Pharmaceutical Technology13 天
Risk adjusted net present value: What is the current valuation of MeiraGTx’s AAV-AQP1?
AAV-AQP1 is a gene therapy commercialized by MeiraGTx, with a leading Phase II program in Sicca Syndrome (Sjogren).
Pharmaceutical Technology12 天
AAV-AQP1 by MeiraGTx for Sicca Syndrome (Sjogren): Likelihood of Approval
AAV-AQP1 is under clinical development by MeiraGTx and currently in Phase II for Sicca Syndrome (Sjogren). According to GlobalData, Phase II drugs for Sicca Syndrome (Sjogren) does not have sufficient ...
The EMBO Journal12 天
Gene therapy for epilepsy targeting neuropeptide Y and its Y2 receptor to dentate gyrus ...
Co-expressing NPY and its inhibitory receptor Y2 in excitatory hippocampal cells allows inhibitory autoregulation of ...
BridgeBio's BBP-812 Canavan Disease Gene Therapy Receives FDA RMAT Designation
BridgeBio Pharma Inc. (BBIO) announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy ...
GlobalData on MSN5 天
Pipeline Moves: Novo Nordisk’s liraglutide scores in Phase III paediatric trial
The Clinical Trials Arena team investigates assets in infectious diseases, metabolic disorders, oncology and ophthalmology.
Medpage Today on MSN8 天
Gene Therapy Shows Promise in Rare Eye Disease That Causes Early Blindness
A subretinal gene therapy was well tolerated 1 year after treatment, with no drug-related serious adverse events, in a small ...
BridgeBio Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for ...
Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which ...