[{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Hawaii Pacific Neuroscience","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-ATN1-002","moa":"ATN1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Columbia University","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-CHCHD-001","moa":"CHCHD10","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Columbia University","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Columbia University"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Columbia University","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-CHCHD-001","moa":"CHCHD10","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Columbia University","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Columbia University"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Methodist Hospital Research Institute | Columbia University","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-TARD-001","moa":"TDP43","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ The Methodist Hospital Research Institute | Columbia University","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ The Methodist Hospital Research Institute | Columbia University"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Hongene Biotech","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"3","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","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":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"n-Lorem Foundation \/ The Conrad Prebys Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ The Conrad Prebys Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Parexel Biotech","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":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Argonaut Manufacturing Services","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":"Undisclosed","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"1","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Cytiva","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":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"St. Jude Children","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ n-Lorem Foundation"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"GondolaBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ GondolaBio","highestDevelopmentStatusID":"3","companyTruncated":"n-Lorem Foundation \/ GondolaBio"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"University of California, San Diego","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-PRPH2-001","moa":"PRPH2","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ University of California, San Diego","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ University of California, San Diego"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"University of North Carolina, Chapel Hill","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-ASXL3-001","moa":"ASXL3 gene","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ University of North Carolina, Chapel Hill","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ University of North Carolina, Chapel Hill"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Columbia University","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-MAPK8-001","moa":"Undisclosed","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Columbia University","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Columbia University"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Dell Children's Medical Center of Central Texas","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-ATN1-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"n-Lorem Foundation \/ Dell Children's Medical Center of Central Texas","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Dell Children's Medical Center of Central Texas"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"University of Texas Health Science Center at Houston","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-GARS1-001","moa":"GARS1 gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ University of Texas Health Science Center at Houston","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ University of Texas Health Science Center at Houston"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-GARS1-001 is an oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Charcot-Marie-Tooth Disease.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          November 10, 2025

                          Lead Product(s) : nL-GARS1-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : University of Texas Health Science Center at Houston

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-ATN1-001 is an oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Myoclonic Epilepsies, Progressive.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          October 28, 2025

                          Lead Product(s) : nL-ATN1-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Dell Children's Medical Center of Central Texas

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-ASXL3-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Bainbridge-Ropers Syndrome.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 29, 2025

                          Lead Product(s) : nL-ASXL3-001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : University of North Carolina, Chapel Hill

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-MAPK8-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Neurodevelopmental Disorders.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 29, 2025

                          Lead Product(s) : nL-MAPK8-001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Columbia University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-PRPH2-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Retinal Dystrophies.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 16, 2025

                          Lead Product(s) : nL-PRPH2-001

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : University of California, San Diego

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-CHCHD-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Amyotrophic Lateral Sclerosis.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 31, 2025

                          Lead Product(s) : nL-CHCHD-001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Columbia University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Lead Product(s) : nL-TARD-001

                          Therapeutic Area : Neurology

                          Study Phase : Phase I/ Phase II

                          Sponsor : The Methodist Hospital Research Institute | Columbia University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : NL-TARD-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Amyotrophic Lateral Sclerosis.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 31, 2025

                          Lead Product(s) : nL-TARD-001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : The Methodist Hospital Research Institute | Columbia University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-ATN1-002 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Myoclonic Epilepsies, Progressive.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 24, 2025

                          Lead Product(s) : nL-ATN1-002

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Hawaii Pacific Neuroscience

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : NL-CHCHD-001 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Amyotrophic Lateral Sclerosis.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          May 18, 2025

                          Lead Product(s) : nL-CHCHD-001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Columbia University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Global ChemShow
                          Not Confirmed
                          Global ChemShow
                          Not Confirmed

                          Details : The collaboration aims to discover novel antisense oligonucleotide (ASO) medicines for patients who are not currently served by available treatment options.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          December 17, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery Platform

                          Sponsor : GondolaBio

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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