News

The CAR T-cell therapy, marketed as Yikaida in China, has been approved for second-line treatment for patients with refractory/relapsed large B-cell lymphoma.

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 assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.

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.

The chief development officer of Anixa Biosciences discussed how the company is tackling 2 major challenges presented by solid tumors in its ovarian cancer clinical trial.

Stephen Gottschalk, MD, is using grant funding from ACGT to develop CAR T-cell therapy to treat children with sarcomas. He spoke about the challenges of treating sarcomas, the need for new therapies, and the importance of ACGT.

ACGT recently awarded a half-million dollar grant to brain cancer researcher Juan Fueyo, MD, who is developing an oncolytic virus therapy to treat the brain cancer glioblastoma.

September is Childhood Cancer Awareness Month, serving as a chance to assess what progress has been made in advancing cell and gene therapy research for children with cancer.

Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.

Review top news and interview highlights from the week ending September 1, 2023.

In a small sample of patients, the CRISPR–Cas9–edited CD34+ hematopoietic stem- and progenitor-cell therapy showed expected safety while inducing red cell fetal hemoglobin.

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

In recent data from the phase 3 VITAL trial, EB-101 decreased pain and improved wound healing.

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

Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.

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.

TSHA-102 has previously received orphan drug and rare pediatric disease designations from the agency and is being investigated in the REVEAL phase 1/2 trial (NCT05606614).

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.

In a small study, administration of the Généthon therapy encoding UGT1A1 resulted in no serious adverse events. It is also being evaluated in a larger pivotal trial.

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.

The founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.

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.

The company also recently entered into a collaboration with Roche and was invested in by Astellas.

Review top news and interview highlights from the week ending August 25, 2023.

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 interventional cardiologist and professor, Duke University School of Medicine, discussed how XC001 could improve outcomes for patients with refractory angina.