Genetic Disease | Phase II Developments | UNITED KINGDOM

GlaxoSmithKline

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Orchard Licenses Gene Therapy Technology from GSK

Lead Product(s): OTL-103

Therapeutic Area: Genetic Disease Product Name: OTL-103

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

Partner/Sponsor/Collaborator: Orchard Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement July 15, 2020

Details:

Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.

ReNeuron

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ReNeuron Announces Further Positive Data in Retinal Clinical Trial

Lead Product(s): Human retinal progenitor cell therapy

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: 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 29, 2020

Details:

The Company reports that the latest data continue to demonstrate the efficacy of the therapy (hRPC stem cell therapy) , with a clinically meaningful benefit being observed at all time-points.

Oxford Biomedica

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Sanofi Returns Rights to Gene Therapies to Oxford Biomedica

Lead Product(s): SAR422459

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: Sanofi

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement June 09, 2020

Details:

Oxford Biomedica has been informed by Sanofi that following completion of a company-wide portfolio review, it intends to return to OXB the rights to the following ophthalmology gene therapy programmes: SAR422459 for Stargardt disease & SAR421869 for Usher’s Syndrome type 1b.

IntraBio

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IntraBio Receives FDA Fast Track Designation for Niemann-Pick Disease

Lead Product(s): N-Acetyl-L-Leucine

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 25, 2020

Details:

IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of NPC in a multinational clinical trial (IB1001-201).

Mereo BioPharma Group

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Mereo BioPharma Announces Additional Positive Data from Phase 2b ASTEROID Study of Setrusumab in Adults with Osteogenesis Imperfecta and Pro...

Lead Product(s): Setrusumab

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 14, 2020

Details:

Setrusumab demonstrated a dose dependent increase in both failure load and stiffness at the radius at 12 months, achieving statistical significance in the high dose cohort.