CSBio CSBio

X
[{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"US FDA Grants Once Daily Dosing Option for NITYR (nitisinone) Tablets for HT-1 Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Cycle Pharmaceuticals"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Genpharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Secures Partnerships to Broaden Access to LibmeldyTM for Eligible Patients in Middle East & Turkey","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Citrine","pharmaFlowCategory":"D","amount":"$13.2 million","upfrontCash":"$0.5 million","newsHeadline":"Citrine Medicine Announces Strategic Partnership with Diurnal To Bring Alkindi\u00ae To China For Use In Pediatric Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces $150 Million Strategic Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Citrine Medicine","pharmaFlowCategory":"D","amount":"$2.2 million","upfrontCash":"$1.0 million","newsHeadline":"Diurnal Triggers $1.25m in Milestone Payments from Citrine Medicine","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group plc - Diurnal receives European Commission approval for Efmody\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group launches Efmody in UK to Treat Patients with Rare Condition Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cycle Pharmaceuticals Launches SAJAZIRTM (icatibant) Injection, A New Treatment Option in Hereditary Angioedema (HAE)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Cycle Pharmaceuticals"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"NHS England","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Historic Agreement Making Libmeldy Available by NHS England for MLD Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Italian Medicines Agency","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Reimbursement Agreement Making Libmeldy Available for All Eligible MLD Patients in Italy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Announces Multiple Presentations at 2022 ESGCT Annual Congress Showing the Potential of HSC Gene Therapy in Several Therapeutic Areas","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"New Therapies Council","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Agreement Enabling Reimbursed Access to Libmeldy for All Eligible MLD Patients in Sweden","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD and Receives Priority Review","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$387.4 million","upfrontCash":"$387.4 million","newsHeadline":"Kyowa Kirin to Acquire Orchard Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Beneluxa Initiative","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

Loading...
Filters Filter
×
FILTER:
filter News Type
    filter Company
      filter Product Type
        filter Deal Size
          filter Upfront Payment

            Active Filter(s):

            Product Type

            Companies

            Details:

            The agreement aims for enabling reimbursed access to Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Beneluxa Initiative

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement January 25, 2024

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Through the acquisition, Kyowa expands its portfolio, enables the development of promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Kyowa Kirin

            Deal Size: $387.4 million Upfront Cash: $387.4 million

            Deal Type: Acquisition October 05, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 18, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 03, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: New Therapies Council

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement February 27, 2023

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel or OTL-200), has been approved for the treatment of metachromatic leukodystrophy (MLD) in eligible early-onset patients characterized by biallelic mutations in ARSA gene leading to reduction of ARSA enzymatic activity in children.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 10, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 29, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Italian Medicines Agency

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement April 11, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            The agreement coincides with publication of the positive National Institute for Health and Care Excellence’s evaluation determination, which recognized the clinical impact and economic benefit of Libmeldy according to the institute’s Highly Specialized Technologies process.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: NHS England

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement February 04, 2022

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity

            Details:

            SAJAZIR™ is a bradykinin B2 receptor antagonist indicated for treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older.

            Lead Product(s): Icatibant Acetate

            Therapeutic Area: Genetic Disease Product Name: Sajazir

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 28, 2021

            ASK
            US
            PHARMACOMPASS
            Upload
            your Pipeline Activity
            Post Enquiry
            POST ENQUIRY