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Inapplicable"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Elixirgen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Hemab Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Haemnet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Hemab Therapeutics \/ Haemnet","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Haemnet"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Radiprodil","moa":"NMDA receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"USH2A","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"BBP-681","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"BridgeBio Pharma \/ Venthera","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Venthera"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Nationwide Children\u2019s Hospital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Financing","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Miglustat","moa":"Glucosylceramide synthase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Taysha Gene Therapies

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Taysha Gene Therapies

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                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Offering of Common Stock and Pre-Funded Warrants

                            Details : The proceeds will be used to fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy, in Rett syndrome.

                            Product Name : TSHA-102

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

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                            02

                            Taysha Gene Therapies

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Taysha Gene Therapies

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Pricing of Stock and Pre-Funded Warrants

                            Details : The company will fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome.

                            Product Name : TSHA-102

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

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                            03

                            Theranexus

                            France arrow
                            Discovery Europe
                            Not Confirmed

                            Theranexus

                            France arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Theranexus & BBDF Reveal Real-World Data Supporting Batten-1 for Batten

                            Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The MOA of this substance blocks the accumulation of glycosphingolipids and neuroinflammation.

                            Product Name : Batten-1

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 13, 2025

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus gets RMAT Designation for OPGx-LCA5 Gene Therapy in Vision Restoration

                            Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                            Product Name : OPGx-LCA5

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 06, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Alcyone Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Alcyone Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Nationwide Children’s Hospital

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Alcyone Gains Strategic Funding for Pediatric Neuro Therapies

                            Details : The financing aims to advance the clinical developed of company's ealry-stage product CLN-301, which is being evaluated for the treatment of CLN3 Batten disease.

                            Product Name : CLN-301

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            April 24, 2025

                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Nationwide Children’s Hospital

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            06

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus Shares One-Month Data from Pediatric Gene Therapy OPGx-LCA5 Trial

                            Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                            Product Name : OPGx-LCA5

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 08, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Benitec Biopharma

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Benitec Biopharma

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Benitec Biopharma Inc. Announces Pricing of $30 Million Common Stock Offering

                            Details : The net proceeds from the proposed financing will support the clinical development of BB-301, which is being evaluated for Oculopharyngeal Muscular Dystrophy.

                            Product Name : BB-301

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            March 25, 2025

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

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                            08

                            Entrada Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Entrada Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : ENTR-601-45

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Entrada Gets UK Approval for Phase 1/2 Study of ENTR-601-45 in DMD

                            Details : ENTR-601-45, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer. It is being evaluated for the treatment of duchenne muscular dystrophy.

                            Product Name : ENTR-601-45

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 24, 2025

                            Lead Product(s) : ENTR-601-45

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Perceptive Advisors

                            Deal Size : $21.4 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Opus Genetics Prices Public Offering & Private Placement Over $20M

                            Details : Opus intends to use the net proceeds to fund clinical development of its lead gene therapy programs, OPGx-LCA5 for the treatment of leber congenital amaurosis.

                            Product Name : OPGx-LCA5

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            March 21, 2025

                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Perceptive Advisors

                            Deal Size : $21.4 million

                            Deal Type : Public Offering

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                            10

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ABO-101

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Arch Ventures

                            Deal Size : $73.9 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Arbor Raises $73.9M Series C for Gene Editing Therapies

                            Details : The proceeds will support clinical development of the company's lead therapeutic candidate, ABO-101, in primary hyperoxaluria type 1.

                            Product Name : ABO-101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            March 18, 2025

                            Lead Product(s) : ABO-101

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Arch Ventures

                            Deal Size : $73.9 million

                            Deal Type : Series C Financing

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