Noah Stansfield

The company is seeking out other companies to continue development of its autologous CAR-T platform.

The CR was achieved at 1-month post-treatment.

The trial will incorporate findings from CYGNET, a natural history study designed to assess the disease progression of AMN.

NFS-02 previously received orphan drug designation from the FDA in January of this year.

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

The agency granted OCU400 orphan drug designation for additional indications and agreed to a phase 3 trial design for NeoCart.

No dose-limiting toxicities or treatment-related serious adverse events were reported among 24 treated patients.

The hold was originally placed in December 2021 and was related to a case of persistent, non-transfusion-dependent anemia in a pediatric patient.

No treatment-related serious adverse events were reported.

Four of 17 patients who received UCART123v1.2 showed clinical benefit.

The complete metabolic response rate was 70% 3 months after infusion.

100% of patients who achieved transfusion independence reported an overall benefit from treatment.

Among the 38 patients now dosed and evaluable, the ORR remains at 100%.

A complete response rate of 67% was reported and no treatment-limiting toxicity occurred.

REGENXBIO expects to provide updated interim data from the trial in the first half of next year.

IN8bio intends to initiate a multicenter phase 2 clinical trial for INB-400 in glioblastoma in 2023.

The patient tolerated administration of Mylotarg after engraftment.

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

Two of 4 evaluable patients showed stable disease in data presented at ESMO-IO 2022.

Full data readouts have not been shared.

Sensorion indicated it is still on track to submit a clinical trial application in the first half of next year.

There was no significant difference in OS and CR rate for patients based on household poverty and neighborhood opportunity.

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

In preclinical research, TN-401 was shown to significantly increase the lifespan of PKP2-knock-out mice.

The study has now dosed 8 patients in total.

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

Omidubicel’s BLA was originally accepted for priority review in August of this year.

Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.

The gene therapy was approved in the European Union earlier this year.

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