Drugs in Dev.
Genetic Disease
Phase I/ Phase II
Spain 
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Lead Product(s) : Ciclopirox
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ATL-001 Oral Solution Safety & Effect On Photosensitivity-Related Skin Lesions In CEP
Details : Ciclopirox is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Porphyria, Erythropoietic.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
June 17, 2025
Lead Product(s) : Ciclopirox
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : SB-007 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Stargardt Disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 24, 2025
Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
SpliceBio Doses First Patient in Phase 1/2 ASTRA Trial of SB-007 for Stargardt
Details : SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene. It is being evaluated for the treatment of stargardt disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
March 17, 2025
Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ARTHEx Biotech Receives FDA Rare Pediatric Designation for ATX-01
Details : ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
November 07, 2024
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ARTHEx Doses First Patient in ArthemiR™ Trial for Myotonic Dystrophy
Details : ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
October 18, 2024
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of ATX-01 in Participants With DM1
Details : ATX-01 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Myotonic Dystrophy.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
March 08, 2024
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Alpha-1 Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Alpha-1 Proteinase Inhibitor is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
January 25, 2021
Lead Product(s) : Alpha-1 Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : AXO-AAV-GM1
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Sio Gene Therapies
Deal Size : Undisclosed
Deal Type : Partnership
Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership
Details : Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercial...
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
September 15, 2020
Lead Product(s) : AXO-AAV-GM1
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Sio Gene Therapies
Deal Size : Undisclosed
Deal Type : Partnership

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Lead Product(s) : SCD-Omegatex
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Robin E. Miller
Deal Size : Inapplicable
Deal Type : Inapplicable
Omega-3 Fatty Acids in Sickle Cell Disease
Details : SCD-Omegatex is a Other Small Molecule drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 27, 2016
Lead Product(s) : SCD-Omegatex
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Robin E. Miller
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Alpha1-proteinase Inhibitor,Alpha-1 Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Grifols International
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Prolastin-C (Alpha1-proteinase Inhibitor) is a Protein drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.
Product Name : Prolastin-C
Product Type : Undisclosed
Upfront Cash : Inapplicable
August 17, 2016
Lead Product(s) : Alpha1-proteinase Inhibitor,Alpha-1 Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Grifols International
Deal Size : Inapplicable
Deal Type : Inapplicable
