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    [{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prevail Therapeutics Provides PR001 Program Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Prevail Therapeutics"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"GeneTx Biotherapeutics"},{"orgOrder":0,"company":"Emerald Health Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Emerald Health Pharmaceuticals Granted Orphan Designation in Europe for Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Emerald Health Pharmaceuticals"},{"orgOrder":0,"company":"Passage Bio","sponsor":"J.P. 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EU"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"US FDA Designates PYC'S Lead as a Fast Track Development Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"PYC Therapeutics"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-006, the First-ever RNA Editing Clinical Candidate, and Plans for Upcoming Virtual \u201cR&D Day\u201d","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":"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. 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":"Prime Medicine"},{"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":"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 million","upfrontCash":"$3.0 million","newsHeadline":"Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to License ZKN-013 for Rare Dermatological Diseases","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":"Eloxx Pharmaceuticals"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pasithea Therapeutics Announces Positive In Vivo Preclinical Efficacy Data for PAS-004 from NRAS Mutation Cancer Xenograft Models","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Pasithea 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"}]

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              Vertex Pharmaceuticals

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              Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

              Details:

              VX-407 is a first-in-class small molecule corrector that is designed to inhibit PKD1. It is being evaluated for the treatment of autosomal dominant polycystic kidney disease.

              Lead Product(s): VX-407

              Therapeutic Area: Genetic Disease Product Name: VX-407

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 21, 2024

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              DepYmed

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              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

              Details:

              DPM-1003 orally bioavailable drug candidates that act by inhibiting PTP1B, which is being evaluated for the treatment of patients suffering from Rett Syndrome.

              Lead Product(s): DPM-1003

              Therapeutic Area: Genetic Disease Product Name: DPM-1003

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 19, 2024

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              Eloxx Pharmaceuticals

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              Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to License ZKN-013 for Rare Dermatological Diseases

              Details:

              Almirall obtains exclusive global rights to develop and commercialize ZKN-013, Eloxx’s lead TURBO-ZM based molecule, for the treatment of rare dermatological and other diseases associated with nonsense mutations.

              Lead Product(s): ZKN-013

              Therapeutic Area: Genetic Disease Product Name: ZKN-013

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Almirall

              Deal Size: $473.0 million Upfront Cash: $3.0 million

              Deal Type: Licensing Agreement March 13, 2024

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              Nacuity Pharmaceuticals

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              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

              Details:

              NPI-001 (AT-001 ) combats rare hereditary forms of dementia. Currently, it is being evaluated in the IND enabling stage for the treatment of Hereditary Cystatin C Amyloid Angiopathy.

              Lead Product(s): Grapiprant

              Therapeutic Area: Genetic Disease Product Name: NPI-001

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Arctic Therapeutics

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 12, 2024

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              Fibrocor Therapeutics

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              Fibrocor Therapeutics Enters into Research and Development Collaboration with McQuade Center for Strategic Research and Development to Advance Alport Syndrome Program

              Details:

              The collaboration aims to support the clinical development of Fibrocor's FIB918, a monoclonal antibody. Currently, it is being evaluated in the IND-enabling phase for the treatment of Alport Syndrome.

              Lead Product(s): FIB918

              Therapeutic Area: Genetic Disease Product Name: FIB918

              Highest Development Status: IND Enabling Product Type: Large molecule

              Partner/Sponsor/Collaborator: McQuade Center

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Collaboration March 05, 2024

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              ARTHEx Biotech

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              ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)

              Details:

              ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.

              Lead Product(s): ATX-01

              Therapeutic Area: Genetic Disease Product Name: ATX-01

              Highest Development Status: IND Enabling Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 28, 2024

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              Prime Medicine

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              Prime Medicine Announces Closing of Upsized Public Offering and Full Exercise of the Underwriters' Option to Purchase Additional Shares

              Details:

              The net proceeds will be used for the development of PM359, which involves the use of prime editing technology to modify autologous hematopoietic stem cells ex vivo. This blood-targeting therapy is being developed as a potential treatment for chronic granulomatous disease.

              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: J.P. Morgan

              Deal Size: $161.0 million Upfront Cash: Undisclosed

              Deal Type: Public Offering February 20, 2024

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              Prime Medicine

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              Prime Medicine Announces Proposed Public Offering of Common Stock

              Details:

              The net proceeds will be used for the development of PM359, which involves the use of prime editing technology to modify autologous hematopoietic stem cells ex vivo. This blood-targeting therapy is being developed as a potential treatment for chronic granulomatous disease.

              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: J.P. Morgan

              Deal Size: $125.0 million Upfront Cash: Undisclosed

              Deal Type: Public Offering February 14, 2024

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              Tyra Biosciences

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              Tyra Biosciences Receives FDA Rare Pediatric Disease Designation for TYRA-300 for the Treatment of Achondroplasia

              Details:

              TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including achondroplasia.

              Lead Product(s): TYRA-300

              Therapeutic Area: Genetic Disease Product Name: TYRA-300

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 01, 2024

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              Jaguar Gene Therapy

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              Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome

              Details:

              JAG201 is being evaluated in preclinical studies for the treatment of SHANK-3 mutated autism spectrum disorder & Phelan-McDermid syndrome. It aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.

              Lead Product(s): JAG201

              Therapeutic Area: Genetic Disease Product Name: JAG201

              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 January 31, 2024

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              Minakem offers CDMO services for API & HPAPI, generics, regulatory expertise, track record performance & FDA & GMP certifications.
              ChemWerth works in generic API development & supply, non-infringement patent strategy development and regulatory support.
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