Neurology

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.

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 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.

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

The study will be carried out in Paris and is expected to begin before the end of the year.

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

All patients with NMOSD who received CT103A showed improved Expanded Disability Status Scale scores.

Merit Cudkowicz, MD, discussed corrected data from a phase 3 trial of NurOwn that BrainStorm believes supports a BLA submission.