[{"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","year":"2020","type":"Series B Financing","leadProduct":"OMNI nuclease","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0.059999999999999998,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Bio.."},{"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":"","productType":"Large molecule","productStatus":"Undisclosed","date":"November 2020","year":"2020","type":"Licensing Agreement","leadProduct":"Enzyme replacement therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"XOMA","amount2":0.14999999999999999,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.14999999999999999,"dosageForm":"","sponsorNew":"XOMA \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"XOMA \/ Chi.."},{"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","year":"2021","type":"Collaboration","leadProduct":"AAV-based gene therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Allen Institute","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Allen Institute \/ BioMarin","highestDevelopmentStatusID":"1","companyTruncated":"Allen Inst.."},{"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":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotides","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Argonaut Manufacturing Services","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Argonaut Manufacturing Services \/ n-Lorem Foundation","highestDevelopmentStatusID":"1","companyTruncated":"Argonaut M.."},{"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":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","year":"2022","type":"Not Applicable","leadProduct":"RVB-001","moa":"Proteolysis","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"ResVita Bio","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"ResVita Bio \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"ResVita Bi.."},{"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":"","productType":"Undisclosed","productStatus":"Approved","date":"January 2023","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Medable","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Medable \/ Every Cure","highestDevelopmentStatusID":"1","companyTruncated":"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":"","productType":"Small molecule","productStatus":"Approved","date":"January 2023","year":"2023","type":"Licensing Agreement","leadProduct":"Eflornithine Hydrochloride","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Orbus Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Solution","sponsorNew":"Orbus Therapeutics \/ Michigan State University","highestDevelopmentStatusID":"1","companyTruncated":"Orbus Ther.."},{"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":"U.A.E","productType":"Small molecule","productStatus":"Undisclosed","date":"July 2023","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Burjeel Holdings","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Burjeel Holdings \/ BridgeBio Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Burjeel Ho.."}]

Find Novel Drugs for Rare Diseases and Disorders under Development

Weekly News Recap
#Phispers

Stock Recap
#PipelineProspector

Filters

×

filter

Company Name

filter

Year

filter

DEALS // DEV.

filter

Country

filter

Sponsor

filter

Therapeutic Area

filter

Study Phase

filter

Deal Type

filter

Product Type

filter

Dosage Form

filter

Lead Product

filter

Target

All Developments

Therapeutic Area by Lead Product

Study Phase by Lead Product

Company by Lead Product

Top Deals by Deal Size (USD bn)

Reset All

01

Burjeel Holdings

Country

26th North American ISSX

Not Confirmed

02

Orbus Therapeutics

U.S.A

26th North American ISSX

Not Confirmed

03

Medable

U.S.A

26th North American ISSX

Not Confirmed

04

ResVita Bio

U.S.A

26th North American ISSX

Not Confirmed

05

Argonaut Manufacturing Services

U.S.A

26th North American ISSX

Not Confirmed

06

Allen Institute

Country

26th North American ISSX

Not Confirmed

07

XOMA

U.S.A

26th North American ISSX

Not Confirmed