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Find Novel Drugs for Genetic Disease under Development

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Eli Lilly

            Deal Size: $555.0 million Upfront Cash: $100.0 million

            Deal Type: Collaboration November 20, 2020

            Details:

            Lilly will use Precision's proprietary ARCUS® genome editing platform for the research and development of potential in vivo therapies for genetic disorders, with an initial focus on Duchenne muscular dystrophy and two other undisclosed gene targets.

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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: Sarepta Therapeutics

            Deal Size: $107.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing October 22, 2020

            Details:

            AavantiBio will pursue a diversified gene therapy pipeline, including a lead program in Friedreich’s Ataxia (FA), a rare inherited genetic disease characterized by cardiac and central nervous system dysfunction.

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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 September 28, 2020

            Details:

            EMA has granted Priority Medicines designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

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            Lead Product(s): Afamelanotide

            Therapeutic Area: Genetic Disease Product Name: Scenesse

            Highest Development Status: Undisclosed Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 10, 2020

            Details:

            Having commercialised SCENESSE® in Europe and the USA for the rare genetic disorder porphyria (EPP), CLINUVEL is expanding its clinical research, aiming to confirm how intervention with the drug enhances elimination of photoproducts and regeneration of DNA.

            Cornell University

            • Deals

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            Lead Product(s): REV-0100

            Therapeutic Area: Genetic Disease Product Name: REV-0100

            Highest Development Status: Undisclosed Product Type: Undisclosed

            Partner/Sponsor/Collaborator: reVision Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 01, 2020

            Details:

            Under the terms if the agreement reVision Therapeutics gains rights for the development and commercialization of Cornell's proprietary technology for the treatment of Stargardt disease, a genetic disorder that affects central vision.

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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 September 01, 2020

            Details:

            New preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI.

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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 August 24, 2020

            Details:

            New interim data from OTL-203, an investigational gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I), will be shared as part of an invited oral presentation titled ‘Gene Therapy in Leucodystrophies and Other Metabolic Disorders’.

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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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