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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.
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