Neurology

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.

Review some of our most-viewed coverage of advancements in cell therapies, including study data and clinical trial updates.

Review some of our most-viewed coverage of advancements in gene therapies, including study data and clinical trial updates.

Review some of our most-viewed interviews with clinicians, researchers, and biotech companies about expanding applications of cell and gene therapies.

The trial will incorporate findings from CYGNET, a natural history study designed to assess the disease progression of AMN.

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

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.

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.