CSBio CSBio

X
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Morgan","pharmaFlowCategory":"D","amount":"$86.9 million","upfrontCash":"Undisclosed","newsHeadline":"Gene Editing Biotech Metagenomi Plans Modest $87M IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sangamo Therapeutics Announces Updated Phase 1\/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit and Differentiated Safety Profile","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$100.0 million","newsHeadline":"BridgeBio Pharma and Kyowa Kirin Announce Partnership with an Upfront Payment of $100 Million for an Exclusive License on Infigratinib in Skeletal Dysplasias in Japan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Titan Partners Group LLC","pharmaFlowCategory":"D","amount":"$15.0 million","upfrontCash":"Undisclosed","newsHeadline":"Immix Biopharma Announces Closing of $15 Million Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$93.7 million","upfrontCash":"Undisclosed","newsHeadline":"Metagenomi Announces Pricing of Initial Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Synlogic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Synlogic Announces Decision to Discontinue Synpheny-3 Study and Provides Corporate Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Biogen Received European Commission Approval for SKYCLARYS\u00ae (omaveloxolone), the First Therapy to Treat Friedreich's Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich's Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$125.0 million","upfrontCash":"Undisclosed","newsHeadline":"Prime Medicine Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","amount":"$150.0 million","upfrontCash":"Undisclosed","newsHeadline":"Larimar Therapeutics Announces Pricing of Underwritten Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KalVista Pharmaceuticals Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor\/Tezacaftor\/Deutivacaftor, Next-in-Class Triple Combination Treatment for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Update on FDA Review Timeline of KRESLADI\u2122 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"KalVista Pharmaceuticals Reports Phase 3 KONFIDENT Trial Meets All Endpoints for Sebetralstat as First Oral On-demand Therapy for Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Mabxience","sponsor":"Biosidus S.A.","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"mAbxience and Biosidus Sign CDMO Agreement for the Manufacture of Agalsidase Beta for the Treatment of Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SPAIN","productType":"Large molecule","productStatus":"Biosimilar","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"ReCode Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Petrovax","sponsor":"ISU ABXIS","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fabagal\u00ae for Fabry Disease Management is Now Available in Russia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"RUSSIA","productType":"Large molecule","productStatus":"Biosimilar","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$93.7 million","upfrontCash":"Undisclosed","newsHeadline":"Metagenomi Announces Closing of Initial Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Larimar Therapeutics Announces Proposed Underwritten Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"$160.1 million","upfrontCash":"Undisclosed","newsHeadline":"KalVista Pharmaceuticals Announces Pricing of a $160.1 Million Public Offering of Common Stock and Pre-Funded Warrants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Files for Additional Indication of Evrysdi for Pre-Symptomatic Spinal Muscular Atrophy and Additional Dosage for Infants up to 2 Months of Age","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$161.0 million","upfrontCash":"Undisclosed","newsHeadline":"Prime Medicine Announces Closing of Upsized Public Offering and Full Exercise of the Underwriters' Option to Purchase Additional Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Syncona","pharmaFlowCategory":"D","amount":"$28.3 million","upfrontCash":"$28.3 million","newsHeadline":"Acquisition of Freeline by Syncona Becomes Effective","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Peptide","graph2":"Phase I"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Launches ORLADEYO\u00ae (berotralstat) in Italy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces Completion of Patient Enrollment for Opvika\u00ae Phase I\/II Clinical Trial in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Mabwell","sponsor":"Disc medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to 9MW3011","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Harmony Biosciences Receives U.S. Food and Drug Administration Orphan Drug Designation for Pitolisant in Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Study of Inhaled mRNA-Based Genetic Medicine, RCT2100, for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"jCyte","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"jCyte, Inc. Announces Positive Pre-Phase 3 FDA Type B Meeting and Outlines Plans to Start Pivotal Trial of jCell\u00ae for Retinitis Pigmentosa in the Second Half of 2024","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ocugen, Inc. Announces Dosing Completion of Subjects with Stargardt in Cohort 1 of Phase 1\/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arcturus Therapeutics Receives Orphan Medicinal Product Designation from the European Commission (EC), for ARCT-032, for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"Undisclosed","newsHeadline":"Denali Therapeutics Announces $500 million Private Placement Equity Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"$200.0 million","upfrontCash":"Undisclosed","newsHeadline":"Tyra Biosciences, Inc. Announces $200 Million Private Placement Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR\u2122 Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"SPAIN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","amount":"$1,865.0 million","upfrontCash":"$165.0 million","newsHeadline":"Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Adage Capital Partners LP","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Avidity Biosciences Announces Oversubscribed $400 Million Private Placement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"}]

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            Details:

            Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 25, 2023

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            Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

            Lead Product(s): Ganaxolone

            Therapeutic Area: Genetic Disease Product Name: Ztalmy

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Marinus Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 31, 2023

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            SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: SAR-439774

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 04, 2023

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            Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).

            Lead Product(s): Eliglustat

            Therapeutic Area: Genetic Disease Product Name: Cerdelga

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2023

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            Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 24, 2023

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            Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 23, 2023

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            BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 25, 2023

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            BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 30, 2022

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            Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 10, 2022

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            SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: SAR-439774

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 10, 2022

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