AbbVie Contract Manufacturing AbbVie Contract Manufacturing

X
[{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","amount":"$61.0 million","upfrontCash":"Undisclosed","newsHeadline":"Emendo Biotherapeutics Raises $61 Million to Advance Next Generation Genome Editing Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Emendo Biotherapeutics"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","amount":"$150.0 million","upfrontCash":"$1.2 million","newsHeadline":"XOMA Acquires Royalty Interest in Four Lysosomal Storage Disorder Enzymes Being Developed Under the Chiesi Group\u2013Bioasis Strategic Alliance","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"XOMA"},{"orgOrder":0,"company":"Allen Institute","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Allen Institute and BioMarin Team Up To Develop Gene Therapies For Rare Brain Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Allen Institute"},{"orgOrder":0,"company":"Argonaut Manufacturing Services","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Argonaut to Manufacture ASO Medicines for Patients with Nano-rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Argonaut Manufacturing Services"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ResVita Bio Receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ResVita Bio"},{"orgOrder":0,"company":"Medable","sponsor":"Every Cure","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Medable Partners with Every Cure to Accelerate Discovery of Treatments for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Undisclosed","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Undisclosed","graph2":"Medable"},{"orgOrder":0,"company":"Orbus Therapeutics","sponsor":"Michigan State University","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orbus Therapeutics Enters Exclusive License Agreement for Intellectual Property Related to Treatment of Rare Pediatric Syndrome with Eflornithine","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Orbus Therapeutics"},{"orgOrder":0,"company":"Burjeel Holdings","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Burjeel Holdings to Launch Rare Disease Research & Development Project 'NADER' in Partnership with US-based BridgeBio Pharma","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Burjeel Holdings"}]

Find Novel Drugs for Rare Diseases and Disorders under Development

Loading...
Filters Filter
×
FILTER:
filter Company
    filter Sponsor
      filter Country
        filter Product Type
          filter News Type

            Active Filter(s):

            Product Type

            Companies

            Burjeel Holdings

            Contact Supplier
            • Deals
            Virtual Booth Virtual Booth
            Digital Content Digital Content

            Details:

            Under the partnership, Burjeel and BridgeBio will revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Small molecule

            Partner/Sponsor/Collaborator: BridgeBio Pharma

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership July 12, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            DFMO (eflornithine) is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in mammalian polyamine biosynthesis that is up-regulated in certain types of cancer.

            Lead Product(s): Eflornithine Hydrochloride

            Therapeutic Area: Rare Diseases and Disorders Product Name: DFMO

            Highest Development Status: Undisclosed Product Type: Small molecule

            Partner/Sponsor/Collaborator: Michigan State University

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement January 10, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Medable will partner with Every Cure by providing specialized software and services to conduct global, remote clinical trials for drug repurposing candidates.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Every Cure

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 04, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            RVB-001, a genetically engineered probiotic that is topically applied to temporarily colonize the skin and continuously release LEKTI, thereby inhibiting proteolysis and restoring the integrity of the epidermal barrier.

            Lead Product(s): RVB-001

            Therapeutic Area: Rare Diseases and Disorders Product Name: RVB-001

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 09, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            The partnership will supports and reduces the cost of manufacturing of n-Lorem’s experimental antisense oligonucleotide (ASO) medicines developed for nano-rare disease patients (1 to 30 patients worldwide) for free, for life.

            Lead Product(s): Antisense Oligonucleotides

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: n-Lorem Foundation

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership February 02, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Allen Institute

            Contact Supplier
            • Deals
            Virtual Booth Virtual Booth
            Digital Content Digital Content

            Details:

            The Allen Institute and BioMarin Pharmaceutical will use technologies developed at the Allen Institute to create new gene therapies against rare genetic diseases linked with CNS. BioMarin will receive an exclusive license to each program for R&D and commercialization.

            Lead Product(s): AAV-based gene therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: BioMarin Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 28, 2021

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            XOMA gains rights to future milestone and royalty revenues associated with the advancement of the enzymes being investigated as potential treatments for four different lysosomal storage disorders (LSD) under Bioasis’ strategic alliance with Chiesi Group.

            Lead Product(s): Enzyme replacement therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Chiesi Group

            Deal Size: $150.0 million Upfront Cash: $1.2 million

            Deal Type: Licensing Agreement November 03, 2020

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            The funding will accelerate novel OMNI gene editing platform into a broad therapeutic product pipeline.

            Lead Product(s): OMNI nuclease

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: AnGes

            Deal Size: $61.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing January 15, 2020

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity
            Post Enquiry
            POST ENQUIRY