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    [{"orgOrder":0,"company":"Paradigm Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Pentosan Polysulfate Sodium","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Paradigm Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Paradigm Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Paradigm Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Percheron Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Percheron Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Percheron Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Percheron Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Trofinetide","moa":"IGF-1 receptor||peptides: neurologic indications","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamifeport","moa":"SLC40A1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Percheron Therapeutics

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                            ISPE Annual Meeting
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                            Percheron Therapeutics

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                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

                            Details : ATL1102 is a Oligonucleotide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Muscular Dystrophy, Duchenne.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            July 10, 2023

                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Neuren Pharmaceuticals

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                            ISPE Annual Meeting
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                            Neuren Pharmaceuticals

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                            Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

                            Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Prader-Willi Syndrome.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 30, 2023

                            Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Clinuvel Pharmaceuticals

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                            Clinuvel Pharmaceuticals

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                            ISPE Annual Meeting
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                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

                            Details : Afamelanotide is a Peptide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Porphyria, Variegate.

                            Product Name : Undisclosed

                            Product Type : Peptide, Unconjugated

                            Upfront Cash : Inapplicable

                            May 11, 2023

                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
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                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Clinuvel Trial Results Show Drug Reduces DNA Damage

                            Details : Scenesse® (afamelanotide) belongs to the family of melanocortins, hormones, which activates human pigmentation, reduce oxidative damage, inflammation and swelling, and optimise the response of skin cells to UV-induced damage. It is being investigated fo...

                            Product Name : Scenesse

                            Product Type : Peptide, Unconjugated

                            Upfront Cash : Inapplicable

                            January 16, 2023

                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Paradigm Biopharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            Paradigm Biopharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed
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                            Lead Product(s) : Pentosan Polysulfate Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            MPS Clinical Program Update and Paradigm to Present at the International Conference on Lysosomal Diseases

                            Details : Three of the four subjects included in Paradigm's open-label phase 2 trial of pentosan polysulfate sodium (PPS) in MPS-I have completed the 48-week study, with a 6-month treatment extension available.

                            Product Name : Zilosul

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 12, 2022

                            Lead Product(s) : Pentosan Polysulfate Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
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                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
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                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V

                            Details : Afamelanotide is a Peptide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Xeroderma Pigmentosum.

                            Product Name : Undisclosed

                            Product Type : Peptide, Unconjugated

                            Upfront Cash : Inapplicable

                            May 11, 2022

                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Antisense Therapeutics

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            Antisense Therapeutics

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed
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                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Antisense Therapeutics to Present Poster at World’s Largest NMD Conference

                            Details : ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4. Antisense inhibition of VLA-4 expression has demonstrated activity in animal models of inflammatory disease. ATL1102 has also shown to be very effective in reducing inflammatory brain lesion...

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 09, 2022

                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            Clinuvel Pharmaceuticals

                            Australia arrow
                            ISPE Annual Meeting
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                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)

                            Details : Afamelanotide is a Peptide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Xeroderma Pigmentosum.

                            Product Name : Undisclosed

                            Product Type : Peptide, Unconjugated

                            Upfront Cash : Inapplicable

                            December 16, 2021

                            Lead Product(s) : Afamelanotide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            CSL

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            CSL

                            Australia arrow
                            ISPE Annual Meeting
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                            Lead Product(s) : Vamifeport

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            First Patient Enrolled in Phase-IIa Study of Vamifeport in Patients with Sickle Cell Disease

                            Details : VIT-27639 (Vamifeport), is the first oral ferroportin inhibitor investigated for treatment of diseases with ineffective production of red blood cells and iron overload such as sickle cell disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            December 10, 2021

                            Lead Product(s) : Vamifeport

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Antisense Therapeutics

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            Antisense Therapeutics

                            Australia arrow
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Antisense Therapeutics Announces a Poster Presentation on the Muscular Dystrophy Association Virtual Conferenc...

                            Details : The poster presentation includes the recently reported data from the Phase II clinical trial of the Company’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) where ATL1102 met the primary endpoint.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            June 11, 2020

                            Lead Product(s) : ATL1102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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