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Dystrophy Gene Therapy Candidate SGT-003","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"}]

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            Development Status

            Details:

            SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding microdystrophin, containing the R16-R17 nNOS binding domain. It is evaluated in preclinical studies for duchenne muscular dystrophy.

            Lead Product(s): SGT-003

            Therapeutic Area: Genetic Disease Product Name: SGT-003

            Highest Development Status: Phase I/ 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 January 16, 2024

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            The Company expects to use net proceeds to fund ongoing pipeline development programs, including SGT-003, a next-generation gene therapy candidate for the treatment of duchenne muscular dystrophy.

            Lead Product(s): SGT-003

            Therapeutic Area: Genetic Disease Product Name: SGT-003

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Citigroup

            Deal Size: $108.9 million Upfront Cash: Undisclosed

            Deal Type: Private Placement January 08, 2024

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            SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein, which is investigated for the treatment of Duchenne Muscular Dystrophy.

            Lead Product(s): SGT-003

            Therapeutic Area: Genetic Disease Product Name: SGT-003

            Highest Development Status: Phase I/ 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 November 14, 2023

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            The collaboration will integrate Solid Biosciences’ vector biology, manufacturing capabilities and drug development experience with Phlox’s deep expertise in genetic cardiomyopathies and RNA therapeutics to develop novel precision genetic medicines for this form of DCM.

            Lead Product(s): Genetic Medicine

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: Phlox Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 11, 2023

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            AVB-202 is being developed with a transgene to encode full-length frataxin protein (210 amino acids) packaged into an AAV9 capsid under the control of a promoter specifically designed to drive expression in neurons and cardiac cells.

            Lead Product(s): AVB-202

            Therapeutic Area: Genetic Disease Product Name: AVB-202

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

            Recipient: AavantiBio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger December 05, 2022

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            Details:

            SGT-003 is Solid's next-generation AAV gene transfer therapy candidate that utilizes a rationally designed, novel muscle-tropic AAV capsid, called AAV-SLB101, to deliver Solid’s proprietary and differentiated nNOS microdystrophin for the treatment of Duchenne.

            Lead Product(s): AAV-SLB101

            Therapeutic Area: Genetic Disease Product Name: SGT-003

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 17, 2022

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            AVB-202 is being developed with a transgene to encode full-length frataxin protein (210 amino acids) packaged into an AAV9 capsid under the control of a promoter specifically designed to drive expression in neurons and cardiac cells.

            Lead Product(s): AVB-202

            Therapeutic Area: Genetic Disease Product Name: AVB-202

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

            Recipient: AavantiBio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger September 30, 2022

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            Details:

            SGT-003 is a next-generation AAV gene transfer therapy candidate that utilizes a rationally designed, novel muscle-tropic AAV capsid, called AAV-SLB101, to deliver Solid’s proprietary and differentiated nNOS microdystrophin for the treatment of Duchenne.

            Lead Product(s): SGT-003

            Therapeutic Area: Genetic Disease Product Name: SGT-003

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

            Partner/Sponsor/Collaborator: Perceptive Advisors

            Deal Size: $75.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement September 30, 2022

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            Details:

            The partnership will advance the development and manufacturing of SGT-003, a preclinical candidate that combines a next-generation and capsid with Solid’s proprietary nNOS-containing microdystrophin and has demonstrated enhanced muscle tropism and microdystrophin expression.

            Lead Product(s): SGT-003

            Therapeutic Area: Genetic Disease Product Name: SGT-003

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

            Partner/Sponsor/Collaborator: Forge Biologics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership October 05, 2021

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            Details:

            Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage. SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy.

            Lead Product(s): SGT-001

            Therapeutic Area: Genetic Disease Product Name: SGT-001

            Highest Development Status: Phase I/ 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 September 29, 2021

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