Apertura Gene Therapy Launches Open Aperture, a Material Transfer Agreement Program to Facilitate Non-Commercial Access to TfR1 CapX for Academic Research…

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...
Seeking Alpha

REGENXBIO Highlights AAV Pipeline with Interim Results from Retinal and Duchenne Programs at its Virtual Investor Day on July 11, 2023

-Interim results from Phase II AAVIATE and ALTITUDE trials of dose level 3 cohorts with short-course prophylactic steroid eye drops resulted in zero cases of intraocular inflammation -Initial safety ...
Seeking Alpha

REGENXBIO to Host Virtual Investor Day Highlighting AAV Pipeline Programs with Upcoming Interim Trial Updates

-Focus on programs for Wet AMD, Diabetic Retinopathy, and Duchenne Muscular Dystrophy ROCKVILLE, Md., June 27, 2023 /PRNewswire/ -- REGENXBIO Inc. (RGNX) today announced that it will host a virtual ...
Business Wire

Forge Biologics Selected as an AAV Manufacturing Partner for California Institute for Regenerative Medicine to Help Accelerate Gene Therapy Programs

COLUMBUS, Ohio--(BUSINESS WIRE)--Forge Biologics (Forge), a leading manufacturer of genetic medicines, announced today that it has joined the California Institute for Regenerative Medicine’s (CIRM) ...
Business Insider

Axovant Announces Global Licensing Agreement for AXO-AAV-OPMD Program for Treatment of Oculopharyngeal Muscular Dystrophy and Broader Platform Collaboration with Benitec Biopharma

License grants Axovant worldwide rights to AXO-AAV-OPMD utilizing proprietary Silence-and-Replace technology, which suppresses mutant protein production while restoring expression of functional ...
Nasdaq

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline with New Development Programs for Duchenne Muscular Dystrophy and Myotonic Dystrophy

Call may be accessed by dialing (833) 659-8620 (U.S.) or (409) 767-9247 (international) and using conference ID# 8879216 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic ...
Nasdaq

Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership

NEW YORK and BASEL, Switzerland, Sept. 15, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, ...
Business Insider

Amicus Therapeutics Acquires Gene Therapy Portfolio of Ten Clinical and Pre-Clinical Stage AAV Programs in Neurologic Lysosomal Storage Disorders

License with Nationwide Children’s Hospital (NCH) through the Acquisition of Celenex (NCH Spinout) Establishes Amicus as Leading Gene Therapy Company in Neurologic Lysosomal Storage Disorders (LSD) ...
GEN

AAV Vectors: Select Permutations, Favorable Destinations

Adeno-associated virus (AAV) vectors may be the best option to deliver genetic cargo to cells in the body. But they are far from perfect. They require large doses, hold small amounts of cargo, and can ...