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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"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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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                            02

                            Benitec Biopharma

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Benitec Biopharma

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • 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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                            03

                            Entrada Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Entrada Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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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                            04

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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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                            05

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • 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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                            06

                            Elixirgen Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Elixirgen Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Elixirgen Gets FDA Orphan Drug Designation for Telomere Disorders

                            Details : EXG-34217 is a dose of autologous CD34+ hematopoietic stem cells (HSCs) that is being investigated for the treatment of telomere biology disorders.

                            Product Name : EXG-34217

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 20, 2025

                            Lead Product(s) : EXG-34217

                            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

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : OPGx-LCA5

                            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 Genetics Announces Updates on OPGx-LCA5 Clinical Program

                            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

                            February 18, 2025

                            Lead Product(s) : OPGx-LCA5

                            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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                            08

                            Elixirgen Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Elixirgen Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Elixirgen's EXG-34217 Gets FDA RMAT for Treating Telomere Biology Disorders

                            Details : EXG-34217 is a dose of autologous CD34+ hematopoietic stem cells (HSCs) that is being investigated for the treatment of telomere biology disorders.

                            Product Name : EXG-34217

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 13, 2025

                            Lead Product(s) : EXG-34217

                            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

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ABO-101

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Arbor Gets FDA Orphan & Rare Pediatric Designations for ABO-101 in PH1 Treatment

                            Details : ABO-101, a novel gene editing therapeutic, is currently being evaluated to treat patients suffering from primary hyperoxaluria type 1.

                            Product Name : ABO-101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ABO-101

                            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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                            10

                            Kanglin Biotechnology

                            China arrow
                            German Wound Congress
                            Not Confirmed

                            Kanglin Biotechnology

                            China arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : KL003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : $20.0 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Kanglin Secures $20M Series A to Advance Gene Therapy Developments

                            Details : The proceeds will be used to support the clinical development for the company’s lead asset, KL003, a lentiviral vector gene therapy, for the treatment of beta thalassemia and sickle cell disease.

                            Product Name : KL003

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            November 25, 2024

                            Lead Product(s) : KL003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : $20.0 million

                            Deal Type : Series A Financing

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