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Find Novel Drugs for Genetic Disease under Development

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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): 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).

            UMass Medical School

            • Development Update

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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: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 08, 2020

            Details:

            The researchers used a gene therapy technique that implements a virus to introduce synthetic microRNAs to bind and effectively switch off the proteins linked to a gene called SOD1, mutations of which are associated with a specific type of familial ALS.

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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.

            University of Massachusetts Medical School

            • Deals

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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: CANbridge

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 09, 2020

            Details:

            This collaboration with the Horae Gene Therapy Center at UMass, a pioneer in gene therapy research, affords CANbridge the potential to extend its rare disease pipeline by integrating next-generation technology.

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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): 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.

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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.

            PharmaCompass