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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"}]

Find FDA Investigational New Drug (IND) Submissions for Genetic Disease in UNITED STATES

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            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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            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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            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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            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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            ACDN-01 is the first-ever clinical-stage RNA exon editor, which is being evaluated in preclinical studies for the treatment of Stargardt disease & ABCA4 retinopathies.

            Lead Product(s): ACDN-01

            Therapeutic Area: Genetic Disease Product Name: ACDN-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 January 29, 2024

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            JUV-161, which was identified by Juvena’s AI-driven drug discovery and development platform, has demonstrated an ability to restore muscle fiber formation, counter muscle atrophy & enhance muscle strength. It is being evaluated in preclinical studies for the treatment of DM1.

            Lead Product(s): JUV-161

            Therapeutic Area: Genetic Disease Product Name: JUV-161

            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 January 23, 2024

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            VCA-894A (2'-O-methoxyethyl Phosphorothioate Oligonucleotide Sodium) specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2 (IGHMBP2). It is being evaluated in preclinical studies for Charcot-Marie-Tooth Disease, Type 2S.

            Lead Product(s): 2\'-O-methoxyethyl Phosphorothioate Oligonucleotide Sodium

            Therapeutic Area: Genetic Disease Product Name: VCA-894A

            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 January 23, 2024

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            The financing will support the Phase 2 Alpestria-1 study and further profiling of EYP001 (vonafexor), a highly selective NR1H4 agonist, in other kidney diseases, such as Autosomal Dominant Polycystic Kidney Disease (ADPKD).

            Lead Product(s): Vonafexor

            Therapeutic Area: Genetic Disease Product Name: EYP001

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: OrbiMed

            Deal Size: $42.5 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing January 03, 2024

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            SAT-3247 as lead drug candidate that target AAK1, a protein kinase, which is investigated for the treatment of Duchenne Muscular Dystrophy. The patent applications for its drug target and drug development candidates, and remains on track to initiate clinical development in mid-2024.

            Lead Product(s): SAT-3247

            Therapeutic Area: Genetic Disease Product Name: SAT-3247

            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 November 14, 2023

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            The funds will be used to advance Ascidian's lead program, an RNA exon editor to halt the progression of Stargardt disease or other ABCA4 retinopathies, and additional programs in the company's pipeline.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Apple Tree Partners

            Deal Size: $40.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing November 08, 2023

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