Latest Conference Coverage

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.

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.

The clinical assistant professor at Stanford Medicine also shared his excitement on the recent approvals of lovo-cel and exa-cel.

The chief medical advisor of the Muscular Dystrophy Association discussed what seasoned clinicians and newcomers to the field alike can look forward to at the 2024 MDA Meeting.

The associate professor at Fred Hutch Cancer Center discussed trends he observed in the field in 2023 and at ASH 2023.

The postdoctoral researcher at Laboratory for Translational Cancer Immunology, Ludwig-Maximilians-Universität München, discussed research he was excited to see at ASH 2023 and in the field in general.

Sharon Hesterlee, PhD, the chief research officer of MDA, discussed highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting.

The Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed unmet needs that remain after gene therapy approval for SCD.

TAC01-CLDN18.2 integrates TAC, Triumvira’s proprietary chimeric receptor that is intended to activate and direct T-cells against tumor cells.

Tami John, MD, a clinical associate professor at Stanford Medicine, also discussed ongoing trends in sickle cell disease research.

Dose level 3 showed the most efficacy and has been selected for the phase 2 portion of the trial.

The chief hematology/oncology fellow at University of Chicago discussed further research his center is working on with cell therapy in B-cell acute lymphoblastic leukemia.

The cofounder and CEO of Neurona Therapeutics discussed the company’s goals to evaluate the cell therapy in more types of epilepsy and potentially even in Alzheimer disease.

The cofounder, executive vice president, and chief medical officer, CLL Society, discussed unmet needs that remain for people with CLL and how the society is working to advance research in the field.

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed research on racial differences in the Factor VIII gene that can impact treatment efficacy for hemophilia A.

The clinical assistant professor at Stanford Medicine discussed outcomes of the first patient that received KMAU-011.