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ISP-001 (Iduronicrin Genleukocel-T) is the first product candidate developed using the company’s proprietary technique which is under phase 1 clinical development for the treatment of mucopolysaccharidosis type I (MPS I).


Lead Product(s): Iduronicrin Genleukocel-T

Therapeutic Area: Genetic Disease Product Name: ISP-001

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 December 15, 2023

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This funding will support a Phase I study to evaluate the safety and tolerability of ISP-001 (for delivery of alpha-L-iduronidase, or IDUA) in MPS I a rare, childhood genetic disease.


Lead Product(s): Iduronicrin Genleukocel-T

Therapeutic Area: Genetic Disease Product Name: ISP-001

Highest Development Status: Phase IProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: California Institute for Regenerative Medicine

Deal Size: $8.0 million Upfront Cash: Undisclosed

Deal Type: Funding January 27, 2023

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ISP-001 (iduronicrin genleukocel-T) represents the first product candidate developed using the company’s proprietary technique for the treatment of mucopolysaccharidosis type I (MPS I).


Lead Product(s): Iduronicrin Genleukocel-T

Therapeutic Area: Genetic Disease Product Name: ISP-001

Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 01, 2022

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The funding will support the development of its ISP- 002 (for delivery of iduronate sulfatase) program in mucopolysaccharidosis type II (MPS II), an inherited disease for which patients have limited options.


Lead Product(s): Iduronate Sulfatase

Therapeutic Area: Genetic Disease Product Name: ISP-002

Highest Development Status: IND EnablingProduct Type: Large molecule

Partner/Sponsor/Collaborator: California Institute for Regenerative Medicine

Deal Size: $4.0 million Upfront Cash: Undisclosed

Deal Type: Funding November 29, 2021

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