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The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.

At the 90 day timepoint, the patient showed improvements in swallowing compared to their own natural history data, as measured by videofluoroscopic swallowing studies.

The associate professor of dermatology at Stanford University discussed how the May 2023 approval of B-VEC may shift the treatment field for RDEB.

Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.

The medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.

Review top news and interview highlights from the week ending April 19, 2024.

The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 CAR-T therapies currently on the market.

Nanoscope Therapeutics received feedback from the FDA endorsing significant change in BCVA as a primary endpoint supporting approval.

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

Data from a meta-analysis of 3 groups of patients were presented at AAN 2024.

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.

For World Hemophilia Day, CGTLive takes a look at how the hemophilia field has adapted to the introduction of gene therapy to the treatment landscape.

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

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

An interim analysis of a phase 1/2 study suggest the REGENXBIO therapy also showed efficacy signals.

The specific autoimmune disease(s) that will be evaluated in the trial is as of yet undetermined, but the company hopes to initiate the trial in Q4 of 2024.

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

In light of the IND clearance, Tr1X anticipates initiating a phase 1 clinical trial within the second quarter of this year.

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

The more recent data were presented at the 2022 ASGCT Meeting.

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 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.