An Avant-Garde Approach: AAV Gene Therapy Targets Rare Disease

Working hand in hand, our contract development and manufacturing organization (CDMO) team is committed to supporting AAVantgarde, a clinical-stage biotech focused on developing adeno-associated viral (AAV) vector platforms to treat inherited retinal diseases.

With two proprietary AAV-based large gene delivery platforms, both of which are based in ophthalmology and aim to enable efficient delivery of large genes to tissue and cells in vivo, AAVantgarde will utilize off-the-shelf Rep/Cap and pHelper plasmid products in addition to custom GMP grade plasmid manufactured at our plasmid DNA center of excellence in the UK.

Once manufactured by our team, the plasmid DNA will serve as the active drug substance for this therapeutic, which is entering Phase I clinical trials.

Kerstin Dolph, CSVP, Global Manufacturing, Charles River, commented in a press release announcing the collaboration : "Charles River is thrilled to produce GMP plasmid DNA to help advance AAVantgarde's platform for the treatment of Stargardt's disease - a condition in which there is high unmet need. Our team brings more than two decades of CDMO expertise, and we look forward to leveraging these capabilities to make a difference for patients."

Stargardt's Disease

Stargardt's disease causes progressive loss of central vision starting from childhood or adolescence, resulting in significant vision loss. AAVantgarde's AAVB-039 program targets this indication which is the most prevalent inherited macular dystrophy, affecting approximately 1 in 6,500 people, and represents a high unmet need with no existing therapies available.

The Stargardt's program is driven by the AAV-intein platform, which works by protein trans-splicing within the cell, and has demonstrated efficient recombination to deliver therapeutically meaningful protein levels.

"By collaborating with Charles River, we are one step closer to beginning clinical trials to ensure our therapeutic for Stargardt's Disease (STGD) is safe and efficacious for patients," added Nina Kotsopoulou, PhD, CTO, AAVantgarde, "We trust the team's decades of success developing, producing, and reliably delivering plasmid DNA and look forward to expanding treatment options for this patient population."

For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.

¹Trapani I., Colella P., et. al. Effective delivery of large genes to the retina by dual AAV vectors. EMBO Molecular Medicine, February 2014.

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