In February, the biotech reported improvements in 10 of 11 children who received DB-OTO. The AAV gene therapy, which ...
Preclinical data demonstrates that Shape’s engineered AAV5 variant, SHP-DB1, efficiently targets the NHP brain after intravenous injection and ...
Oral Presentation at ESGCT Highlights Potential of JCR’s Proprietary JUST-AAV Platform to Facilitate More Efficient Delivery ...
MedPage Today on MSN
Gene Therapy Restores Hearing for Rare Cause of Congenital Deafness
A novel gene therapy enabled children with congenital deafness due to otoferlin (OTOF) gene variants to hear for the first ...
Zacks Investment Research on MSN
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study. This gene therapy is being evaluated for profound genetic hearing loss due ...
MeiraGTx Holdings Receives RMAT Designation from FDA for AAV-GAD Gene Therapy in Parkinson's Disease
MeiraGTx Holdings plc announced that the FDA has granted its investigational gene therapy AAV-GAD the Regenerative Medicine Advanced Therapy (RMAT) designation for treating Parkinson's disease ...
The AAV gene therapy market size is calculated at USD 3.85 billion in 2025 and is expected to reach around USD 78.56 billion by 2034, growing at a CAGR of 40.1% for the forecasted period.Ottawa, Oct.
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...
A significant advance in treatment of patients with immunity to AAVs previously ineligible for clinical trials and existing ...
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