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Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ GordonMD Global Investments","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GordonMD Global Investments"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ Not 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Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Vaccine","year":"2024","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase 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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Praxis Precision Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Elsunersen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Praxis Precision Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Praxis Precision Medicines \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Praxis Precision Medicines \/ Not 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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Hemab Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Citizens 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Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"AJ201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avenue Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Not Applicable"}]

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                          01

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Radiprodil is a potent negative allosteric modulator of the NMDA receptor subtype 2B. It is being evaluated for the treatment of Tuberous Sclerosis Complex and Focal Cortical Dysplasia Type II.

                          Brand Name : RGH-896

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 08, 2024

                          Lead Product(s) : Radiprodil

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : OPGx-LCA5 is an 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.

                          Brand Name : OPGx-LCA5

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 20, 2024

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : ABBV-CLS-7262 (fosigotifator) is an eIF2B activator, a factor that is essential for protein synthesis and a key regulator of the ISR. It is being investigated for vanishing white matter disease.

                          Brand Name : ABBV-CLS-7262

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 07, 2024

                          Lead Product(s) : Fosigotifator

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : 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).

                          Brand Name : AJ201

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          May 16, 2024

                          Lead Product(s) : AJ201

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : BB-301 is a modified AAV9 capsid expressing a bifunctional construct for co-expressing codon-optimized PABPN1 & siRNAs against mutant PABPN1, evaluated for treating oculopharyngeal muscular dystrophy.

                          Brand Name : BB-301

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          April 18, 2024

                          Lead Product(s) : BB-301

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : The company intends to use proceeds for BB-301's clinical development, evaluated in early-stage trials for oculopharyngeal muscular dystrophy treatment.

                          Brand Name : BB-301

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 18, 2024

                          Lead Product(s) : BB-301

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Citizens JMP

                          Deal Size : $40.0 million

                          Deal Type : Private Placement

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                          07

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : OPGx-LCA5 uses an AAV8 vector to deliver a functional LCA5 gene to the retina for patients with Leber congenital amaurosis from LCA5 mutations.

                          Brand Name : OPGx-LCA5

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 26, 2024

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : 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).

                          Brand Name : SAR439483

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          January 16, 2024

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

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

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

                          Brand Name : HMB-001

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          December 11, 2023

                          Lead Product(s) : HMB-001

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : 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.

                          Brand Name : ATSN-101

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 04, 2023

                          Lead Product(s) : ATSN-101,Prednisone,Triamcinalone Acetonide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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