United Kingdom

Freeline

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The Boulder Peptide Society

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Freeline Presents New Haemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting

Lead Product(s): FLT180a

Therapeutic Area: Genetic Disease Product Name: FLT180a

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 13, 2020

Details:

Updated FLT180a data in the B-AMAZE study in severe Haemophilia B patients demonstrated the potential for sustained normal FIX activity levels.

Bluebird bio

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Data Show Near Elimination of Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Clinical Study of bluebird bio’s LentiGlobin™

Lead Product(s): LentiGlobin

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase I/ Phase II 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:

LentiGlobin™ gene therapy for adult and adolescent patients with sickle cell disease (SCD) show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS).

Orchard Therapeutics

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Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome

Lead Product(s): OTL-201

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 27, 2020

Details:

First patient has been dosed in an open-label, investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of mucopolysaccharidosis type IIIA.

Freeline

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Freeline receives Orphan Drug Designation from the European Commission

Lead Product(s): FLT190

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 10, 2020

Details:

A 3 to 4-fold increase in plasma αGLA activity was achieved by week 4 and sustained through the data cutoff at week 20.

Freeline

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Freeline presents data on its AAV-based gene therapies

Lead Product(s): FLT190

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 12, 2020

Details:

As of the data cutoff date, there have been no infusion-related adverse events. Two Grade 1 or 2 serious adverse events have been reported in the study.

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Freeline

Freeline Presents New Haemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting

Bluebird bio

Data Show Near Elimination of Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Clinical Study of bluebird bio’s LentiGlobin™

Orchard Therapeutics

Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome

Freeline

Freeline receives Orphan Drug Designation from the European Commission

Freeline

Freeline presents data on its AAV-based gene therapies