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EMA has granted Priority Medicines designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: OTL-203
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 28, 2020
Details:
New preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI.
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: OTL-203
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 01, 2020
Details:
New interim data from OTL-203, an investigational gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I), will be shared as part of an invited oral presentation titled ‘Gene Therapy in Leucodystrophies and Other Metabolic Disorders’.
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: OTL-203
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 24, 2020
Details:
Orchard Therapeutics has received both orphan drug designation and rare pediatric disease designation from the U.S FDA for OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: OTL-203
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 20, 2020
Details:
MPS-I clinical trial interim results demonstrated- all patients, treated with OTL-203, achieving hematological recovery in less than 45 days – a primary outcome measure – as well as supraphysiological levels of IDUA enzyme in the blood.
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 15, 2020
Details:
Accepted abstracts include data on investigational gene therapies for neurometabolic conditions, including updated interim data from proof-of-concept trial in OTL-203 for Mucopolysaccharidosis Type I.
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 29, 2020
Details:
Early clinical data support ex vivo hematopoietic stem cell gene therapy as a potentially promising treatment option for X-CGD.
Lead Product(s): OTL-102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 29, 2020
