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[{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Present Axiomer RNA Editing Platform Technology at RNA Editing Summit","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Life Science Partners","pharmaFlowCategory":"D","amount":"$31.0 million","upfrontCash":"Undisclosed","newsHeadline":"Vico Therapeutics Raises $31 Million in Series A Financing Round","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Large molecule","productStatus":"Undisclosed","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Vico Therapeutics"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Huntington Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Vico Therapeutics"},{"orgOrder":0,"company":"VectorY","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"VectorY to Present Poster on Huntington\u2019s Preclinical Data at the Hereditary Disease Foundation, August 24-27 in Cambridge, MA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"VectorY"},{"orgOrder":0,"company":"Xinvento","sponsor":"Rhythm Pharmaceuticals","pharmaFlowCategory":"D","amount":"$211.0 million","upfrontCash":"$5.0 million","newsHeadline":"Rhythm Pharmaceuticals Announces Acquisition of Xinvento B.V. and Portfolio of Investigational Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Peptide","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Xinvento"}]

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            Imcivree (setmelanotide) is a melanocortin 4 (MC4) receptor agonist indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity

            Lead Product(s): Setmelanotide Acetate

            Therapeutic Area: Genetic Disease Product Name: Imcivree

            Highest Development Status: Preclinical Product Type: Peptide

            Partner/Sponsor/Collaborator: Rhythm Pharmaceuticals

            Deal Size: $211.0 million Upfront Cash: $5.0 million

            Deal Type: Acquisition February 27, 2023

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            VecTabs, which are secreted- or intracellular-antibody fragments that are AAV-vectorized and delivered to neurons and/or astrocytes, have previously been shown to efficiently target such misfolded proteins (TDP-43) and toxic lipids (oxPL).

            Lead Product(s): AAV Vector

            Therapeutic Area: Genetic Disease Product Name: VecTabs

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 23, 2022

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            Axiomer editing oligonucleotides are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct change of adenosine to inosine in the RNA, and Inosine is translated as guanosine.

            Lead Product(s): RNA-based Therapeutic

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 04, 2022

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            VICO’s lead antisense oligonucleotide product, VO659 is designed to suppress mutant proteins and slow or halt disease progression.

            Lead Product(s): VO659

            Therapeutic Area: Genetic Disease Product Name: VO659

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 29, 2021

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            Vico will use this funding to further advance its late preclinical stage Antisense OligoNucleotides lead platform for the development of therapies for different forms of Spinocerebellar Ataxia (SCA) and Huntington Disease (HD) into first-in-human clinical trials in late 2021.

            Lead Product(s): Antisense Oligonucleotides

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Life Science Partners

            Deal Size: $31.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing July 30, 2020

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