Data from Oral Presentation Identifies Three Novel AAV Capsids with High Retinal Transduction
Additional Data Supports Utility of a Novel Cell Line for Efficient AAV Manufacturing
CAMBRIDGE, Mass., May 19, 2022 – Vedere Bio II, Inc., a company developing transformative, next-generation therapies for vision restoration and preservation, today will present new preclinical data at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place in Washington, D.C., and virtually, May 16-19, 2022.
“These data represent a significant step forward for the field of ocular gene therapy, as we have now identified three novel AAV capsids which provide exceptional retinal transduction, as well as a new cell line that enables the manufacturing of AAV vectors at higher titers and percent-full capsids,” said Gabor Veres, Ph.D., Chief Scientific Officer of Vedere Bio II. “Taken together, these advances not only enhance the probability of success for Vedere’s lead program, but also the ability to build a pipeline and scale the production of high-quality vector preps.”
Data from the ASGCT presentations are summarized below.
Three proprietary AAV capsids are exceptionally efficient at transducing retinal cells following intravitreal (IVT) injection
Oral title: Novel AAV Capsids for Intravitreal Delivery: Identifying and Characterizing Novel AAV Variants in Non-Human Primates (Abstract #1199)
Date/Time/Location: Thursday, May 19, 2022 from 11:15 AM – 11:30 AM in Ballroom A
Session: Novel AAV Capsids for the Brain, Eye, and Kidney
- The inner limiting membrane (ILM), is a physical barrier on the retina that prevents therapeutics, including adeno-associated viruses (AAVs), from reaching the diseased retinal cells through intravitreal injection (IVT).
- To overcome this barrier, a directed evolution approach was utilized to identify three novel capsids that:
- Display superior transduction efficiency to retinal cells, transducing all retinal layers in non-human primates
- Show reduced off-target transduction (Ciliary body)
- Limit inflammatory response (preclinical assay in non-human primates)
- Show more efficient manufacturability (>2 fold of parental AAV2 serotype)
New cell line enables the manufacturing of AAV vectors at higher titers and percent-full capsids
Poster title: X-RAP™ Suspension Cell Line and Its Platform: Vedere’s Solution for rAAV Manufacturing (Abstract #416)
Date/Time/Location: Monday, May 16, 2022 from 5:30 PM – 6:30 PM in Hall D
Session: Vector Product Engineering, Development or Manufacturing I
- Manufacturing AAV vectors at high titers and percent-full capsids is essential for both commercial viability as well as clinical success (given regulatory requirements).
- Vedere successfully developed a suspension cell line (X-RAP™), which along with demonstrating zero cell aggregation in high density cultures, consumes less nutrients, quintuples the percent-full capsids and has a titer ~1.8x greater than a commercially available cell line (Gibco™ Viral Production Cells) for rAAV production.
- Performance was consistent across different cell passage stages, in different scales of shake flask and in a 2L bioreactor. Vedere plans to scale production to 50L bioreactors to confirm that performance is consistent for an industrial-scale process.
About Vedere Bio II, Inc.
Vedere Bio II is a privately held, emerging biopharmaceutical company leveraging proprietary optogenetics and photoswitch technologies along with novel AAV capsids to restore vision in all patients with vision loss due to photoreceptor cell death. Comprising a diverse team of pioneering scientists and drug developers, Vedere Bio II is discovering and developing next generation ocular therapies to restore and preserve vision for those patients. The company is headquartered in Cambridge, MA and is funded by Atlas Ventures, Octagon Capital, Mission BioCapital, Samsara BioCapital, the RD Fund and Casdin Capital. For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn.
Verge Scientific Communications