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Find Clinical Drug Pipeline Developments & Deals by Regenxbio
The net proceeds will be used for the development of ABBV-RGX-314, being developed as a novel, one-time treatment for wet AMD that includes the NAV® AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment.
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin, which is being evaluate for the treatment of duchenne muscular dystrophy.
RGX-121 is an investigational, one-time gene therapy designed to deliver the IDS gene that encodes the iduronate-2-sulfatase enzyme (I2S) using the NAV® AAV9 vector. It is under development for the treatment of Hunter syndrome.
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin. It is being developed for the treatment of duchenne muscular dystrophy in patients with ages 4 to 11 years old.
ABBV-RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit VEGF. It is believed to inhibit the VEGF pathway. It is under phase 2 clinical development or the treatment of wet AMD.
RGX-202 is a Cell and Gene therapy drug infused intravenously for the treatment of Duchenne Muscular Dystrophy, transferring the micro-dystrophin gene.
Company will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases i.e. Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio syndrome.
RGX-202 delivers a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal domain found in naturally occurring dystrophin, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic patients.
RGX-111 is designed to use the AAV9 vector to deliver the alpha-l-iduronidase gene to the CNS, which provides a permanent source of secreted IDUA beyond the blood-brain barrier, potentially preventing the progression of cognitive deficits in MPS I patients.
RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase gene which encodes the iduronate-2-sulfatase (I2S) enzyme to the CNS, allowing for long-term cross correction of cells throughout the CNS.
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