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    Find Drugs for Genetic Disease in Phase I Clinical Development

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                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            Novasep and Lysogene Announce their New Collaboration for Development and Production of GM1 Gangliosidosis Gen...

                            Details : With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene's lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3.

                            Product Name : LYS-GM101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 26, 2020

                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            02

                            Minovia Therapeutics

                            Israel arrow
                            German Wound Congress
                            Not Confirmed

                            Minovia Therapeutics

                            Israel arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : MNV-201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Minovia Gets FDA Nod for Phase II Trial of MNV-201 in Pearson Syndrome

                            Details : MNV-201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. It is being evaluated for the treatment of pearson syndrome.

                            Product Name : MNV-201

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 03, 2025

                            Lead Product(s) : MNV-201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Keros Therapeutics

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                            German Wound Congress
                            Not Confirmed

                            Keros Therapeutics

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : KER-065

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Keros Announces Initial Topline Results from the Phase 1 Trial of KER-065

                            Details : KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA/IIB, being investigated for duchenne muscular dystrophy.

                            Product Name : KER-065

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            March 31, 2025

                            Lead Product(s) : KER-065

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Regulus Therapeutics

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                            German Wound Congress
                            Not Confirmed

                            Regulus Therapeutics

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : Farabursen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Regulus Completes Phase 1b Trial Of Farabursen for ADPKD Treatment

                            Details : RGLS8429 (farabursen) is an miR-17 inhibitor, antisense oligonucleotide, which is currently being evaluated for the treatment of autosomal dominant polycystic kidney disease.

                            Product Name : RGLS8429

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 27, 2025

                            Lead Product(s) : Farabursen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Kaerus Bioscience

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                            German Wound Congress
                            Not Confirmed

                            Kaerus Bioscience

                            United Kingdom arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Kaerus' KER-0193 Shows Proof of Mechanism in Phase 1 for Fragile X Syndrome

                            Details : KER-0193, a novel BK channel modulator, small molecule drug candidate, which is being evaluated for the treatment of Fragile X syndrome.

                            Product Name : KER-0193

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 11, 2025

                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            ENCell

                            South Korea arrow
                            German Wound Congress
                            Not Confirmed

                            ENCell

                            South Korea arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : EN001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            ENCell’s EN001 Gains FDA Orphan Drug Status for Charcot-Marie-Tooth

                            Details : EN001 is a mesenchymal stem cell therapy, it targets damaged nerves, promotes the secretion of regenerative factors, and facilitates remyelination. Currently being investigated for CMT.

                            Product Name : EN001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 06, 2025

                            Lead Product(s) : EN001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Actio Biosciences

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Actio Biosciences

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : ABS-0871

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Saniona

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Actio Biosciences Doses First Participant in ABS-0871 Phase 1 Trial

                            Details : ABS-0871 is a TRPV4 antagonist. small molecule drug candidate, which is currently being evaluated for the treatment of charcot marie tooth disease 2C.

                            Product Name : ABS-0871

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 03, 2025

                            Lead Product(s) : ABS-0871

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Saniona

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Entrada Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Entrada Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : ENTR-601-44

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44

                            Details : ENTR-601-44, a proprietary EEV-conjugated phosphorodiamidate morpholino oligomer, which is an investigational therapy for the potential treatment of people living with Duchenne muscular dystrophy.

                            Product Name : ENTR-601-44

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 24, 2025

                            Lead Product(s) : ENTR-601-44

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Satellos Bioscience

                            Canada arrow
                            German Wound Congress
                            Not Confirmed

                            Satellos Bioscience

                            Canada arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Satellos Completes Phase 1 Enrollment for SAT-3247 in Healthy Volunteers

                            Details : SAT-3247 is a once-daily, oral small molecule, AAK1 inhibitor that targets the root cause of muscle loss in degenerative diseases. It is currently in investigation for duchenne muscular dystrophy.

                            Product Name : SAT-3247

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 10, 2025

                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Aro Biotherapeutics

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                            German Wound Congress
                            Not Confirmed

                            Aro Biotherapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : ABX1100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Aro Biotherapeutics Gets FDA IND Clearance for ABX1100 in Pompe Disease

                            Details : ABX1100 targets CD71, a specific receptor, to transport siRNA therapy into muscle tissues, effectively inhibiting the GYS1 enzyme, being investgated in late-onset Pompe disease (LOPD) patients.

                            Product Name : ABX1100

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ABX1100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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