Latest Conference Coverage

There were no serious adverse events reported and no patients discontinued from the study.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.

All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.

The CEO, founder, and president of Siren Biotechnology discussed the preclinical data she presented at ASGCT’s 2023 meeting on the use of AAV immuno-gene therapy for high-grade gliomas.

The chief scientific officer at Candel Therapeutics discussed late-breaking data presented at ASGCT 2023.

The trial compared data from external controls including a recent domagrozumab null study and a propensity score weighted comparison.

The senior investigator at the National Cancer Institute Center for Cancer Research discussed updated data from a phase 1/2 trial presented at ASGCT 2023.

The chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

The neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.

The senior research fellow at Fred Hutch Cancer Center discussed findings from the PLAT-08 trial presented at ASGCT 2023.

All 8 patients with light chain amyloidosis who received HBI0101 responded.

The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,

The professor of medicine at University of California San Diego discussed new research from her lab presented at the 2023 ASGCT meeting.

The chief scientific officer of Sangamo Therapeutics discussed data from 2 of the company’s programs that were presented at ASGCT’s 2023 conference.

The senior scientist at Eterna Therapeutics discussed the company’s numerous presentations at ASGCT’s 2023 conference.

Data trends suggesting stabilization of disease were seen in patients with Tay-Sachs, but more research is needed, especially for those with Sandoff disease.

Updated data from the RESTORE study on MCO-010, Nanoscope Therapeutics' investigational gene therapy, were presented at the 2023 ASGCT meeting.

The vice president of research at Prime Medicine discussed a preclinical study on a novel autologous HSCT treatment that was presented at ASGCT’s 2023 annual meeting.

Following implantation with CellGenTech’s LCAT-GMAC, the patient’s serum LCAT activity increased by approximately 50% of baseline.

ImmTOR induces immune tolerance and may reduce or eliminate Nab formation, allowing potential redosing of gene therapy.

Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response.

The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.

Updated data from a phase 1 study of PCXR201, formerly known as Flexion’s FX201, were presented at the 2023 ASCGT Annual Meeting.

Vitalgen’s gene therapy was generally well-tolerated with no serious adverse events reported.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed the need to reevaluate the goal posts used to determine whether gene therapies for eye indications come to market.

The cofounder and chief scientific officer of Xcell Biosciences discussed the company’s efforts to introduce new technologies for improving cell therapy development and manufacturing.

The assistant professor of stem cell transplantation at MD Anderson Cancer Center discussed positive data in ccRCC that he believes should excite the solid tumor field as a whole.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed new real world data regarding Spark Therapeutics’ Luxturna that was presented at ARVO’s 2023 conference.

The director of clinical research at Sierra Eye Associates discussed 4D Molecular Therapeutics’ plans for 4D-150 in both wet AMD and diabetic macular edema.