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Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure cerebrotendinous xanthomatosis in patients.
Through the divestment, Tenpoint has acquired SparingVision's ex vivo GIRK technology. SparingVision will remain focused on in vivo genomic medicines, with SPVN20, a GIRK-based gene therapy candidate.
Primera is advancing pioneering mtDNA gene editing therapies developed in the Mayo Clinic lab to precisely fix a patient’s mutated mtDNA and potentially achieve a cure for the disease.
The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN® gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal storage diseases.
The publication of a peer-reviewed article for FXS01 in EMBO Molecular Medicine showing that adeno-associated viral (AAV) vector-delivered diacylglycerol kinase (DGKk) achieves long-term rescue of fragile X syndrome (FXS) in a mouse model of disease.
First approval of ATA-100, was received from UK MHRA its gene therapy for the treatment of the fukutin-related protein limb-girdle muscular dystrophy Type 2I/R9.
VAR002 is a CRX-expressing AAV vector designed as a relevant therapeutic approach for gene supplementation and a promising drug candidate for mutation-agnostic gene therapy. VAR002 prevents photoreceptor degeneration without adverse effects linked with CRX overexpression.
Lysogene will be responsible for the preclinical and clinical development, manufacturing, regulatory activities, and commercialization of the drug candidate, globally. which includes FXS01, a gene therapy candidate for the treatment of Fragile X syndrome.
Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs.
The funding round supports company's program focusing on identifying new vectors. Using the company's VECTrans® platform, nanoparticles of mRNA, as well as ASOs and siRNAs, can be vectorized in order to treat both diseases with a genetic component and cancers.