American Society for Gene & Cell Therapy (ASGCT)

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.

Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.

The cofounder, president, and chief executive officer of Forge Biologics discussed the company’s approach to gene therapy manufacturing.

The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.

RP-L301 also improved hemolysis and eliminated the need for red blood cell transfusions for up to 1year after therapy.

The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.

The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.

The chief scientific officer of Sangamo Therapeutics discussed the first-in-human STEADFAST trial.

All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.

The director of the Liver Tumor Program at Texas Children’s Hospital discussed a phase 1 study of CAR NK T-cells in neuroblastoma.

The cell therapy was tolerated although there was a SUSAR of GGT elevations.

The senior investigator from NINDS discussed findings investigating serious AEs across different trials.

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.

Review top news and interview highlights from the week ending May 20, 2022.

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.

The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.

Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.

Safety and efficacy data suggest this to be a feasible and clinically meaningful treatment for a patient population with high disease burden.

Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.

Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.