Lysosomal Disorders

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.

Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.

Review top news and interview highlights from the week ending October 14, 2022.

The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.

The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII.

Review top news and interview highlights from the week ending October 7, 2022.

The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.

Rocket Pharma presented updated data from adult and pediatric patients at the 2022 HFSA meeting.

Newborn Cell Therapies Group at Monash University has received a $50,000 grant from Lions Cord Blood foundation.

Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending September 23, 2022.

The professor of genetics at Federal University of Rio Grande do Sul discussed updated data from the CAMPSIITE trial.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Preliminary data from an ongoing clinical trial suggest that AVR-RD-04 has been well-tolerated.

In a mouse model of MPS II, IDS.ApoEII effected completely normalized brain pathology and behavior.

Review top news and interview highlights from the week ending September 16, 2022.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending September 9, 2022.