With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.
Neurology
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No treatment-related serious adverse events were reported.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
REGENXBIO expects to provide updated interim data from the trial in the first half of next year.
AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Full data readouts have not been shared.
Updated data were presented at the AES annual meeting in Nashville.
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
SRP-9001's PDUFA date is set for May 23, 2023.
Review top news and interview highlights from the week ending November 25, 2022.
The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.
The gene therapy was approved in the European Union earlier this year.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
BrainStorm received indication from the FDA that it is able to request a Type A meeting to discuss the content of the refusal to file letter.
The FDA action follows a slew of recent cell and gene therapy updates in the field of amyotrophic lateral sclerosis.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The trial was launched through a partnership between Cure Rare Disease and UMass Chan Medical School.
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.
Review top news and interview highlights from the week ending November 4, 2022.
