Genetic Disease | Phase II Developments | UNITED KINGDOM

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.