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[{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Jaguar Gene Therapy"},{"orgOrder":0,"company":"SpliSense","sponsor":"Biotel Limited","pharmaFlowCategory":"D","amount":"$28.5 million","upfrontCash":"Undisclosed","newsHeadline":"SpliSense Secures $28.5 Million in Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"SpliSense"}]

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            Details:

            JAG201 is being evaluated in preclinical studies for the treatment of SHANK-3 mutated autism spectrum disorder & Phelan-McDermid syndrome. It aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.

            Lead Product(s): JAG201

            Therapeutic Area: Genetic Disease Product Name: JAG201

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 31, 2024

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            Proceeds will be used to advance the Company’s pipeline, including its lead Anti Sense Oligonucleotide product, SPL84-23. SPL84-23 to treat the 3849+10kb C->T CFTR mutation, that has showed ability to completely restore CFTR channel function in patient-derived cell cultures.

            Lead Product(s): SPL84-23

            Therapeutic Area: Genetic Disease Product Name: SPL84-23

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Biotel Limited

            Deal Size: $28.5 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing May 13, 2021

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