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Find Novel Drugs for Genetic Disease under Development

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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: Sanofi Pasteur SA

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration February 23, 2021

            Details:

            Sanofi, together with SIRION will develop new and modified AAV capsids that exhibit a safe product profile with improved specificity and higher gene delivery efficiency.

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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: Pfizer Inc

            Deal Size: $239.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration January 06, 2021

            Details:

            Dewpoint Therapeutics and Pfizer will collaborate for the development of potential therapeutics for the treatment of myotonic dystrophy type 1, DM1, a rare genetic disorder and one of two types of myotonic dystrophy.

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            Lead Product(s): Cell-penetrating peptides conjugates

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Peptide

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: $45.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing December 09, 2020

            Details:

            PepGen will use the funding to advance into the clinic their next-generation cell-penetrating peptides conjugated to phosphorodiamidate morpholino oligomers (PPMOs) that are designed to correct genetic defects in diseases with high unmet medical need.

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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.