Lead Product(s): Betibeglogene autotemcel
Therapeutic Area: Genetic Disease Product Name: Zynteglo
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 11, 2021
With 51 patients enrolled, data from the long-term follow-up study (LTF-303) show that all patients treated with beti-cel who achieve transfusion independence (TI) remain free from transfusions, with the longest follow-up of seven years.
Lead Product(s): Hydrocortisone
Therapeutic Area: Genetic Disease Product Name: Efmody
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Citrine Medicine
Deal Size: $2.2 million Upfront Cash: $1.0 million
Deal Type: Partnership June 01, 2021
The $1.25 million milestone payment has been triggered as a result of the recent European Commission’s approval for Efmody® (hydrocortisone modified-release hard capsules) and the attainment by Citrine of certain development milestones in China for Alkindi.