
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : nL-GARS1-001
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : University of Texas Health Science Center at Houston
Deal Size : Inapplicable
Deal Type : Inapplicable
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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Lead Product(s) : nL-ATN1-001
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Dell Children's Medical Center of Central Texas
Deal Size : Inapplicable
Deal Type : Inapplicable
Personalized Antisense Oligonucleotide for ATN1 Gene Mutation Patient
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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Lead Product(s) : nL-ASXL3-001
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : University of North Carolina, Chapel Hill
Deal Size : Inapplicable
Deal Type : Inapplicable
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
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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Lead Product(s) : nL-MAPK8-001
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Columbia University
Deal Size : Inapplicable
Deal Type : Inapplicable
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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Lead Product(s) : nL-PRPH2-001
Therapeutic Area : Ophthalmology
Study Phase : Phase I/ Phase II
Sponsor : University of California, San Diego
Deal Size : Inapplicable
Deal Type : Inapplicable
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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Lead Product(s) : nL-CHCHD-001
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Columbia University
Deal Size : Inapplicable
Deal Type : Inapplicable
Personalized Antisense Oligonucleotide for Participants with CHCHD10 ALS
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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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
Personalized Antisense Oligonucleotide Therapy for A Single Participant with TARDBP ALS
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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Lead Product(s) : nL-ATN1-002
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Hawaii Pacific Neuroscience
Deal Size : Inapplicable
Deal Type : Inapplicable
Personalized Antisense Oligonucleotide for A Single Participant with ATN1 Gene Mutation
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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Lead Product(s) : nL-CHCHD-001
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Columbia University
Deal Size : Inapplicable
Deal Type : Inapplicable
Personalized Antisense Oligonucleotide for a Single Participant with CHCHD10 ALS
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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Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Discovery Platform
Sponsor : GondolaBio
Deal Size : Undisclosed
Deal Type : Collaboration
GondolaBio, n-Lorem Partner for ASO Therapies for Genetic Disease Advancement
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
