MavriX Bio Receives FDA Fast Track Designation for MVX-220 for Treatment of Angelman Syndrome
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MavriX Bio MVX-220 Update
MavriX Bio’s gene therapy MVX-220 receives FDA Fast Track designation, with its Phase 1/2 ASCEND-AS trial now listed and a community webinar set for October 9.
Ionis Receives Breakthrough Therapy Designation for ION582 in Angelman Syndrome
Ionis Pharmaceuticals’ ION582 has received FDA Breakthrough Therapy Designation for Angelman syndrome, a status reserved for drugs showing substantial improvement over existing treatments.
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx announces the Phase 3 ASPIRE study for apazunersen (GTX-102) is fully enrolled with 129 participants, reaching this milestone in just seven months.
Oak Hill Bio Announces Publication of Rugonersen Phase 1 Study Results in Nature Medicine
Oak Hill Bio’s Phase 1 TANGELO study results, published in Nature Medicine, show improvements in brain activity and development for children with Angelman syndrome on rugonersen.
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Ultragenyx’s GTX-102 (apazunersen) has received FDA Breakthrough Therapy Designation for Angelman syndrome, marking a key milestone in its path toward potential approval.
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
Ionis Pharmaceuticals has dosed the first participant in its global Phase 3 REVEAL trial, evaluating the safety and efficacy of ION582 as a potential treatment for Angelman syndrome.
MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome
The FDA has cleared MavriX Bio’s IND application for MVX-220, an investigational AAV gene therapy for Angelman syndrome.
Oak Hill Bio to continue the development of rugonersen, an investigational ASO previously owned by Roche.
Oak Hill Bio has entered into an exclusive global licensing agreement with Roche to acquire and advance rugonersen, a Phase 3-ready investigational ASO treatment for Angelman syndrome.
IONIS REVEAL Phase 3 Clinical Trial Update
The next chapter for ION582 is here: Ionis’ Phase 3 REVEAL trial is now listed, marking an important step toward a potential treatment for Angelman syndrome. Join FAST, ASF, and Ionis to learn what comes next.