[{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"CPRIT","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Funding","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ CPRIT","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ CPRIT"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Private Placement","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Public Offering","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leucocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Mereo BioPharma Group \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Inapplicable"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leucocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Mereo BioPharma Group \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"GSK-3-beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Panakes Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Series B Financing","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Enterprise Therapeutics \/ Panakes Partners","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Panakes Partners"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"30 Technology","sponsor":"ConvaTec","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Divestment","leadProduct":"RESP301","moa":"Membrane fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"30 Technology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution for Inhalation","sponsorNew":"30 Technology \/ ConvaTec","highestDevelopmentStatusID":"8","companyTruncated":"30 Technology \/ ConvaTec"},{"orgOrder":0,"company":"Healx","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Healx \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Inapplicable"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mereo BioPharma Group \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Inapplicable"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mereo BioPharma Group \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Inapplicable"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody","year":"2024","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mereo BioPharma Group \/ Jefferies","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Jefferies"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody","year":"2020","type":"Collaboration","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mereo BioPharma Group \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ReNeuron \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"ReNeuron \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"SAR422459","moa":"ABCR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Oxford Biomedica \/ Sanofi"},{"orgOrder":0,"company":"Cell and Gene Therapy Catapult","sponsor":"INmune Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Partnership","leadProduct":"Umbilical Cord Derived Mesenchymal Stromal Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cell and Gene Therapy Catapult","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cell and Gene Therapy Catapult \/ INmune Bio","highestDevelopmentStatusID":"8","companyTruncated":"Cell and Gene Therapy Catapult \/ INmune Bio"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"}]

Find Drugs for Genetic Disease in Phase II Clinical Development in UNITED KINGDOM

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                          Top Deals by Deal Size (USD bn)

                          01

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          May 12, 2025

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : The collaboration will focus on scaling production of CORDStrom, a proprietary pooled, allogeneic, human umbilical cord-derived mesenchymal Stromal/Stem cell (hucMSCs) platform, for RDEB.

                          Product Name : CORDStrom

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 14, 2025

                          Lead Product(s) : Umbilical Cord Derived Mesenchymal Stromal Cell

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : INmune Bio

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          03

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          March 10, 2025

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : HLX-1502 is being evaluated through a mid-stage clinical trial study for the treatment of patients suffering from neurofibromatosis Type 1.

                          Product Name : HLX-1502

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 24, 2025

                          Lead Product(s) : HLX-1502

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          February 12, 2025

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          February 10, 2025

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : eRapa is a proprietary oral capsule formulation of rapamycin (mTOR inhibitor), also known as sirolimus. It is being developed for treating Familial Adenomatous Polyposis.

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          November 07, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 15, 2024

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : ETD001, is a first-in-class potential, targets the epithelial sodium channel. It is being evaluated in mid-stage clinical trial studies for the treatment of Cystic Fibrosis.

                          Product Name : ETD001

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 26, 2024

                          Lead Product(s) : ETD001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : The proceeds will be used to fund company's upcoming Phase 3 registrational study of eRapa, a proprietary oral tablet formulation of rapamycin, in Familial Adenomatous Polyposis.

                          Product Name : eRapa

                          Product Type : Antibiotic

                          Upfront Cash : Undisclosed

                          September 16, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : CPRIT

                          Deal Size : $17.0 million

                          Deal Type : Funding

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