REGENXBIO
NEWS
Gene therapies developed by Janssen, Atsena, REGENXBIO and AbbVie showed promise against eye diseases this weekend at the American Academy of Ophthalmology 2022 meeting.
It’s been a busy week for research on rare diseases, with several clinical trials from AnaptysBio, Regenxbio, Sangamo and more posting results from ongoing activities.
CRD’s CRD-TMH-001 is expected to upregulate an alternate form of the dystrophin protein with CRISPR technology and stabilize or reverse the symptoms associated with DMD.
Ayala announced positive data for its desmoid tumor treatment, while Vertex and Dyne announced the FDA had lifted the hold on their clinical studies.
BioSpace looks at several companies developing new gene therapies in the wake of the FDA recommending two of bluebird bio’s lentiviral vector gene therapies.
Six months after the one-time treatment, 47% of patients in Cohort 1 demonstrated a promising two-step or greater improvement in their diabetic retinopathy.
REGENXBIO presented results from its ongoing Phase I/II trial of RGX-111, an investigational one-time gene therapy for people with Mucopolysaccharidosis Type I (MPS I).
The FDA has granted Cambridge-based bluebird Bio a Priority Review of its Biologics License Application for betibeglogene autotemcel (beti-cel).
Collaboration is key to faster breakthroughs. The NIH, FDA, and 15 private organizations have announced they are joining forces for the sake of the 30 million Americans suffering from a rare disease.
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