News

Review top news and interview highlights from the week ending September 8, 2023.

Significant improvements in ON time without troublesome dyskinesia were reported with the cell therapy after 1 year, with more apparent effects observed in the high-dose group.

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.

New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.

The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.

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 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.