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[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lonza Expands Partnership with Rocket Pharmaceuticals for Childhood Disorder Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Receives Authorization from FDA To Begin Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 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Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the ESID 2020 Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Announces Dosing of First Subject in Phase 1\/2 Clinical Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene 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Patient in the BRILLIANCE Clinical Trial of EDIT-101 for LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Announces Oral Presentations at the Upcoming 2022 IRSF Rett Syndrome Scientific Symposium and the ASCEND National Summit","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene 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Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Announces Positive Results from Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-Associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not 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Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Genethon"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics to Present Positive Interim Encore Data from the Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1) at the 46th Annual Macula Society Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Atsena 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Pharma"},{"orgOrder":0,"company":"Wacker Chemie AG","sponsor":"Caeregen Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Caeregen Therapeutics and Wacker Biotech Announce Collaboration Agreement to Produce the Regenerative Medicine Clinical Candidate CTR-107 (Noregen\u2122) for Retinal Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Wacker Chemie AG"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Announces Updates on Global Development Strategy of INZ-701 for the Treatment of ENPP1 Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ 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            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.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 18, 2024

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            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.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Citizens JMP

            Deal Size: $40.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement April 18, 2024

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            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).

            Lead Product(s): OPGx-LCA5

            Therapeutic Area: Rare Diseases and Disorders Product Name: OPGx-LCA5

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2024

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            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).

            Lead Product(s): ATSN-101,Prednisone,Triamcinolone

            Therapeutic Area: Rare Diseases and Disorders Product Name: SAR439483

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 16, 2024

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            HMB-001 is a novel bispecific antibody designed to be the first prophylactic treatment for Glanzmann Thrombasthenia (GT) and other debilitating bleeding disorders

            Lead Product(s): HMB-001

            Therapeutic Area: Rare Diseases and Disorders Product Name: HMB-001

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 11, 2023

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            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

            Therapeutic Area: Rare Diseases and Disorders Product Name: ATSN-101

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 04, 2023

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            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.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 30, 2023

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            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.

            Lead Product(s): Elsunersen

            Therapeutic Area: Rare Diseases and Disorders Product Name: PRAX-222

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 16, 2023

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            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.

            Lead Product(s): ATSN-101

            Therapeutic Area: Rare Diseases and Disorders Product Name: ATSN-101

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 14, 2023

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            HG004 is an investigational gene replacement therapy product. It uses the recombinant AAV9 vector to deliver a functional human RPE65 gene to the retina and to restore, treat, and prevent blindness of children and adults with RPE65-Inherited Retinal Diseases.

            Lead Product(s): HG004

            Therapeutic Area: Rare Diseases and Disorders Product Name: HG004

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 01, 2023

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