Phase-Appropriate Plasmid Manufacturing: It’s in Our DNA

Supporting cell and gene therapy (CGT) developers as they navigate the clinical path to market is in our DNA; to meet the growing demand for plasmid DNA development, manufacturing, and testing services, we have extended our integrated CGT capacity and capabilities to streamline complex supply chains. We are proud to offer our clients a comprehensive "concept to cure" advanced therapies solution spanning plasmid, viral vector, and cell therapy products and services.

Critical Starting Material for Engineered Stem Cell Cancer Therapies

In March we announced an extension of an historic research-grade plasmid manufacturing collaboration with National University of Singapore's (NUS) Yong Loo Lin School of Medicine (NUS Medicine), a leading medical education institution with a world-renowned medical research field of stem cell biology and therapy, adding a High Quality (HQ) plasmid DNA contract development and manufacturing (CDMO) agreement.

Leveraging the eXpDNA™ universal plasmid manufacturing platform, HQ plasmid serves as a phase-appropriate solution, combining key features of good manufacturing practice (GMP) manufacture with a rapid turnaround to accelerate time to clinic.

NUS Medicine will utilize our Keele and Alderley Park (UK) CDMO team's expertise in HQ and GMP-compliant plasmid DNA, which serves as a critical starting material for its engineered stem cell cancer therapy.

Gene Therapy Studies for Rare Diseases with No Cure

Supporting the development of gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS), a condition with limited treatment options and no cure, our team has also manufactured HQ gene of interest (GOI) plasmid for Axovia Therapeutics.

"This collaboration brings us closer to accomplishing our goal to develop potentially transformative therapies for ciliopathies," commented Victor Hernandez, Co-Founder and Chief Scientific Officer, Axovia. "We are excited to leverage Charles River's expertise in plasmid DNA production to help drive our work forward."

With over 20 ciliopathies currently identified, affecting an estimated 1 in 1,000 people, the diseases can cause blindness, deafness, chronic respiratory infections, kidney disease, heart disease, infertility, obesity, and diabetes.

Granted FDA Orphan Drug and Rare Pediatric Disease Designations, Axovia's lead program, AXV101, is an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with BBS. In preclinical studies, the gene therapy modified the underlying disease, rescuing vision loss by halting retinal degeneration, also stopping BBS-induced weight gain and the development of obesity.

An Active Drug Substance for HOX Biologics

Ship of Theseus, a therapeutics company developing degradation-resistant homeobox (HOX) family biologics, is also leveraging our plasmid DNA CDMO expertise, utilizing GMP-grade plasmid to serve as the active drug substance for its lead candidate.

Developing a proprietary suite of biologics based on the HOX family of transcription factors, which are responsible for driving the differentiation of tissue stem cells towards their respective lineages to repair and maintain the correct function of tissues and organs, proprietary modifications may enable these regulators to be used as therapeutics.

The collaboration will support therapeutic development for a broad variety of target indications such as diabetic and chronic wounds, neutropenia, psoriasis, androgenetic alopecia, women's health and infertility, and epithelial cancers.

"Ship of Theseus is proud to collaborate with Charles River as our manufacturing partner of choice," remarked Jeremy Elser, Founding CEO, Ship of Theseus. "We trust the team's decades of success developing, producing, and reliably delivering plasmid DNA and look forward to bringing our therapies to patients."

Our GMP plasmid DNA CDMO center of excellence will lead the collaboration, providing services that include plasmid DNA backbone generation, plasmid synthesis, GMP master cell banking (MCB), pre-production evaluation, and GMP plasmid DNA manufacture including in-house release testing.

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.

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