Review top news and interview highlights from the week ending February 3, 2023.
Neurology
Latest News
Advertisement
Advertisement
Mehra and Subramanian discussed preclinical research with the investigational gene therapy SLS-004.
Cartesian Therapeutics’ Descartes-08 will be administered as 6 weekly infusions and does not require preconditioning chemotherapy.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.
Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
Review top news and interview highlights from the week ending January 27, 2023.
No dose-limiting toxicities were observed at 28 days of follow-up.
The DGX-A01 study met its primary safety and efficacy endpoints in its highest dose cohort of IDCT.
Patients continued to show statistically significant upper limb performance improvements.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
REGENXBIO has also initiated recruitment for AFFINITY BEYOND, an observational study assessing the prevalence of AAV8 antibodies in male patients with DMD.
Review top news and interview highlights from the week ending January 20, 2023.
The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.
The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
It was previously announced that CT103A received FDA clearance of its investigational new drug application for the treatment of relapsed/refractory (r/r) multiple myeloma.
Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.
Seelos Therapeutics’ SLS-004 showed the ability to downregulate α-synuclein, and CENTOGENE’s ROPAD study will be extended.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
No serious adverse events related to the gene therapy have been reported.
Promising results were previously reported from a proof-of-concept study involving 8 patients.
Initial data from the trials of HMI-103 and HMI-203 are expected in 2023.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
