Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.
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Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.
Review top news and interview highlights from the week ending September 30, 2022.
Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.
SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.
A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.
The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.
Xue Zhong Liu, MD, PhD, and research partner Zheng-Yi Chen, PhD, will investigate gene editing and CRISPR/Cas9 systems to potentially treat Usher syndrome.
The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.
Review top news and interview highlights from the week ending September 23, 2022.
The company previously presented positive preclinical data at the 2021 ASGCT meeting.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The approval follows unanimous support of eli-cel's benefits in CALD from an FDA AdComm meeting and an approval for beti-cel in beta-thalassemia.
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.
Postmortem analyses revealed cell survival and GDNF production in transplanted areas.
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The chief medical officer of Passage Bio discussed research needs in FTD.
Review top news and interview highlights from the week ending September 2, 2022.
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.
The chief medical officer of Passage Bio discussed the phase 1/2 upliFT-D study of PBFT02.
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