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FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy. It is being evaluated in phase 1/2 clinical trials for the treatment of Krabbe Disease.


Lead Product(s): FBX-101

Therapeutic Area: Genetic Disease Product Name: FBX-101

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 29, 2024

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Forge will help to advance Life Bio’s partial epigenetic reprogramming platform to address aging-related diseases, including its lead program targeting ophthalmic indications by providing cGMP manufacturing and development services for its novel AAV gene therapy platform.


Lead Product(s): Undisclosed

Therapeutic Area: Ophthalmology Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Life Biosciences

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership May 08, 2023

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FBX-101 is an adeno-associated viral serotype rh10 gene therapy which delivers a functional copy of the GALC gene to cells in both the CNS and PNS. It has been shown to functionally correct the central and peripheral neuropathy associated with krabbe.


Lead Product(s): FBX-101

Therapeutic Area: Genetic Disease Product Name: FBX-101

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 17, 2023

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The partnership will advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS).


Lead Product(s): ET-101

Therapeutic Area: Neurology Product Name: ET-101

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Eikonoklastes Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership November 29, 2022

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Subjects treated with FBX-101 (AAVrh.10-hGALC) have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF), normal white matter myelination and normalization of motor development in two children 90 days and 9 months post-treatment


Lead Product(s): AAVrh.10-hGALC

Therapeutic Area: Genetic Disease Product Name: FBX-101

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 October 11, 2022

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Forge will provide research-grade and GMP-Pathway plasmid manufacturing services, in addition to adeno-associated viral vector (AAV) process development, scale-up engineering, and cGMP manufacturing services for Ray Therapeutics’ program, RTx-015.


Lead Product(s): RTx-015

Therapeutic Area: Genetic Disease Product Name: RTx-015

Highest Development Status: IND EnablingProduct Type: Large molecule

Recipient: Ray Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration October 10, 2022

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Forge will provide research-grade and GMP-Pathway plasmid manufacturing services as well as cGMP adeno-associated viral (AAV) process development and scale-up manufacturing services for Myrtelle’s program, Myr-201 (gene therapy delivery of a therapeutic TMPRSS3).


Lead Product(s): AAV-based TMPRSS3 Gene Therapy

Therapeutic Area: Otolaryngology (Ear, Nose, Throat) Product Name: Myr-201

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

Partner/Sponsor/Collaborator: Myrtelle

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership October 03, 2022

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The Company will use the funding to expand client offerings, including proprietary technologies, manufacturing systems, cell lines, and additional services to bolster its end-to-end manufacturing platform.


Lead Product(s): AAV-based Gene Therapy

Therapeutic Area: Technology Product Name: Undisclosed

Highest Development Status: UndisclosedProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Drive Capital

Deal Size: $90.0 million Upfront Cash: Undisclosed

Deal Type: Series C Financing September 12, 2022

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FBX-101 is an adeno-associated viral serotype rh10 (AAVrh.10-hGALC) gene therapy that is typically delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system.


Lead Product(s): AAVrh.10-hGALC

Therapeutic Area: Genetic Disease Product Name: FBX-101

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 30, 2022

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Ray-001 is intended for use in treating patients with retinitis pigmentosa (RP). Optogenetics is a promising approach that has the potential to restore useful vision to visually-impaired and blind individuals.


Lead Product(s): Ray-001

Therapeutic Area: Genetic Disease Product Name: Ray-001

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Ray Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership March 01, 2022

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