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rAAV-Olig001-ASPA is the first gene therapy designed to target the oligodendrocytes, which are critical for myelination and nerve function. Therepy reduces N-Acetylaspartate concentration, allowing myelination to occur. It is currently being investigated for canavan disease.
Lead Product(s): rAAV-Olig001-ASPA
Therapeutic Area: Genetic Disease Product Name: rAAV-Olig001-ASPA
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 09, 2023
Details:
rAAV-Olig001-ASPA is a novel vector from a class of recombinant AAVs that selectively target oligodendrocytes–the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brain’s white matter.
Lead Product(s): rAAV-Olig001-ASPA
Therapeutic Area: Genetic Disease Product Name: rAAV-Olig001-ASPA
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 04, 2022
Details:
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
Recipient: Forge Biologics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership October 03, 2022
Details:
Following the licensing agreement, Myrtelle is now conducting a first-in-human Phase I/II clinical study for CD under an open U.S. Investigational New Drug (IND) AVASPA.
Lead Product(s): rAAV-Olig001-ASPA
Therapeutic Area: Genetic Disease Product Name: AVASPA
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Pfizer Inc
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement December 14, 2021
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