Oligonucleotide Delivery

In response to critical unmet needs in oligonucleotide delivery, Genzyme Pharmaceuticals provides value-added solutions for delivering these therapeutic actives using a unique combination of products, services, technologies, and strategic relationships.

Products

The following readily available products can be used in liposomal and other lipid-based delivery systems which are designed for oligonucleotide-based delivery:

Custom Manufacturing Services

Through an integrated resource of custom manufacturing experience and expertise, Genzyme Pharmaceuticals’ core competencies in lipids, peptides, polymers, carbohydrates, lipo-peptides, and other small molecules provide proprietary, high quality, GMP excipients needed for cutting-edge oligonucleotide-based delivery systems.

Additional advantages to custom manufacturing through Genzyme Pharmaceuticals are optimized synthesis routes, development of scalable processes, efficient transition into cGMP production from pre-clinical to commercial scale, and step-wise implementation of analytical and regulatory support required for filing.

Drug Delivery Technologies

Genzyme Pharmaceuticals offers two proprietary lipid-based drug delivery technologies that may be considered for oligonucleotide delivery:

LipoBridge^® – transport across the blood brain barrier into the CNS

LipoMask^TM – increasing circulation half life in liposomal delivery

Strategic Relationships

Genzyme Pharmaceuticals offers a series of strategic relationships designed to provide synergies with our core competencies:

Cerense^SM – developed through a collaboration with Pharmidex, Ltd., providing in-vivo and in-vitro measurements for CNS therapies.

Design for Peptide Delivery^SM – developed through the SurModics Pharmaceuticals - Genzyme Pharmaceuticals Collaboration, with emphasis on optimizing peptides for controlled release as well as contract formulation development.

Supplying Every Link in the Peptide Chain – developed through the Mimotopes – Genzyme Pharmaceuticals Global Alliance, providing seamless transition from supply of R&D peptide candidates through scale up and GMP production of peptides.