Noah Stansfield

The study will utilize a pediatric safety run-in that will include 2 patients.

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, spoke about how his lab’s findings may imply a need to rethink epilepsy treatment development.

Kyverna stated that the patient, who received KYV-101 in July at the University of Colorado Anschutz Medical Campus, did not experience any ICANS.

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

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, discussed his nonclinical research on disease pathogenesis in MTLE.

Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.

The company also announced its plans for a modified study protocol that may allow a roadway to approval.

Study author Mya C. Schiess, MD, commented on trends seen in the blinded findings so far.

ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (NCT05224505) in Denmark, France, and United Kingdom.

In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.

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.