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