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Health"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Curamys","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"MaxCyte \/ Curamys","highestDevelopmentStatusID":"1","companyTruncated":"MaxCyte \/ Curamys"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"FT-002","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Frontera Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"Frontera Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Genevant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"RNA-based Therapeutic","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Korro Bio \/ Genevant Sciences","highestDevelopmentStatusID":"1","companyTruncated":"Korro Bio \/ Genevant Sciences"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"1","companyTruncated":"2Seventy Bio \/ Novo Nordisk"}]

Find Novel Drugs for Genetic Disease under Development

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                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Through the acquisition, Crosswalk will gain Codexis' investigational AAV based gene therapy, that is being developed to target fabry and pompe diseases.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 01, 2024

                          Lead Product(s) : AAV Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Crosswalk Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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                          02

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : The collaboration is focused on an in vivo gene editing treatment for hemophilia A by utilizing 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $5.0 million

                          May 01, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Novo Nordisk

                          Deal Size : $40.0 million

                          Deal Type : Collaboration

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                          03

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : The Collaboration to combine Korro’s RNA editing technology with Genevant’s industry-leading LNP technology to develop a differentiated therapeutic option for patients with Alpha-1 Antitrypsin Deficiency (AATD).

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          March 07, 2023

                          Lead Product(s) : RNA-based Therapeutic

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Genevant Sciences

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          04

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. It treat patients with X-linked retinitis pigmentosa caused by RPGR gene muta...

                          Brand Name : FT-002

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 17, 2023

                          Lead Product(s) : FT-002

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Under the terms of the agreement, Curamys obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 05, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Curamys

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          06

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Mytesi (crofelemer) is an antidiarrheal indicated in the U.S. for the symptomatic relief of noninfectious diarrhea in adult patients with HIV/AIDS on antiretroviral therapy (ART). Mytesi is not indicated for the treatment of infectious diarrhea.

                          Brand Name : Mytesi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 17, 2022

                          Lead Product(s) : Crofelemer

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Napo Therapeutics

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Exosomes are small membrane vesicles, which are among the most productive and conducive to wound healing and countering inflammation. The benefits will help treat the skin and address signs of aging when delivered as a booster through Hydrafacial’s exc...

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          September 14, 2022

                          Lead Product(s) : Exosome Based Therapeutic

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Beauty Health

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          08

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Using patient-derived retina organoids, the discovered that a type of LCA caused by mutations in the AAV-based NPHP5 Gene Thearapy (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 08, 2022

                          Lead Product(s) : AAV-based NPHP5 Gene Thearapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Replay’s synHSV™ technology, a high payload capacity HSV vector capable of delivering up to 30 times the payload of AAV, is utilized by Replay’s four gene therapy product companies, bringing big DNA treatments to diseases affecting the skin, eye, b...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 25, 2022

                          Lead Product(s) : AAV-based Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : KKR

                          Deal Size : $55.0 million

                          Deal Type : Financing

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                          10

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Lead Product(s) : Re-engineered AAV-LPL

                          Therapeutic Area : Genetic Disease

                          Study Phase : Undisclosed

                          Sponsor : Innovation, Science and Economic Development Canada

                          Deal Size : $0.4 million

                          Deal Type : Collaboration

                          Details : Funding supports Virica’s collaboration with the National Research Council of Canada’s Cell and Gene Therapy Challenge program to enhance the manufacturing of an affordable, AAV-LPL, a gene therapy being developed for people with debilitating lipopro...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          May 02, 2022

                          Lead Product(s) : Re-engineered AAV-LPL

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Innovation, Science and Economic Development Canada

                          Deal Size : $0.4 million

                          Deal Type : Collaboration

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