Victoria Johnson

The trial has completed enrollment and all 14 patients have demonstrated islet cell engraftment and endogenous insulin production.

No unifying characteristics were found in a small subset of 6 patients from the BENEGENE-2 trial.

The FDA and EMA have accepted marketing authorization applications and decisions are pending.

The AAV-SLB101 capsid used in SGT-003 may yield increased transgene expression and improved safety compared with first-generation AAV gene therapies.

The approval of exa-cel, the first gene editing therapy, may herald a new era in developing gene editing strategies.

Ocugen and the FDA have agreed on trial design aspects including end point, patient enrollment strategy, and study duration.

CGTLive spoke with Donald Wood, PhD, president and CEO of the MDA while looking ahead to its March conference.

uniQure plans to initiate regulatory discussions in the US and Europe in the first quarter of 2024.

Data from an analysis of patients from the BEYOND study were presented at ASH 2023.

Clinical holds have been placed on BCMA-targeted CT053, GPRC5D-targeted CT071, and Claudin18.2-targeted CT041.

December is HIV/AIDs Awareness Month and CGTLive is taking a look at Excision's first-in-human CRISPR therapy trial for people with HIV-1.

Exa-cel was recently approved for treating sickle cell disease under the name Casgevy.

An analysis of participants from GENEr8-1 who had low transgene-derived Factor VIII expression was presented at ASH 2023.

Rabi Hanna, MD, presented updated data from the RUBY and EdiThal trials at ASH 2023.

Marcela Maus, MD, PhD, Associate Professor of Medicine at Harvard Medical School discussed innovative new research in the field and in her own lab.

A pooled analysis of the ALLCAR19 and FELIX trials including patients with R/R B-ALL, B-CLL, and B-NHL were presented at ASH 2023.

Data from 3 early phase clinical trials assessing BRL-101 in 10 patients were presented at the ASH 2023 meeting.

Stempeutics mesenchymal stem cell therapy is approved in India for treating critical limb ischemia due to multiple causes.

The cell therapy yielded a median overall survival of around 17 months compared with a historical OS of less than 12 months.

ORR was 40% in the 10-mg group and 32% in the 100-mg group.

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

ASC618 is designed to minimize cellular stress and may increase durability of gene therapy for hemophilia.

Meanwhile, the company’s lead CAR Macrophage candidate CT-0508, continues to show efficacy in multiple solid tumor trials.

The IND clearance sets AMT-191 to be evaluated in a clinical trial alongside the company's gene therapies for ALS and Huntington disease.

The company also plans to submit an MAA to the EMA in the first half of 2024.

The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.

Pz-cel has a PDUFA date of May 25, 2024.

GC012F recently yielded a 100% ORR in an investigator-initiated trial in patients with newly diagnosed multiple myeloma.

Maria Pia Morelli, MD, PhD, assistant professor, MD Anderson Cancer Center, discussed her work on the EVEREST-1 trial of logic-gated CAR T-cell therapy currently being evaluated in colorectal and pancreatic cancers.

After a negative AdComm meeting and withdrawing its BLA, the company seeks guidance on a phase 3 trial.