Latest Conference Coverage

The postdoctoral scholar at University of California – Irvine discussed research aiming to link genetic variants and neurodegeneration.

The infectious diseases specialist at Memorial Sloan Kettering Cancer Center discussed the session she chaired for the Infectious Diseases Track at Tandem’s 2024 Meeting.

Aimee C. Talleur, MD, a physician at St. Jude Children’s Research Hospital, discussed obstacles to understanding late effects of CAR-T.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed highlights from the meeting.

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

Tiffany Chen, PhD, the vice president of discovery at GentiBio, discussed the general and unique challenges of transitioning cell therapies to clinical trials in autoimmune disease.

The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital discussed recently approved and still-upcoming gene therapy options for SCD.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed the NIH’s genome editing program and more.

Amar Kelkar, MD, a stem cell transplantation physician at the Dana-Farber Cancer Institute, discussed his research on the value of advanced therapeutics.

The director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine discussed novel innovations that lie on the horizon.

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing and its applications.

Shalini Shenoy, MD, MBBS, discussed when the choice should be made to transition from symptom management to curative therapies.

The best response was very good partial response in 3 of 5 participants.

The associate professor in the Translational Science and Therapeutics Division at Fred Hutch Cancer Center discussed her lab’s research on making TCR T-cell therapies more effective.

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.