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Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Be Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Recombinant Human Heparan N-sulfatase","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"GC Biopharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"GC Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Recombinant Human Heparan N-sulfatase","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"5","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"EV\u014cQ Nano","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"EVQ-218","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"EV\u014cQ Nano","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"EV\u014cQ Nano \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"EV\u014cQ Nano \/ Not Applicable"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"SAT-3247","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Satellos Bioscience \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Satellos Bioscience \/ Not Applicable"}]

    Find FDA Investigational New Drug (IND) Submissions for Genetic Disease

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                            01

                            Genespire

                            Italy arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Genespire

                            Italy arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
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                            Lead Product(s) : GENE202

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Sofinnova Partners

                            Deal Size : $52.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Genespire Raises €46.6M For Pediatric Gene Therapy Clinical Trials

                            Details : The financing will enable the development of GENE202, the company’s lead candidate, up to a Phase I/II clinical trial patients affected by methylmalonic acidemia.

                            Brand Name : GENE202

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 24, 2024

                            Lead Product(s) : GENE202

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Sofinnova Partners

                            Deal Size : $52.0 million

                            Deal Type : Series B Financing

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                            02

                            Papillon Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Papillon Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : PPL-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Papillon Receives Orphan Drug Designation for PPL-001 in Friedreich's Ataxia

                            Details : PPL-001 is an experimental gene-corrected CD34+ HSPC therapy. Currently, it is being investigated for the treatment of Friedreich's ataxia.

                            Brand Name : PPL-001

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            September 17, 2024

                            Lead Product(s) : PPL-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            OmniaBio

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            OmniaBio

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SMT-M01

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Somite Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Somite and OmniaBio Partner To Advance Somite's Cell Therapy Program

                            Details : The collaboration aims to manufacture and produce SMT-M01, Somite's flagship program, which is being evaluated in early-stage clinical studies for treating patients with Duchenne muscular dystrophy.

                            Brand Name : SMT-M01

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 09, 2024

                            Lead Product(s) : SMT-M01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Somite Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            04

                            GC Biopharma

                            South Korea arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            GC Biopharma

                            South Korea arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Lead Product(s) : alpha-galactosidase A

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Hanmi Pharmaceutical

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            GC Biopharma and Hanmi Receive IND Clearance for Phase 1/2 Trial in the US

                            Details : GC1134A/HM15421 is a long-acting alpha-galactosidase that can be administered subcutaneously once a month in patients with fabry disease, also to improve patient's convenience.

                            Brand Name : GC1134A

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 03, 2024

                            Lead Product(s) : alpha-galactosidase A

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Hanmi Pharmaceutical

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Satellos Announces Filing to Start Phase 1 Trial with SAT-3247

                            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.

                            Brand Name : SAT-3247

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 19, 2024

                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Satellos Receives FDA Rare Pediatric Designation for SAT-3247 in Duchenne Dystrophy

                            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.

                            Brand Name : SAT-3247

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 08, 2024

                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Intellia Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Intellia Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : NTLA-3001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Intellia Gets Authorization for NTLA-3001 Phase 1/2 for Alpha-1 Antitrypsin Deficiency

                            Details : NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD)

                            Brand Name : NTLA-3001

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            July 30, 2024

                            Lead Product(s) : NTLA-3001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Satellos Submits Filing for Phase 1 Trial with SAT-3247

                            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.

                            Brand Name : SAT-3247

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 11, 2024

                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            JCR Pharmaceuticals

                            Japan arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            JCR Pharmaceuticals

                            Japan arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : JR-446

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Medipal Holdings Corporation

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            MEDIPAL and JCR Complete CTN for Phase I/II Study of JR-446 in Mucopolysaccharidosis IIIB

                            Details : JR-446, is a blood-brain barrier-penetrating α-N-acetylglucosaminidase, developed using JCR’s proprietary J-Brain Cargo technology, is under investigation for mucopolysaccharidosis type IIIB.

                            Brand Name : JR-446

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            July 02, 2024

                            Lead Product(s) : JR-446

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Medipal Holdings Corporation

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Satellos Bioscience

                            Canada arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Satellos Presents Data Showing Muscle Repair from SAT-3247 in Canine Model of DMD

                            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.

                            Brand Name : SAT-3247

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 02, 2024

                            Lead Product(s) : SAT-3247

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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