Polpharma European CDMO Partner & API Manufacturer since 1951 Polpharma European CDMO Partner & API Manufacturer since 1951

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molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Wave Life Sciences"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"PepGen"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I\/R9 (LGMD2I\/R9), Cleared to Proceed By FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atamyo Therapeutics"},{"orgOrder":0,"company":"Porton Advanced Solutions","sponsor":"BRL Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Porton Advanced and BRL Medicine Build Strategic Partnership to Accelerate Commercialization of Gene and Cell Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Porton Advanced Solutions"},{"orgOrder":0,"company":"Sarcomatrix","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","amount":"$1.9 million","upfrontCash":"Undisclosed","newsHeadline":"Sarcomatrix Therapeutics Corp.\u2019s Exclusive Research Partner Strykagen Awarded SBIR Phase 2 Grant from the National Institutes of Health (NIH) to Advance Small Molecules Promoting Muscle Regeneration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Sarcomatrix"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Announces FDA Clearance of Investigational New Drug Application for KB408 for the Treatment of Type 1 Alpha-1 Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calliditas Therapeutics Granted Orphan Drug Designation by the FDA for the Treatment of Alport Syndrome with Setanaxib","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"SWEDEN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Calliditas Therapeutics AB"},{"orgOrder":0,"company":"Baudax Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Baudax Bio Announces Orphan Drug Designation Granted by U.S. FDA for TI-168 for the Treatment of Hemophilia A with Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Baudax Bio"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Medicines Agency Committee for Orphan Medicinal Products Provides Positive Opinion On Calliditas' Application for Setanaxib in Alport Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"SWEDEN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Calliditas Therapeutics AB"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Inc. Announces FDA has Lifted the Clinical Hold on its Investigational New Drug Application for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"PepGen"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Apple Tree Partners","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ascidian Therapeutics Raises $40 Million in Series A Extension Financing from Apple Tree Partners and Appoints Michael Ehlers as Interim CEO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Ascidian Therapeutics"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Satellos Discloses Drug Target and Provides Development Update on Duchenne Muscular Dystrophy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Satellos Bioscience"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"OrbiMed","pharmaFlowCategory":"D","amount":"$42.5 million","upfrontCash":"Undisclosed","newsHeadline":"ENYO Pharma Announces a \u20ac39 Million Series C Financing and FDA Clearance to Advance Vonafexor in a Phase 2 Clinical Trial for Patients With Alport Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Enyo Pharma"},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Juvena Therapeutics Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Juvena Therapeutics"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Vanda Pharmaceuticals"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ascidian Therapeutics Announces First-Ever IND for an RNA Exon Editor as FDA Approves Trial Plan and Fast Tracks ACDN-01 in Stargardt Disease and Other ABCA4 Retinopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Ascidian Therapeutics"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Jaguar Gene Therapy"},{"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":"Prime Medicine"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. 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Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"ARTHEx Biotech"},{"orgOrder":0,"company":"Fibrocor Therapeutics","sponsor":"McQuade Center","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Fibrocor Therapeutics Enters into Research and Development Collaboration with McQuade Center for Strategic Research and Development to Advance Alport Syndrome Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CANADA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Fibrocor Therapeutics"},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"DepYmed Receives Clearance from U.S. Food and Drug Administration to Initiate Phase 1 Clinical Trial for DPM-1003 for the Treatment of Rett Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"DepYmed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Almirall","pharmaFlowCategory":"D","amount":"$473.0 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Therapeutics"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Tyra Biosciences Receives FDA Rare Pediatric Disease Designation for TYRA-300 for the Treatment of Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Tyra Biosciences"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Arctic Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arctic Therapeutics and Nacuity Pharmaceuticals Announce European Medicines Agency Approval to Initiate First Clinical Trial of AT-001 (NPI-001) for the Treatment of HCCAA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Nacuity Pharmaceuticals"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-191 Gene Therapy for Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prime Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for PM359 for the Treatment of Chronic Granulomatous Disease (CGD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Prime Medicine"}]

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            PM359, Prime Medicine’s first product candidate within its hematology and immunology area of focus, targets the p47phox variant of chronic granulomatous disease (CGD), a serious, life-threatening disease that presents in childhood.

            Lead Product(s): PM359

            Therapeutic Area: Genetic Disease Product Name: PM359

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 29, 2024

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