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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.
OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5gene to retinal photoreceptors. Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001.
EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), by deleting the IVS26 CEP290 mutant allele.
LX1004 is an adeno-associated virus based gene therapy for the treatment of CLN2 Batten disease. In Phase 1/2 clinical trial, LX1004 demonstrated a favorable safety profile and showed statistically significant reduction in rate of decline compared to natural history studies.
Data demonstrated that subretinal delivery ATSN-101 (SAR439483) was well tolerated and patients treated with highest dose clinically meaningful improvements in vision, as measured by full-field stimulus testing and multi-luminance mobility testing.
The data presented are preliminary results from an open-label, dose-finding study of VT30 (BBP-681) topical gel in patients with cutaneous VM/LM/VLM lesions associated with mutations that result in dysregulated PI3K activation.
EDIT-101 program for the treatment for Leber Congenital Amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, demonstrating a favorable immunogenicity profile.
TSHA 102 is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, a truncated version of MECP2, and miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel, packaged in self-complementary AAV9.
EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder.
CAN106 is a novel, long-acting recombinant human monoclonal antibody that binds to and neutralizes C5, a key component of the complement system for treatment of paroxysmal nocturnal hemoglobinuria.
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