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Kids"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0.56000000000000005,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56000000000000005,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Eli Lilly"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"G542X","graph1":"Genetic 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Foundation"},{"orgOrder":0,"company":"Endure Bio","sponsor":"National Cancer Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Endure Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Endure Bio \/ National Cancer Institute","highestDevelopmentStatusID":"4","companyTruncated":"Endure Bio \/ National Cancer Institute"},{"orgOrder":0,"company":"Neucore Bio","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neucore 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Children\u2019s Research Institute \/ Moderna Therapeutics"},{"orgOrder":0,"company":"LambdaVision","sponsor":"NASA","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LambdaVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"LambdaVision \/ NASA","highestDevelopmentStatusID":"4","companyTruncated":"LambdaVision \/ NASA"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Kinea Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Licensing Agreement","leadProduct":"KNA-155","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solid 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Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"NS-051\/NCNP-04","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NS Pharma \/ Undisclosed"}]

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                            Lead Product(s) : KNA-155

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Kinea Bio

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            Solid Biosciences Inks Licensing Deal with Kinea Bio for Next-Gen Capsid AAV-SLB101 Use

                            Details : Kinea Bio will use Solid’s next-generation capsid, AAV-SLB101, for the development of KNA-155, an investigational dual AAV gene therapy.

                            Product Name : KNA-155

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            September 23, 2025

                            Lead Product(s) : KNA-155

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Kinea Bio

                            Deal Size : Undisclosed

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                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            GENFIT Ends VS-01 Program in ACLF, Refocuses Development on UCD

                            Details : VS-01 acts as an "ammonia scavenger" to enhance the clearance of toxins, such as ammonia, from the blood, being investigated for UCD disordrers.

                            Product Name : VS-01

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 19, 2025

                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            NS Pharma

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                            BioJapan
                            Not Confirmed

                            NS Pharma

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                            BioJapan
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                            Lead Product(s) : NS-051/NCNP-04

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for Duchenne Muscular Dystrophy

                            Details : NS-051/NCNP-04 promotes the skipping of exon 51 within the dystrophin gene. This is expected to have the effect of stabilizing or improving muscle function in DMD patients.

                            Product Name : NS-051/NCNP-04

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            September 19, 2025

                            Lead Product(s) : NS-051/NCNP-04

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            CAMP4 Therapeutics

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                            BioJapan
                            Not Confirmed

                            CAMP4 Therapeutics

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                            Lead Product(s) : CMP-SYNGAP

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Coastlands Capital

                            Deal Size : $100.0 million

                            Deal Type : Private Placement

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                            CAMP4 Announces an Oversubscribed Private Placement of up to $100 Million

                            Details : CAMP4 intends to use the net proceeds from the private placement to fund the preclinical and clinical development of its SYNGAP1 program, CMP-SYNGAP.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            September 10, 2025

                            Lead Product(s) : CMP-SYNGAP

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Coastlands Capital

                            Deal Size : $100.0 million

                            Deal Type : Private Placement

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                            05

                            LambdaVision

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                            BioJapan
                            Not Confirmed

                            LambdaVision

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : NASA

                            Deal Size : Undisclosed

                            Deal Type : Funding

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                            LambdaVision Secures NASA Phase 2 InSPA Award to Advance Manufacturing Efforts

                            Details : This NASA award will enable LambdaVision to further develop layer-by-layer manufacturing processes to support artificial retina production.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            September 04, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : NASA

                            Deal Size : Undisclosed

                            Deal Type : Funding

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                            06

                            Neucore Bio

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                            BioJapan
                            Not Confirmed

                            Neucore Bio

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                            BioJapan
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : National Institutes of Health

                            Deal Size : $0.3 million

                            Deal Type : Funding

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                            Neucore Awarded NIH STTR Grant to Pursue Exosome-based Genetic Medicine for CMT1A

                            Details : The proceeds will be used to evaluate the Company’s targeted exosome platform to deliver an RNA-based therapy to treat Charcot-Marie-Tooth Disease Type 1a (CMT1A).

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            September 02, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : National Institutes of Health

                            Deal Size : $0.3 million

                            Deal Type : Funding

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                            07

                            Amplo Biotechnology

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                            BioJapan
                            Not Confirmed

                            Amplo Biotechnology

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                            BioJapan
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                            Lead Product(s) : AMP-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Andelyn Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            Andelyn and Amplo Partner to Manufacture Novel Gene Therapies

                            Details : The partnership centers on manufacturing clinical-grade AAV vectors using Andelyn's suspension AAV Curator® platform to support Amplo’s NMJ-focused gene therapy candidates, including AMP-101.

                            Product Name : AMP-101

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            August 21, 2025

                            Lead Product(s) : AMP-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Andelyn Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            08

                            Endure Bio

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                            BioJapan
                            Not Confirmed

                            Endure Bio

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                            BioJapan
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : National Cancer Institute

                            Deal Size : Undisclosed

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Endure Receives SBIR Phase I Grant to Advance Engineered Native Bacteria Therapy

                            Details : The funding will support the development of a novel Engineered Native Bacteria (ENB) candidate for the treatment of Familial Adenomatous Polyposis (FAP).

                            Product Name : Undisclosed

                            Product Type : Microorganism

                            Upfront Cash : Undisclosed

                            August 12, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : National Cancer Institute

                            Deal Size : Undisclosed

                            Deal Type : Funding

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                            09

                            Neucore Bio

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                            BioJapan
                            Not Confirmed

                            Neucore Bio

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                            BioJapan
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : National Science Foundation

                            Deal Size : $0.3 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Neucore Awarded Competitive Grant from the NSF for Exosome-based Therapeutics

                            Details : The funding will support to evaluate scalable manufacturing efficiencies to deliver exosome-based therapeutics for challenging genetic disorders affecting the peripheral nerve system (PNS).

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            August 06, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : National Science Foundation

                            Deal Size : $0.3 million

                            Deal Type : Funding

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                            10

                            Repair Biotechnologies

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                            BioJapan
                            Not Confirmed

                            Repair Biotechnologies

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                            BioJapan
                            Not Confirmed
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                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Repair Biotech Wins FDA Orphan Drug Tag for Homozygous Familial Hypercholesterolemia

                            Details : REP-0003 is a cutting-edge messenger RNA therapy that clears harmful excess free cholesterol inside cells while preserving essential cholesterol, for homozygous familial hypercholesterolemia.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 19, 2025

                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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