Seqens Seqens

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Applicable","newsHeadline":"Regeneron Genetics Center Discovers Rare Mutations In The CIDEB Gene That Protect Against Liver Disease","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Preclinical"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam to Present Data from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at the 18th International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Reports Positive Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy","therapeuticArea":"Nephrology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Nephrology","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Cerveau Technologies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cerveau Technologies Inc. and Alnylam\u00ae Pharmaceuticals Sign Agreement to Provide Novel Tau Imaging Biomarker for Disease Modifier Research","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam to Present Additional Data from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at the Heart Failure Society of America Annual Scientific Meeting 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Presents Positive Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam and Regeneron Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regeneron and Alnylam Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Receives Approval in Europe for AMVUTTRA\u00ae (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult Patients with Stage 1 or Stage 2 Polyneuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Presents Additional Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at Heart Failure Society of America Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO\u00ae (lumasiran) in Advanced Primary Hyperoxaluria Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration (FDA) for ONPATTRO\u00ae (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes","therapeuticArea":"Endocrinology","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Endocrinology","graph2":"IND Enabling"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces U.S. Food and Drug Administration (FDA) Acceptance of Supplemental New Drug Application for ONPATTRO\u00ae (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Medison Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alnylam Pharmaceuticals and Medison Pharma Announce the Expansion of Their Collaboration to a Multi-Regional Partnership to Commercialize RNAi Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam and Regeneron Report Positive Interim Phase 1 Clinical Data on ALN-APP, an Investigational RNAi Therapeutic for Alzheimer\u2019s Disease and Cerebral Amyloid Angiopathy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Vir Biotechnology","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vir Biotechnology Announces First Patient Dosed in New Phase 2 Chronic Hepatitis B Virus Trial Evaluating Combinations of VIR-2218, VIR-3434, PEG-IFN\u03b1 and an NRTI","therapeuticArea":"Infections and Infectious Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Infections and Infectious Diseases","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces Date of Planned FDA Advisory Committee Meeting for ONPATTRO (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Reports Updated Positive Interim Phase 1 Results for ALN-APP, in Development for Alzheimer\u2019s Disease and Cerebral Amyloid Angiopathy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$310.0 million","newsHeadline":"Roche Enters Partnership with Alnylam to Co-Develop and Co-Commercialise RNAi Therapeutic Zilebesiran to Treat Hypertension in Patients with High Cardiovascular Risk","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Agios Pharmaceuticals","pharmaFlowCategory":"D","amount":"$147.5 million","upfrontCash":"$17.5 million","newsHeadline":"Agios Announces Exclusive Worldwide License Agreement for Novel siRNA for the Potential Treatment of Polycythemia Vera","therapeuticArea":"Oncology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Oncology","graph2":"Preclinical"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche and Alnylam Report Positive Topline Results from Phase 2 Study Kardia-1 of Zilebesiran, an Investigational RNAi Therapeutic in Development to Treat Hypertension in Patients at High Risk of Cardiovascular Disease","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting on Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Reports Additional Positive Interim Phase 1 Results for ALN-APP, in Development for Alzheimer\u2019s Disease and Cerebral Amyloid Angiopathy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche and Alnylam Report Positive Topline Results from The Phase II Kardia-2 Study in People with Hypertension, Demonstrating Clinically Significant Blood Pressure Reductions with Zilebesiran when Added to Standard of Care","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Presents Positive Results from the KARDIA-2 Phase 2 Study of Zilebesiran Added to Standard of Care Antihypertensives in Patients with Inadequately Controlled Hypertension","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II"}]

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            ALN-AGT01 (zilebesiran) investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen in development for the treatment of hypertension in high unmet need populations.

            Lead Product(s): Zilebesiran,Olmesartan Medoxomil

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: ALN-AGT01

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 07, 2024

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            ALN-AGT01 (zilebesiran) is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen. It is being evaluated in combination with standard of care for the treatment of hypertension.

            Lead Product(s): Zilebesiran,Olmesartan Medoxomil

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: ALN-AGT01

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 05, 2024

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            ALN-APP is an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA).

            Lead Product(s): ALN-APP

            Therapeutic Area: Neurology Product Name: ALN-APP

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 25, 2023

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            Onpattro (patisiran) is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference resulting in reduction of serum TTR protein and TTR protein deposits in tissues. It is being developed for cardiomyopathy of ATTR amyloidosis.

            Lead Product(s): Patisiran

            Therapeutic Area: Genetic Disease Product Name: Onpattro

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 09, 2023

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            Onpattro (patisiran) is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference resulting in reduction of serum TTR protein and TTR protein deposits in tissues. It is being developed for cardiomyopathy of ATTR amyloidosis.

            Lead Product(s): Patisiran

            Therapeutic Area: Genetic Disease Product Name: Onpattro

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 13, 2023

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            ALN-AGT01 (zilebesiran), is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations.

            Lead Product(s): Zilebesiran

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: ALN-AGT01

            Highest Development Status: Phase II Product Type: Large molecule

            Recipient: F. Hoffmann-La Roche

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 07, 2023

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            Under the agreement, Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).

            Lead Product(s): siRNA

            Therapeutic Area: Oncology Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Agios Pharmaceuticals

            Deal Size: $147.5 million Upfront Cash: $17.5 million

            Deal Type: Licensing Agreement August 03, 2023

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            Under the partnership, Roche and Alnylam will co-develop and co-commercialise RNAi therapeutic ALN-AGT01 (zilebesiran), an investigational RNAi therapeutic currently in Phase 2 for the treat hypertension in patients with high cardiovascular risk.

            Lead Product(s): Zilebesiran,Olmesartan Medoxomil

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: ALN-AGT01

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

            Deal Size: Undisclosed Upfront Cash: $310.0 million

            Deal Type: Partnership July 24, 2023

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            ALN-APP is an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA).

            Lead Product(s): ALN-APP

            Therapeutic Area: Neurology Product Name: ALN-APP

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 17, 2023

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            Onpattro (patisiran) is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference resulting in reduction of serum TTR protein and TTR protein deposits in tissues. It is being developed for cardiomyopathy of ATTR amyloidosis.

            Lead Product(s): Patisiran

            Therapeutic Area: Genetic Disease Product Name: Onpattro

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 30, 2023

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