Drugs in Dev.
Genetic Disease
Phase I/ Phase II
South Korea 
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Lead Product(s) : HM15421
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Hanmi Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : HM15421 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Fabry Disease.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
March 05, 2025
Lead Product(s) : HM15421
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Hanmi Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : EN001
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
Details : EN001 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Muscular Dystrophy, Duchenne.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
March 25, 2024
Lead Product(s) : EN001
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) : Donaperminogene Seltoplasmid
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea
Details : The trial aims to evaluate Engensis’ safety and tolerability by administering the treatment to the leg of twelve CMT1A patients in Korea.
Product Name : Engensis
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
July 27, 2020
Lead Product(s) : Donaperminogene Seltoplasmid
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) : GC1111
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : GC1111 is a Enzyme drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Mucopolysaccharidosis II.
Product Name : Undisclosed
Product Type : Enzyme
Upfront Cash : Inapplicable
February 23, 2011
Lead Product(s) : GC1111
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
