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Displaying drugs 12751 - 12775 of 14392 in total

SIG-001 is a cultured human retinal pigment epithelial cell line (ARPE-19) genetically modified with a non-viral vector to express B domain deleted human factor VIII protein, encapsulated within two-layer modified alginate spheres.

Investigational

CUR-1916

Investigational

RST-001

RST-001 is a non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein.

Investigational

GS030-DP

GS030-DP is an optogenetic gene therapy being investigated for the treatment of retinitis pigmentosa. It targets retinal ganglion cells that encodes a light-sensitive channelrhodopsin, ChrimsonR-tdTomato (ChR-tdT), delivered by an AAV2.7m8 vector. It is a component of GS030, an investigational optogenetic treatment, in combination with a medical device GS030-MD.[A259816, L46701]

Investigational

WVE-N531

Investigational

OCU-100

Investigational

SPL23-2

SPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.

Investigational

QR-1123

QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to treat autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin (RHO) gene.

Investigational