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AAV gene therapy development and GMP manufacturing services

Gene Therapy Solutions

Gene therapies are revolutionizing treatment for genetic, neurological, ophthalmic, and rare diseases, but translating promising AAV programs into clinic-ready products is complex. ProBio's integrated Gene Therapy Solutions help sponsors accelerate development, de-risk manufacturing, and move confidently from discovery to the clinic.

From AAV Discovery to Commercial AAV Manufacturing

Gene therapy solutions workflow Seven-stage gene therapy workflow from AAV discovery and preclinical studies through clinical development and BLA filing, with corresponding manufacturing grades. AAVDiscovery PreclinicalStudies IND–EnablingCMC IND Filing/FDAApproval Early StageClinical Late StageClinical BLA Filing/FDAApproval Research Grade Pro Grade IND CMC Clinical GMPGrade Clinical GMPGrade PC & PV GMP Grade
Proprietary packaging systems delivering stable and scalable AAV processes Transfer, helper and Rep/Cap plasmids combine with PowerS-293 packaging cells to produce AAV vectors. Proprietary Packaging Systems Delivering Stable and Scalable AAV Processes TransferPlasmid GOI Transfer Plasmid.Proprietary PowerS™–ITRrs Strain,Other Commercial Strains Available AAssistV™–pHelper AAssistV™–pRC Helper Plasmids Rep/CapPlasmids Ready to Use AAssistV™ pHelper andAAssistV™ pRC Plasmids with FDADMF Registration PowerS™–293 Packaging Cell Lines Proprietary PowerS™–293 SuspensionCell Line with FDA DMF Registration Adeno–Associated Viral Vectors(AAV Vectors)
Poly(A) control for plasmid and mRNA whitepaper cover
ProSyn linearized DNA flyer cover

Frequently Asked Questions

Looking for an end-to-end AAV development partner?

Looking for an End-to-End AAV Development Partner?

Explore how our integrated discovery, CMC, and GMP manufacturing solutions can support your program.