Latest Conference Coverage

uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.

The senior vice president and head of research at Kyverna Therapeutics discussed the company’s investigational CAR-T therapies, KYV-101 and KYV-201.

The vice president of discovery at GentiBio discussed the panel she participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.

The life science research professional at Stanford University discussed preclinical research she presented at the American Heart Association’s Scientific Sessions 2023.

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, spoke about his presentation on gene therapy for cardiologists at AHA’s 2023 Scientific Sessions.

The research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.

The postdoctoral research fellow at Cedars Sinai Medical Center discussed the future of RNA therapy in the context of research in systemic sclerosis he presented at AHA’s Scientific Sessions 2023.

The date for the AdComm and the new PDUFA date are yet to be announced.

Eli Lilly’s small interfering RNA therapy showed significant serum reductions with a single dose while remaining well-tolerated. A larger phase 2 study is currently ongoing.

The director of the Mass General Brigham Gene and Cell Therapy Institute shared his main message for cardiologists at the American Heart Association’s Scientific Sessions 2023.

The professor of medicine at University of California San Diego discussed promising preclinical findings she presented at the American Heart Association’s Scientific Sessions 2023.

Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.

The postdoctoral research fellow at Cedars Sinai Medical Center discussed preclinical research he presented at the American Heart Association’s Scientific Sessions 2023.

The director of the Mass General Brigham Gene and Cell Therapy Institute discussed the history and future of gene therapy in cardiology at the American Heart Association’s Scientific Sessions 2023.

One serious AE of myocardial infarction may have been related to VERVE-101 therapy while other AEs of fatal cardiac arrest and tachycardia were determined to be unrelated to the therapy.

The median progression-free survival was 11 months and the SCG101 was well-tolerated.

Microsatellite instability (MSI) tumors are frequently found in people with Lynch syndrome, one of the most common hereditary colorectal cancers.

BRL Medicine is also enrolling a phase 1/2 multicenter trial evaluating BRL-201 at lower dose ranges.

The BASECAMP-1 screening study has now identified 28 patients whose solid tumors are positive for HLA-A*02 LOH.

David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.

Patients with HNSCC had a median overall survival (OS) of 20.8 months and a 12-month OS rate of 59%.

In terms of safety, it was noted that repeated administration of CAR mRNA/LNP was well-tolerated by mice.

CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy.

Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.

The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed findings from several preclinical studies that could help bring engineered B-cell therapies to clinical trials.

Richard James, PhD, an associate professor at the University of Washington and a principal investigator at Seattle Children's Research Institute, discussed a potential alternative to T-cell therapy in cancer.