ABO-202 is an AAV
gene therapy that has shown promising preclinical efficacy in the INCL animal model of disease by extending survival and improving muscle function when administered early in the disease course," noted Steven J.
is attractive as a tool for moving engineered pieces of DNA (or at least short ones, less than ~4000 bases) into target cells for several reasons.
Vimana addresses this hurdle with its patented, industry-first, blockchain-backed airspace ecosystem for managing AAV
Headquartered in Rockville, Maryland, Vigene offers AAV
, lentivirus and adenovirus based products and custom services.
Our autologous cell and gene therapy programs continue to demonstrate remarkable effects in clinical trials of patients with life-threatening diseases with no treatment options, and we are pleased to collaborate with Brammer Bio for commercial AAV
process development, scale up and assay validation for clinical programs and internal capabilities," said Timothy Miller, president and chief executive officer, Abeona.
The percentage of the local component of the products of the AAV
is 45%, and the company seeks to increase it to 60% within eight years.
Mortality is high in AAV
patients with renal involvement.
Classic risk factors for thrombo-embolic event such as immobilization, trauma, malignancy, major surgery, positive family history nor thrombophilia do not show a significant prevalence in AAV
manufacturing has traditionally required unique custom batches of helper plasmids.
HE Mattar Al Tayer, Director General and Chairman of the Board of Executive Directors of RTA said: "The trial run of the first AAV
is in implementation of the directives of HH Sheikh Mohammed bin Rashid Al Maktoum, Vice-President and Prime Minister of the UAE and Ruler of Dubai, to transform Dubai into the smartest city in the world.
The team injected two AAVs
(specifically an AAV
8 serotype discovered in the Wilson lab that has an affinity for liver cells), one expressing Cas9 and the other expressing a guide RNA and a donor DNA, into newborn mice with OTC deficiency.
In addition to the CASQ2-CPVT program, the acquisition includes rights to develop and commercialize AAV
gene therapy products for several additional inherited arrhythmias.