[{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1\/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita\u00ae (burosumab) for Tumor-Induced Osteomalacia (TIO)","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Regenxbio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$7.0 million","newsHeadline":"REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV\u00ae Technology Platform","therapeuticArea":"Endocrinology","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Endocrinology","graph2":"Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"D","amount":"$225.0 million","upfrontCash":"$125.0 million","newsHeadline":"Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology","therapeuticArea":"Oncology","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Oncology","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1\/2 Study of DTX301 Gene Therapy OTC Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Orsini Specialty Pharmacy","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orsini Pharmaceutical Services Expands Partnership With Ultragenyx To Provide Dojolvi\u2122 (triheptanoin)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bayer's Gene Therapy BAY 2599023 Showing Steady FVIII Activity, Safety in Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","amount":"$295.0 million","upfrontCash":"$40.0 million","newsHeadline":"Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce Positive Interim Phase 1\/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"D","amount":"$304.0 million","upfrontCash":"$50.0 million","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701 for the Treatment of Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"N-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines for Patients with Ultra-Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2021","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Finalizes Phase 3 Study Design for DTX301 Ornithine Transcarbamylase Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1\/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Crysvita\u00ae (burosumab) and UX143 (setrusumab) Data Presentations at Upcoming American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Initiates Cyprus2+, a Pivotal Clinical Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Provide Preliminary Update on Phase 1\/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","amount":"$93.0 million","upfrontCash":"$30.0 million","newsHeadline":"Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza\u00ae (Evinacumab) Outside the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces First Patient Dosed in Pivotal Phase 2\/3 Clinical Study of Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Abeona Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and GeneTx Provide Program Update on GTX-102 for Angelman Syndrome Including Promising Interim Data from Phase 1\/2 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$75.0 million","newsHeadline":"Ultragenyx Exercises Option to Acquire GeneTx","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1\/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2\/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces First Patients Dosed in Phase 3 Program Evaluating Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta (OI)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Initiation of Dosing in Second Cohort of Pivotal Phase 1\/2\/3 Cyprus2+ Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Launches Evkeeza\u00ae (evinacumab for injection) in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Interim Phase 2 Data from Phase 2\/3 Orbit Study Demonstrating Setrusumab (UX143) Significantly Reduced Fracture Rates in Patients with Osteogenesis Imperfecta (OI)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$345.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$300.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Pricing of Public Offering of Common Stock and Pre-Funded Warrants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$326.1 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Closing of Public Offering of Common Stock and Pre-Funded Warrants and Full Exercise of Underwriters' Option to Purchase Additional Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Completion of Enrollment in Global Phase 1\/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza\u00ae \u2013\u00bc (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1\/2\/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Receives European Commission Decision for Evkeeza\u00ae (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Positive Interim Phase 1\/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by Ultragenyx Pharmaceutical
GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated in the treating of Patients with Angelman Syndrome.
UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A-AS. It is being phase 1/2 clinical trials for the treatment of Angelman Syndrome.
UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter. It is being evaluated in phase 1/2 clinical trials for the treatment of Wilson Disease.
Evkeeza (evinacumab) is the first angiopoietin-like 3 (ANGPTL3) inhibitor. ANGPTL3 is involved in controlling cholesterol levels. It is approved for the treatment of adolescents and adults aged 12 years and older with Homozygous Familial Hypercholesterolemia (HoFH).
GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS). It is under phase 1/2 clinical development for the treatment of pediatric patients with Angelman Syndrome.
Evkeeza (evinacumab) attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood.
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
Market Place
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
