burden and CNS/non-CNS EMD were found to experience the worst OS and EFS outcomes.

Funding from ACGT will help Chiocca perform FDA-required studies on mice to show the oncolytic virus is safe and can be manufactured for humans. It is the second such grant awarded to Chiocca from ACGT.

The assistant professor at Mayo Clinic School of Medicine discussed the design of the phase 1 trial.

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

The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed challenges on the horizon in this rapidly emerging field.

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 executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

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

The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed the panel he participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.

The clinical assistant professor at Stanford Medicine discussed potential applications for machine learning in analyzing data in medicine.

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

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

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.

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

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.

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.

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.

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed how a personalized gene editing approach may help patients avoid development of FVIII inhibitors.

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.

The internal medicine resident physician at University of Kansas Medical Center also discussed highlights from the ASH 2023 meeting.

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.