Victoria Johnson

The sixth participant dosed experienced grade 3 treatment-induced ALT increases and thrombocytopenia.

Data from the OLE of a phase 2 clinical trial at Hadassah Medical Center were presented at the 2024 ACTRIMS meeting.

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

Many surveyed participants were content with current treatments or wary of gene therapy’s adverse events and durability.

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

Cita-cel, approved as Carvykti, is currently under review for an indication expansion as an earlier line of therapy, which the FDA will decide on by April 5, 2024.

Breyanzi was approved for the expanded indication under the accelerated approval pathway.

Investigators found AGTC-501 to have a favorable benefit-risk profile.

Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.

The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy.

The committee is set to discuss idecabtagene vicleucel’s sBLA on March 15, 2024.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.

RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.

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

The patient is doing well so far and may receive a booster dose of the TAC cell therapy.

BioRestorative has submitted an IND amendment to compare BRTX-100 to sham injection instead of saline injection.

Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.

The expanded donor Treg clones maintained lineage fidelity and were persistent through 1 year post-HCT.

Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed the recent axi-cel manufacturing change approval.

Going forward, the dose for patients with CLL has been reduced to 1x106 cells/kg.

Beleaguered company AlloVir has shut down the program before moving on to phase 2.

Differences between IEC and non-IEC post-relapse outcomes showed some significance in PFS but not in ORR.

The researchers are continuing to investigate in vivo efficacy to pave the way to IND-enabling studies.

Patients with high bone marrow burden and CNS/non-CNS EMD were found to experience the worst OS and EFS outcomes.

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

The ULBP6 target was discovered through 23andMe’s proprietary research platform of de-identified human genetic and phenotypic information.

Promising data on OriCAR-017 were previously published in the Lancet Haematology in 2023.

Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed axi-cel's FDA approved manufacturing changes.

Ascidian Therapeutics presented positive preclinical data at the 2023 ASGCT meeting.

The approval follows the separate US approvals in December 2023 and January 2024.