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                            Lead Product(s) : Zaltenibart

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                            Study Phase : Phase II

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                            Omeros Updates Ongoing Zaltenibart Phase 3 PNH Clinical Trial Program

                            Details : OMS906 (zaltenibart) is a MASP-3 inhibitor, antibody candidate, which is currently being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

                            Product Name : OMS906

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            March 21, 2025

                            Lead Product(s) : Zaltenibart

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

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                            Lead Product(s) : Soquelitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Corvus Starts Phase 2 Soquelitinib Trial in Autoimmune Lymphoproliferative Syndrome

                            Details : CPI-818 (soquelitinib) is an investigational small molecule drug given orally designed to selectively inhibit ITK, which is being evaluated for treatment of autoimmune lymphoproliferative syndrome.

                            Product Name : CPI-818

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : Soquelitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Jaguar Health

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                            Jaguar Health

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                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Jaguar Doses First Patient in IIT Evaluating Crofelemer for Microvillus Inclusion Disease

                            Details : Mytesi (crofelemer) is a plant-based drug extracted & purified from the red bark sap. It is being developed for MVID in pediatric patients.

                            Product Name : Mytesi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 22, 2025

                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Arctic Therapeutics

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                            Not Confirmed

                            Arctic Therapeutics

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                            Lead Product(s) : Grapiprant

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : EIC fund

                            Deal Size : $27.7 million

                            Deal Type : Series A Financing

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                            Arctic Therapeutics Announces €26.5M Oversubscribed Series A Financing

                            Details : The financing aims to fund the development of AT-001 an oral treatment designed to prevent the aggregation of harmful amyloid proteins in the brain. It is being evaluated for treating HCCAA.

                            Product Name : AT-001

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : Grapiprant

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : EIC fund

                            Deal Size : $27.7 million

                            Deal Type : Series A Financing

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                            05

                            Bio-Thera Solutions

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                            Not Confirmed

                            Bio-Thera Solutions

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                            Lead Product(s) : BAT4406F

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Bio-Thera Solutions Publishes Phase I Results for BAT4406F in Neuromyelitis Optica

                            Details : BAT4406F is an ADCC-enhanced fully humanized anti-CD20 monoclonal antibody in Chinese patients with neuromyelitis optica spectrum disorders (NMOSD).

                            Product Name : BAT4406F

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : BAT4406F

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Caliway Biopharmaceuticals

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                            Caliway Biopharmaceuticals

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                            Lead Product(s) : CBL-514

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Caliway Announces EMA Orphan Drug Designation for CBL-514 in Dercum's

                            Details : CBL-514 is a potentially first-in-class small-molecule drug, which can induce adipocytes apoptosis and lipolysis. It is being evaluated for Dercum's disease.

                            Product Name : CBL-514

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 11, 2024

                            Lead Product(s) : CBL-514

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Rocket Pharmaceuticals

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                            Rocket Pharmaceuticals

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                            Lead Product(s) : RP-L102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Morgan Stanley

                            Deal Size : $165.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Rocket Pharmaceuticals Announces Pricing of Upsized Public Offering of Common Stock

                            Details : The net proceeds will be used to fund the clinical development of RP-L102, which is being evaluated for the treatment of Fanconi Anemia.

                            Product Name : RP-L102

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            December 11, 2024

                            Lead Product(s) : RP-L102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Morgan Stanley

                            Deal Size : $165.0 million

                            Deal Type : Public Offering

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                            08

                            Rocket Pharmaceuticals

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                            Rocket Pharmaceuticals

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                            Lead Product(s) : RP-L102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Morgan Stanley

                            Deal Size : $150.0 million

                            Deal Type : Public Offering

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                            Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock

                            Details : The net proceeds will be used to fund the clinical development of RP-L102, which is being evaluated for the treatment of Fanconi Anemia.

                            Product Name : RP-L102

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            December 10, 2024

                            Lead Product(s) : RP-L102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Morgan Stanley

                            Deal Size : $150.0 million

                            Deal Type : Public Offering

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                            09

                            Genethon

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                            Genethon

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                            Lead Product(s) : GNT-0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Genethon/Hansa Start Phase 2 Trial Of Imlifidase Pre-Treatment for Crigler-Najjar

                            Details : GNT-0003 is being developed for crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV vector carrying a normal copy of UGT1A1.

                            Product Name : GNT-0003

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            December 03, 2024

                            Lead Product(s) : GNT-0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            NovelMed Therapeutics

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                            NovelMed Therapeutics

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                            Lead Product(s) : Ruxoprubart

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ruxoprubart Receives FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria

                            Details : NM8074 (ruxoprubart) is an alternative pathway (AP) blocker anti-Bb antibody. It is being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

                            Product Name : NM8074

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            December 02, 2024

                            Lead Product(s) : Ruxoprubart

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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