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TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRARE technology designed to mediate levels of MECP2 in the CNS. It is under clinical development for the treatment of Rett Syndrome.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 29, 2024
Details:
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRARE technology designed to mediate levels of MECP2 in the CNS. It is under phase 1/2 clinical development for the treatment of Rett Syndrome.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 10, 2024
Details:
TSHA-102 is a MECP2 expression regulator cell and gene therapy drug candidate, undergoing evaluation for patients aged 12 and older with Rett syndrome, administered via intrathecal injection.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 29, 2023
Details:
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel miRARE platform designed to regulate cellular MECP2 expression.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 26, 2023
Details:
TSHA-120 is a clinical-stage AAV9 gene therapy program being developed for the treatment of giant axonal neuropathy, or GAN, which is a rare autosomal recessive disease of the central and peripheral nervous systems caused by loss-of-function gigaxonin gene mutations.
Lead Product(s): TSHA-120
Therapeutic Area: Genetic Disease Product Name: TSHA-120
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 19, 2023
Details:
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel miRARE platform designed to regulate cellular MECP2 expression.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 24, 2023
Details:
The net proceeds will be used primarily to fund clinical development of TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome and to provide support for program activities for TSHA-120 in giant axonal neuropathy.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: GordonMD Global Investments
Deal Size: $150.0 million Upfront Cash: Undisclosed
Deal Type: Private Placement August 21, 2023
Details:
The Company will primarily support the clinical development of TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome and provide support for TSHA-120 program activities in Giant axonal neuropathy.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: RA Capital Management
Deal Size: $150.0 million Upfront Cash: Undisclosed
Deal Type: Private Placement August 14, 2023
Details:
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel miRARE platform designed to regulate cellular MECP2 expression.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 31, 2023
Details:
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel miRARE platform designed to regulate cellular MECP2 expression.
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 05, 2023
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