CSBio CSBio

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Therapeutics Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Design Therapeutics"},{"orgOrder":0,"company":"Peak Bio","sponsor":"White Lion Capital","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Peak Bio, Inc. 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therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces Completion of Enrollment in Second Cohort of Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","amount":"$8.6 million","upfrontCash":"Undisclosed","newsHeadline":"ATA-200, Atamyo Therapeutics\u2019 Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C\/R5, Reaches Key Milestones 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            With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene's lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3.

            Lead Product(s): LYS-GM101

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Lysogene

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration May 26, 2020

            Axplora CB

            TMF Summit 2024

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            envelop Contact Supplier

            Details:

            ARCT-032 utilizes Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. It is under phase 1 clinical development for the treatment of Cystic Fibrosis.

            Lead Product(s): ARCT-032

            Therapeutic Area: Genetic Disease Product Name: ARCT-032

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 22, 2024

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            RCT2100 is an inhaled mRNA-based therapy developed using our novel SORT lipid nanoparticle delivery platform. It is being developed for the treatment of cystic fibrosis.

            Lead Product(s): RCT2100

            Therapeutic Area: Genetic Disease Product Name: RCT2100

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 21, 2024

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            9MW3011 is an anti-TMPRSS6 antibody which can upregulate the level of hepcidin expressed by hepatocytes through specific binding. It is being evaluated for the treatment of β-thalassemia and polycythemia vera.

            Lead Product(s): 9MW3011

            Therapeutic Area: Genetic Disease Product Name: DISC-3405

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Disc medicine

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2024

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            The company intends to use the net proceeds for company’s lead cell therapy asset, NXC-201 for autoimmune disease, relapsed/refractory AL Amyloidosis, and relapsed/refractory multiple myeloma, currently being evaluated in an ongoing Phase 1/2 trial.

            Lead Product(s): NXC-201

            Therapeutic Area: Genetic Disease Product Name: NXC-201

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Titan Partners Group LLC

            Deal Size: $15.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 08, 2024

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            The Company intends to use the net proceeds for company’s lead cell therapy asset is CAR-T NXC-201 for autoimmune disease, relapsed/refractory AL Amyloidosis, and relapsed/refractory multiple myeloma, currently being evaluated in an ongoing Phase 1/2 trial.

            Lead Product(s): NXC-201

            Therapeutic Area: Genetic Disease Product Name: NXC-201

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Titan Partners Group LLC

            Deal Size: $15.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 05, 2024

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            The financing will fund the Phase 2a clinical trial of the Company’s lead programme, ETD001, a novel first in class ENaC blocker, to deliver clinical proof of concept to treat cystic fibrosis.

            Lead Product(s): ETD001

            Therapeutic Area: Genetic Disease Product Name: ETD001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Panakes Partners

            Deal Size: $33.1 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing January 30, 2024

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            SION-109 is a small molecule designed to target the interface between the ICL4 region and the first NBD1 of the CFTR protein. It is under phase 1 clinical development for the treatment of delta-F508 mutated Cystic Fibrosis.

            Lead Product(s): SION-109

            Therapeutic Area: Genetic Disease Product Name: SION-109

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 24, 2024

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            Through the acquisition, NAYA Biosciences expands its clinical pipeline to additional regenerative medicine, including clinical stage gene therapy program for leber's hereditary optic neuropathy (LHON).

            Lead Product(s): AAV-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Naya Biosciences

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition January 23, 2024

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            BBI-001 is a novel oral investigational drug. It is designed to rapidly form a strong & selective iron chelate in the digestive tract, thus inhibiting iron absorption. It is being evaluated in preclinical studies for symptomatic iron overload due to hereditary hemochromatosis.

            Lead Product(s): BBI-001

            Therapeutic Area: Genetic Disease Product Name: BBI-001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 11, 2024

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