Genetic Disease | New Developments | UNITED KINGDOM

Orchard Therapeutics

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Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for 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 September 01, 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.

Orchard Therapeutics

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Orchard Therapeutics Announces Clinical Data Presentations at the 46th Annual Meeting of EBMT

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:

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’.

Orchard Therapeutics

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Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of 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:

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).

Orchard Therapeutics

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Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-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 May 15, 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.

Orchard Therapeutics

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Orchard Therapeutics Announces Eight Presentations Across Gene Therapy Portfolio at the ASGCT 23rd Annual Meeting

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:

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.

Orchard Therapeutics

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Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (...

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

Details:

Early clinical data support ex vivo hematopoietic stem cell gene therapy as a potentially promising treatment option for X-CGD.

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Orchard Therapeutics

Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I

Orchard Therapeutics

Orchard Therapeutics Announces Clinical Data Presentations at the 46th Annual Meeting of EBMT

Orchard Therapeutics

Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I

Orchard Therapeutics

Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-I

Orchard Therapeutics

Orchard Therapeutics Announces Eight Presentations Across Gene Therapy Portfolio at the ASGCT 23rd Annual Meeting

Orchard Therapeutics

Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (...