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Re-Dosing With Gene Therapies in Rare Diseases

June 15th 2022

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

Tim Miller, PhD, on Supporting Gene Therapy Development

June 2nd 2022

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

Donald Kohn, MD, on the Trajectory of Gene Therapy

June 1st 2022

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

Lentiviral Cell Therapy Normalizes Hemoglobin in Pyruvate Kinase Deficiency

May 28th 2022

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

Developing Safer Viral Vectors for Gene Therapy

May 26th 2022

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

Amy Pooler, PhD, on Improving Gene Delivery to the CNS

May 26th 2022

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

Jason Fontenot, PhD, on Reducing the Need for Immunosuppression in Renal Transplantation

May 25th 2022

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

Gene Therapy May Eliminate Need for ERT in Late-Onset Pompe Disease

May 25th 2022

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

Andras Heczey, MD, on Antitumor Activity of CAR NKTs in Solid Tumors

May 24th 2022

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

Cell Therapy Modulates Tumor Microenvironment in Glioblastoma

May 23rd 2022

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