Victoria Johnson

Rocket submitted additional CMC data in response to FDA information requests.

The phase 3 trial of tivanisiran failed its primary endpoint.

The BAFFR-CAR T-cell therapy is being investigated in a phase 1 single-center and phase 1 multicenter trial in B-ALL and B-NHL.

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

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.

CDR-Life also recently presented positive preclinical data at the November 2023 SITC Annual Meeting.

Vor Bio recently also announced data from the phase 1/2 trial of trem-cel.

Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.

The company also shared that it has manufactured sufficient IDP-023 to supply the Phase 1 clinical trial through the second half of 2024.

FT825 is an iPSC-derived multiplexed-engineered, CAR T-cell therapy targeting HER2 with a novel antigen binding domain.

Sana also recently announced data from the first patient treated with its CD19-targeted CAR.

An investigator-initiated trial presented positive data at the ESGCT annual meeting in October 2023.

BriaCell also shared specific positive data from one patient with proptosis and ocular pain.

Speech perception was also greatly improved in the children with hearing recovery.

The immune therapy is a replication-incompetent human tumor cell line designed to conjugate to and prime resting natural killer cells.

The trial is assessing satri-cel CAR T-cell therapy, which is currently on clinical hold due to CMC concerns.

Kiromic announced that the first patient had been dosed with the gamma delta cell therapy in December 2023.

Dose level 3 showed the most efficacy and has been selected for the phase 2 portion of the trial.

Updated data from the AAVIATE trial were presented at the Hawaiian Eye and Retina meeting.

Three of CARsgen’s CAR therapies are currently on clinical hold due to CMC questions.

The therapy’s original PDUFA date was March 30, 2024, but the TDT approval followed only a month after exa-cel's sickle cell approval.

Further research is needed to determine if cell therapy may be beneficial to certain subgroups of patients.

The authors noted that this kind of trial is exceedingly complex and future papers will propose further conclusions.

The trial is recruiting 5 more participants for the highest dose cohort following a positive DSMB review.

Kadimasem’s lead clinical candidate, AstroRx, was recently cleared for trials in the US for people with ALS.

Atara plans to submit a BLA for tab-cel, approved in Europe, in the second quarter of 2024.

University College London has initiated the single site trial at Great Ormond Street Hospital, London.

There was 1 serious AE of pneumonia which was found to be possibly related to 4D-710.

Altogether, there was a 91% reduction in median serum TTR at day 28.