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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Short hairpin RNA,Gaboxadol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: University of Connecticut

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration July 23, 2020

            Details:

            Ovid to collaborate with renowned molecular geneticist and Angelman syndrome expert Stormy J. Chamberlain, Ph.D., to advance a short hairpin RNA (shRNA)-based therapeutic with the goal of addressing the underlying genetic cause of Angelman syndrome.

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            Lead Product(s): Transcend-peptide

            Therapeutic Area: Genetic Disease Product Name: xB3

            Highest Development Status: Undisclosed Product Type: Peptide

            Partner/Sponsor/Collaborator: CHIESI USA INC

            Deal Size: $141.0 million Upfront Cash: $3.0 million

            Deal Type: Licensing Agreement June 29, 2020

            Details:

            The unique delivery method of their xB3 platform has the potential to overcome a significant challenge in the treatment of many neurological disorders, which is the ability to cross the blood brain barrier.

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Personalis

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration June 02, 2020

            Details:

            As part of this research collaboration, Sarepta will be working with the Personalis team to characterize immune response to precision genetic therapeutics, utilizing Personalis’ advanced proprietary analytics.

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $1.0 million Upfront Cash: Undisclosed

            Deal Type: Funding June 02, 2020

            Details:

            PPMD and Duchenne UK invite proposals which are investigating ways to mitigate the immune response both in terms of pre-existing immunity and immunity induced by receiving a gene therapy dose.

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            Lead Product(s): Stem cell gene therapy

            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 June 02, 2020

            Details:

            Initially, the alliance will develop treatment options for patients with two rare, life-threatening primary immunodeficiency diseases -- Wiskott-Aldrich Syndrome (WAS) and X-linked Agammaglobulinemia (XLA).

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            Lead Product(s): rAAVrh8-HexA/HexB

            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 07, 2020

            Details:

            Presentations to showcase Axovant's investigational gene therapy programs, AXO-Lenti-PD and AXO-AAV-GM2.

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            Lead Product(s): AAV capsid-gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 27, 2020

            Details:

            Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.

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            Lead Product(s): Non-viral gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: CureDuchenne Ventures

            Deal Size: $1.0 million Upfront Cash: Undisclosed

            Deal Type: Financing April 22, 2020

            Details:

            Myosana will use this initial funding to advance development of its innovative non-viral gene therapy to deliver full length dystrophin for Duchenne muscular dystrophy (DMD).

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            Lead Product(s): mRNA

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Guggenheim Securities

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering April 15, 2020

            Details:

            The Company intends to use the net proceeds for working capital including capital expenditures and research and development expenditures related to the advancement of LUNAR-OTC, LUNAR-COV19.

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            Lead Product(s): Oligonucleotide linked antibody

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: CureDuchenne Ventures

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing March 31, 2020

            Details:

            The funding will support Dyne’s development of precision therapeutics designed to restore muscle health in people living with Duchenne muscular dystrophy (DMD).

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