Andelyn Biosciences Enhances Gene Therapy Options with New CNS Capsids from Broad Institute
In a significant advancement for the field of gene therapy, Andelyn Biosciences, a Columbus-based Contract Development and Manufacturing Organization (CDMO), has announced the expansion of its AAV Curator™ Platform. This expansion comes as a result of a new licensing agreement with the prestigious Broad Institute of MIT and Harvard, enabling Andelyn to integrate CNS (Central Nervous System) capsids developed by the Stanton Lab. This innovative addition aims to bolster the efficacy of gene therapies targeted at nervous system disorders.
The integration of Stanton Lab's CNS capsids into Andelyn’s AAV Curator™ Platform signals a pivotal step towards refining gene therapy techniques. These capsids, specifically designed to enhance brain transduction while minimizing the tropism associated with the liver and peripheral organs, hold the promise of bringing new hope to patients suffering from neurologic diseases. Research indicates that these new capsids could enhance transduction efficiencies significantly, achieving figures up to six times more effective across various regions of the cerebrum and an astounding thirteen times more potent in targeting the neuroretina. Such improvements could ultimately lead to more focused treatments with reduced off-target effects, a common challenge in therapeutic designs.
Matt Niloff, Chief Commercial Officer at Andelyn, emphasized the importance of this collaboration, stating, "Expanding our agreement with the Broad Institute to include these novel CNS capsids enables Andelyn to offer critical tools to our clients who are seeking to manufacture gene therapies for neurologic diseases. We're excited by the potential to provide safer and more effective treatments for rare central nervous system diseases to patients in need of these critical treatments."
The partnership between Andelyn and the Broad Institute is structured to not only enhance Andelyn’s existing capabilities but also to extend their clients' capacities in developing cutting-edge gene therapies. The license agreement permits Andelyn to leverage the Stanton Lab CNS capsids for research and development activities, including screening potential gene therapy candidates and conducting pre-clinical development suitable for IND-enabling studies. Furthermore, the agreement facilitates sublicensing of these advantageous materials to clients for their internal research purposes, thus fostering an ecosystem of innovation within the biotech community.
Andelyn has been at the forefront of biotherapeutics development with over 20 years of experience, specializing in the production and characterization of viral vectors for gene therapy. Operating from their state-of-the-art facilities in Columbus, Ohio, the organization prides itself on maintaining rigorous quality standards while providing scalable solutions for developing and manufacturing gene and cell therapies. They have successfully produced cGMP materials for more than 450 clinical batches across 75 global clinical trials, showcasing an impressive portfolio of service and expertise.
The AAV Curator™ Platform is central to Andelyn's operational model, designed to accelerate programs while ensuring high-quality outcomes. Capabilities extend to cGMP manufacturing for suspension processes of up to 2,000 liters, alongside adherent processes, all supported by a thorough quality system and regulatory frameworks. This framework empowers clients to transition their therapies from concept through engineering, development, and commercialization.
In conclusion, the integration of Stanton Lab CNS capsids into Andelyn's AAV Curator™ Platform is a monumental stride forward not just for the company, but for the entire field of gene therapy targeting neurological conditions. As Andelyn continues to innovate and expand its offerings, the potential for improved treatment pathways grows, promising brighter prospects for patients facing rare and challenging disorders. To learn more about Andelyn Biosciences and their developments, visit their website at andelynbio.com.
The integration of Stanton Lab's CNS capsids into Andelyn’s AAV Curator™ Platform signals a pivotal step towards refining gene therapy techniques. These capsids, specifically designed to enhance brain transduction while minimizing the tropism associated with the liver and peripheral organs, hold the promise of bringing new hope to patients suffering from neurologic diseases. Research indicates that these new capsids could enhance transduction efficiencies significantly, achieving figures up to six times more effective across various regions of the cerebrum and an astounding thirteen times more potent in targeting the neuroretina. Such improvements could ultimately lead to more focused treatments with reduced off-target effects, a common challenge in therapeutic designs.
Matt Niloff, Chief Commercial Officer at Andelyn, emphasized the importance of this collaboration, stating, "Expanding our agreement with the Broad Institute to include these novel CNS capsids enables Andelyn to offer critical tools to our clients who are seeking to manufacture gene therapies for neurologic diseases. We're excited by the potential to provide safer and more effective treatments for rare central nervous system diseases to patients in need of these critical treatments."
The partnership between Andelyn and the Broad Institute is structured to not only enhance Andelyn’s existing capabilities but also to extend their clients' capacities in developing cutting-edge gene therapies. The license agreement permits Andelyn to leverage the Stanton Lab CNS capsids for research and development activities, including screening potential gene therapy candidates and conducting pre-clinical development suitable for IND-enabling studies. Furthermore, the agreement facilitates sublicensing of these advantageous materials to clients for their internal research purposes, thus fostering an ecosystem of innovation within the biotech community.
Andelyn has been at the forefront of biotherapeutics development with over 20 years of experience, specializing in the production and characterization of viral vectors for gene therapy. Operating from their state-of-the-art facilities in Columbus, Ohio, the organization prides itself on maintaining rigorous quality standards while providing scalable solutions for developing and manufacturing gene and cell therapies. They have successfully produced cGMP materials for more than 450 clinical batches across 75 global clinical trials, showcasing an impressive portfolio of service and expertise.
The AAV Curator™ Platform is central to Andelyn's operational model, designed to accelerate programs while ensuring high-quality outcomes. Capabilities extend to cGMP manufacturing for suspension processes of up to 2,000 liters, alongside adherent processes, all supported by a thorough quality system and regulatory frameworks. This framework empowers clients to transition their therapies from concept through engineering, development, and commercialization.
In conclusion, the integration of Stanton Lab CNS capsids into Andelyn's AAV Curator™ Platform is a monumental stride forward not just for the company, but for the entire field of gene therapy targeting neurological conditions. As Andelyn continues to innovate and expand its offerings, the potential for improved treatment pathways grows, promising brighter prospects for patients facing rare and challenging disorders. To learn more about Andelyn Biosciences and their developments, visit their website at andelynbio.com.
Topics Health）
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