Victoria Johnson

After submitting an amended IND, Adverum’s gene therapy is set to be evaluated in the phase 2 LUNA trial.

Verve Therapeutics is currently conducting a clinical trial in New Zealand and expects to be able to open trials in the UK and US later in 2022.

SCG101 will be evaluated in trials across China, Singapore, and the US.

A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials.

A phase 1 trial has completed enrollment in its first cohort and is now enrolling the second.

Myrtelle’s rAAV-Olig001-ASPA was granted fast track, rare pediatric disease, and orphan drug designations in March 2022.

The FDA has lifted a clinical hold on the trial of VX880 placed in May 2022 due to insufficient information.

ADVM-022 was also granted PRIME designation by the EMA on June 24, 2022.

The hold comes after a serious AE of peripheral sensory neuropathy.

The approval builds off of liso-cel's (Breyanzi) previous indication for third-line or later treatment in LBCL.

BioMarin received a positive CHMP opinion after the FDA delayed val-rox's BLA filing for the second time.

B-VEC recently showed efficacy in the phase 3 GEM-3 trial.

NfL peaked 1-month post-treatment as expected and returned to near baseline at 12 months.

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

Annualized bleeding rate fell to 0 at a median of 58 weeks after infusion.

Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed different indications for the iNKT therapy AgenT-797.

ACE1831 will soon be evaluated in a phase 1, first-in-human, clinical trial.

The therapy is now being evaluated in a phase 2 study.

Mustang Bio presented data on MB-106 at the 2022 EHA Congress.

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

Updated date from the B-LIEVE study is expected in the second half of 2022 and first half of 2023.

The trial was placed on hold after elevated liver function test scores.

GC012F also showed efficacy in B-cell non-Hodgkin lymphoma.

The CD-19 CAR T-cell therapy is developed using Curocell’s OVISTM platform.

Results from the CLIMB THAL-111 and CLIMB SCD-121 trials were presented at the EHA 2022 Congress.

Response rates were significantly higher but patients had more cases of ICANS and CRS with axi-cel treatment compared to tisa-cel treatment.

Statistically significant differences were seen in global health status, pain, and fatigue.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee again voted in favor of the risk-benefit of bluebird bio's gene therapy candidate.

Three of 6 patients have an ongoing response.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted in favor of the benefits of bluebird bio's eli-cel in patients with cerebral andrenoleukodystrophy despite substantial safety concerns.