Latest Conference Coverage

Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.

Adipose-derived MSCs did significantly increase salivary flow rate from baseline in patients with previous head and neck cancer.

The stem cell transplantation physician at the Dana-Farber Cancer Institute discussed how to look at the value therapies provide and how to improve the cost-effectiveness of their use.

The cells showed activity toward PDAC cells as well as cancer-associated fibroblasts, which may address challenges with the tumor microenvironment.

The postdoctoral scholar at University of California – Irvine discussed further questions he is continuing to investigate.

Haydar Frangoul, MD, discussed advantages and disadvantages of haploidentical bone marrow transplant and the 2 new gene therapies for SCD.

Nirav Shah, MD, an associate professor of medicine at Medical College of Wisconsin, discussed LV20.19, a bispecific CD19/CD20-targeted CAR-T being evaluated for chronic lymphocytic leukemia and Richter’s transformation.

Uttam Rao, MD, MBA, a transplant physician at St. David's South Austin Medical Center of Sarah Cannon, discussed research comparing patient outcomes on different conditioning regimens for CAR-T.

The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.

The assistant professor at Mayo Clinic School of Medicine discussed plans for further research and a phase 2/3 study.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed how there is still much room for growth for RNA therapeutics despite progress so far.

Vivien Sheehan, MD, PhD, an associate professor of pediatrics at Emory University, discussed how patients should be able to choose to try the recently FDA-approved cell-based therapies for SCD when they are ready.

The clinical investigator in the Department of Bone Marrow Transplantation & Cellular Therapy at St. Jude Children’s Research Hospital discussed several areas of interest for research now that CAR-T is here to stay.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed her research on siRNA technology.

The transplant physician at St. David's South Austin Medical Center of the Sarah Cannon Transplant and Cell Therapy Network discussed a real-world study comparing bendamustine against fludarabine and cyclophosphamide.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the importance of further research into the root cause of MS and other autoimmune diseases.

The associate professor of pediatrics at Emory University also discussed the need to empower patients and families to make their own treatment decisions.

Avidity is currently enrolling in the phase 3 HARBOR trial, which it expects to initiate in the second quarter of 2024.

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his view of the main priorities for research in this area.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed considerations for evaluating CAR-T in patients most likely to obtain meaningful benefit.

Updated data from the RESPOND study evaluating Spinraza in patients with SMA after onasemnogene abeparvovec were presented.

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his team’s early findings on CAR-T and fertility from CIBMTR centers.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the potential impact of CAR-T therapy on the lives of patients with autoimmune disease.

Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from a session on rare disease gene therapy at MDA.

The lead scientist at Percheron Therapeutics discussed the design of the study assessing proteomics of ATL1102.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the need for randomized-control trials for CAR-T in lupus and other autoimmune diseases.

The Assistant Professor of Medicine at Weill Cornell Medical College discussed Orca-T cell therapy and next steps assessing it.