Noah Stansfield

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

The therapy is expected to be able to treat both the lung and liver manifestations of AATD.

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

Acclaim-1, Acclaim-2, and Acclaim-3 will evaluate Reqorsa in combination with osimertinib, pembrolizumab, and atezolizumab, respectively.

It was the first time the Eye Center treated a patient with a gene therapy.

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

Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed what the company learned from relitigating results of legacy trials for a mesenchymal stem cell therapy in Crohn disease.

ATSN-201 utilizes Atsena’s novel spreading capsid AAV.SPR, which is expected to spread laterally and allow transduction of the gene therapy in the central retina.

The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.

In light of the promising results, BlueRock is preparing for a phase 2 trial.

NKGen Biotech’s trial for SNK02 was initiated following the clearance of an investigational new drug application by the FDA in October 2022.

Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.

Because of the lack of neurotoxicity seen in patients treated thus far, Nexcella believes NXC-201 may have potential to serve as an outpatient treatment.

The single-arm, open-label clinical trial will seek to enroll approximately 18 patients aged 18 to 75 years.

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

Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.

The company also reported that WU-NK-101 has received orphan drug designation for treating acute myeloid leukemia from the FDA.

BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.

AlloNK will be evaluated as part of a combination therapy with anti-CD20 monoclonal antibody rituximab.

NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.

IMPT-514 uses the same CAR-T construct as ImmPACT Bio’s IMPT-314, which is already under clinical evaluation for non-Hodgkin lymphoma.

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

The new orphan drug designation follows a recently received fast track designation for Reqorsa in combination with Tecentriq for an extensive-stage small cell lung cancer indication.

The partial clinical hold was originally placed in June 2023 in relation to the death of a patient participating in the trial.

The formal hold comes approximately 2 months after Seattle Children’s paused the trial in accordance with the clinical study protocol stopping rules.

In terms of safety, Taysha stated that TSHA-102 was well-tolerated and that at 6 weeks after dosing no treatment-emergent serious adverse events had occurred.

Kyle Garland, PhD, a senior scientist at Eterna Therapeutics, discussed the company’s preclinical work with partner Factor Bioscience that was presented at ASGCT’s 2023 conference.

Aspen Neuroscience noted that the study will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy.

KMA.CAR-T is directed at Kappa Myeloma Antigen, a receptor exclusively appearing on myeloma cells in kappa-type MM.

LION-CS101, the study evaluating gene therapy AB-1003, is currently recruiting patients aged 18 years through 65 years with LGMD2I/R9 who have a confirmed mutation in FKRP.