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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 company plans to initiate a pivotal trial of 4D-710 in the second half of 2025.

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

One participant experienced a possibly related serious AE of pigmentary changes in the macula with severe vision reduction.

Review top news and interview highlights from the week ending April 12, 2024.

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 data comes from 2 phase 1/2a clinical trials and their respective open-label extension studies.

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.

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

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

The 2-part, multicenter BCT-006-US trial will seek to enroll patients who are in the earlier stages of ALS.

The director of Cell Therapy and Transplant at Penn Medicine discussed his outlook on CAR T-cell therapy research and the future.

AURN001 consists of allogeneic human corneal endothelial cells referred to as “neltependocel” and Y-27632, a small molecule drug.

Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed his lab’s research in the field of neonatal cell therapy.

Caribou stated that it anticipates initiating its planned phase 1 GALLOP clinical trial for CB-010 in patients with LN and ERL by the end of 2024.

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

Eque-cel was previously evaluated in antibody-mediated idiopathic inflammatory disorders of the nervous system in an investigator-initiated trial.

The assistant professor dermatology at the Northwestern University Feinberg School of Medicine discussed the potential for further research enabled by B-VEC.

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.

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

The FDA’s decision was based on data from the phase 3 CARTITUDE-4 clinical trial (NCT04181827).

Review top news and interview highlights from the week ending April 5, 2024.

The approval comes a couple weeks after ODAC voted in favor of Abecma’s benefit-risk profile and sBLA.