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Venture"},{"orgOrder":0,"company":"Everads Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Everads Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Everads Therapy \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Everads Therapy \/ Undisclosed"},{"orgOrder":0,"company":"Zylka Lab","sponsor":"University of North Carolina","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Zylka Lab","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zylka Lab \/ University of North Carolina","highestDevelopmentStatusID":"4","companyTruncated":"Zylka Lab \/ University of North Carolina"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Leaps by Bayer","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Aerosol","sponsorNew":"ReCode Therapeutics \/ Leaps by Bayer","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Leaps by Bayer"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"Injection","sponsorNew":"Affinia Therapeutics \/ EcoR1 Capital","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ EcoR1 Capital"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Avilar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"5Am Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ensoma","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Injection","sponsorNew":"Ensoma \/ 5Am Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Ensoma \/ 5Am Ventures"},{"orgOrder":0,"company":"Resilience","sponsor":"Mayo Clinic","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Resilience \/ Mayo Clinic","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ Mayo Clinic"},{"orgOrder":0,"company":"KBio","sponsor":"Leaf Expression Systems","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"KBio \/ Leaf Expression Systems","highestDevelopmentStatusID":"4","companyTruncated":"KBio \/ Leaf Expression Systems"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Affinia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Avilar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":1,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":1,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":1,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":1,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"KBio \/ ZabBio","highestDevelopmentStatusID":"4","companyTruncated":"KBio \/ ZabBio"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Generation Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Undisclosed"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Generation Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Undisclosed"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"UT Southwestern Medical Center \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"UT Southwestern Medical Center \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Aeterna Zentaris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aeterna Zentaris \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Undisclosed"},{"orgOrder":0,"company":"Latus Bio","sponsor":"8VC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Latus Bio","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Latus Bio \/ 8VC","highestDevelopmentStatusID":"4","companyTruncated":"Latus Bio \/ 8VC"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Apertura Gene Therapy","sponsor":"RSRT","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Apertura Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Apertura Gene Therapy \/ RSRT","highestDevelopmentStatusID":"4","companyTruncated":"Apertura Gene Therapy \/ RSRT"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing 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Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ FOXG1 Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ FOXG1 Research Foundation"},{"orgOrder":0,"company":"Code Bio","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Code Bio","amount2":2,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":2,"dosageForm":"Undisclosed","sponsorNew":"Code Bio \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aeterna Zentaris \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Undisclosed"},{"orgOrder":0,"company":"Strm.Bio","sponsor":"National Center for Advancing Translational Sciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Strm.Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Strm.Bio \/ National Center for Advancing Translational Sciences","highestDevelopmentStatusID":"4","companyTruncated":"Strm.Bio \/ National Center for Advancing Translational Sciences"},{"orgOrder":0,"company":"Vyant Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Vyant Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vyant Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Vyant Bio \/ Undisclosed"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ AskBio"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Protein","year":"2025","type":"Licensing Agreement","leadProduct":"JR-479","moa":"HEXA","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"4","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"}]

Find Drugs for Rare Diseases and Disorders in Preclinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : OTOF-GT,Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Preclinical

                          Sponsor : Sensorion

                          Deal Size : Undisclosed

                          Deal Type : Agreement

                          Details : Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

                          Product Name : OTOF-GT

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          October 27, 2020

                          Lead Product(s) : OTOF-GT,Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Sensorion

                          Deal Size : Undisclosed

                          Deal Type : Agreement

                          Axplora CB

                          02

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The partnership to develop and manufacture NAR-001, NanoRetinal's proprietary nanoparticle technology designed to restore and/or preserve vision in patients with Inherited Retinal Diseases (IRD).

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          September 09, 2025

                          Lead Product(s) : NAR-001

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : NanoRetinal

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          03

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Medipal will have exclusive worldwide rights (excluding Japan) to develop, manufacture, and commercialize JR-479, with sub-licensing rights.

                          Product Name : JR-479

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          September 02, 2025

                          Lead Product(s) : JR-479

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Medipal Holdings Corporation

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          04

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Proceeds will be used to advance Kamari's lead program KM023, a first-in-class oral TRPV3 inhibitor, for three rare genetic skin diseases, Olmsted syndrome, severe keratoderma and ichthyosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          June 03, 2025

                          Lead Product(s) : KM023

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : BRM Group

                          Deal Size : $23.0 million

                          Deal Type : Series A Financing

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                          05

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The collaboration aims to advance the innovative genetic medicine, which is being evaluated in preclinical clinical trial studies for the treatment of Rett Syndrome.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          April 30, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : RSRT

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          06

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : BA-102 is an investigational agent that aims to target and regulate nitric oxide (NO) levels in the brain and being developed for the treatment of PMS, a syndrome associated with ASD.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 21, 2025

                          Lead Product(s) : BA-102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : RVB-003 is based on ResVita Bio's continuous protein therapy platform, which utilizes genetically engineered bacteria applied topically, being developed for Netherton Syndrome.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          April 03, 2025

                          Lead Product(s) : RVB-003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The funding will be used to support the company’s lead program, AXV-101, an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with Bardet-Biedl Syndrome.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          October 30, 2024

                          Lead Product(s) : AXV-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : A Race Against Blindness

                          Deal Size : $1.0 million

                          Deal Type : Funding

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                          09

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage studies for the treatment of LAMA2-congenital muscular dystrophy.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          October 25, 2024

                          Lead Product(s) : MDL-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The collaboration aims to advanced the clinical development of AXV-101, which is being evaluated in the early-stage clinical trial studies for the treatment of Bardet-Biedl syndrome.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          October 24, 2024

                          Lead Product(s) : AXV-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Axovia Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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