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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Autologous CD34+ cell enriched HSPC

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering February 05, 2021

            Details:

            Funds will support Orchard's growing commercial capabilities, including the launch of Libmeldy in Europe, advance its regulatory and clinical development activities and expand its hematopoietic stem cell gene therapy approach into larger indications.

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            Lead Product(s): Libmeldy

            Therapeutic Area: Genetic Disease Product Name: OTL-200

            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 January 28, 2021

            Details:

            Multiple abstracts highlighting clinical and real-world data for OTL-200 and Metachromatic Leukodystrophy (MLD) will be presented at the conference.

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            Lead Product(s): Hydrocortisone

            Therapeutic Area: Genetic Disease Product Name: Alkindi

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Citrine

            Deal Size: $13.2 million Upfront Cash: $0.5 million

            Deal Type: Licensing Agreement January 27, 2021

            Details:

            Citrine will be responsible for obtaining registration for Alkindi® as a treatment for paediatric patients in China and for all commercialisation activities, including pricing and reimbursement.

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            Lead Product(s): Autologous CD34+ cell enriched HSPC

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Genpharma

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 19, 2021

            Details:

            Under terms of the agreements, GenPharm and GEN will be responsible for all activities related to identifying patients, driving disease awareness, and ensuring market access for Libmeldy in their respective territories.

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            Lead Product(s): Nitisinone

            Therapeutic Area: Genetic Disease Product Name: Nityr

            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 14, 2020

            Details:

            NITYR® is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

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            Lead Product(s): Betibeglogene autotemcel

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 12, 2020

            Details:

            89% of evaluable patients (17/19) with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207.