Lysosomal Disorders

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.

iECURE is planning to submit an IND for GTP-506 in mid-2023.

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

Immusoft plans to begin the phase 1 clinical trial of ISP-001 before the end of 2022.

Review top news and interview highlights from the week ending September 2, 2022.

The in vivo genome-editing therapy is the first gene therapy to be evaluated in a clinical trial for preventing HAE attacks.

RGX-121 was well-tolerated across all 3 of the dose cohorts with no treatment-related serious adverse events reported.

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

Updated data from the STAAR clinical trial were presented at the SSIEM Annual Meeting.

Review top news and interview highlights from the week ending August 26, 2022.

iECURE and the University of Pennsylvania Gene Therapy Program have collaborated to develop the GTP-506 program.

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