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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
AJ201 is an oral Nrf1/Nrf2 stimulator which is under phase 1/2 clinical development for the treatment of patients with Spinal and Bulbar Muscular Atrophy (Kennedy's Disease).
BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized PABPN1 & two siRNAs against mutant PABPN1. It is being evaluated for the treatment of oculopharyngeal muscular dystrophy.
The Company intends to use the net proceeds from the financing to fund the clinical development and related commercialization of BB-301, which is being evaluated in early-stage clinical trial studies for treating oculopharyngeal muscular dystrophy.
OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene (LCA5).
SAR439483 (ATSN-101) is a novel GUCY2D modulator which is currently being evaluated in phase 1/2 clinical development for the treatment of GUCY2D-associated leber congenital amaurosis (LCA1).
HMB-001 is a novel bispecific antibody designed to be the first prophylactic treatment for Glanzmann Thrombasthenia (GT) and other debilitating bleeding disorders
ATSN-101 is a LCA1 gene Transference Cell and Gene therapy drug candidate, which is currently being evaluated for the treatment of patients with GUCY2D-mutated Leber Congenital Amaurosis.
Lead Product(s):
ATSN-101,Prednisone,Triamcinalone Acetonide
BB-301 is a Cell and Gene therapy drug designed for Oculopharyngeal Muscular Dystrophy Related Dysphagia, utilizing the PABPN1 gene as a replacer and administered via intramuscular injection.
PRAX-222 (elsunersen) is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression. It is under development for treatment of SCN2A gain of function developmental epilepsies.
ATSN-101, gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.