Advance Your Nucleic Acid Molecule Development
Novel therapies based on nucleic acid polymers are enabling the treatment and cure of human diseases previously unaddressed by small molecule or protein drugs. Given the rapid evolution of nucleic acid-based therapeutics, finding trusted organizations to partner with for your project requires comprehensive services, adaptable approaches, and proven success, particularly when your drug is innovative. We can support your research and development needs from discovery support through pre-clinical development to cGMP manufacturing through stability services. Our flexible development support and cumulative expertise allows you to combine analytical, formulation, and process development needs into custom projects. This approach provides better quality results, condenses timelines, and improves the value for your research budget.
Our diverse experience spans chemically synthesized oligonucleotides (e.g., ASO, siRNA, aptamer, sgRNA, PMO/PPMO, etc.) as well as larger nucleic acid polymers produced by biological processes, such as in vitro transcription (e.g., mRNA, tRNA, circular RNA, etc.) or bacterial expression (e.g., plasmid DNA). Beyond the technical know-how gained from our experience, our scientists are skilled researchers who identify and solve new technical issues preventing readiness for clinical development thereby increasing the value of your intellectual property. Our pre-clinical teams have experience developing formulations and scalable processes to advance your program.
In addition to our experience with nucleic acid-based therapies, our small molecule and protein biologics scientists can support the development of novel drug products that cross disciplinary boundaries. For example, our experience includes drugs products formed from nucleic acids and proteins such as oligonucleotides conjugated to antibodies as well as virus-like particles or viruses (e.g., AAV, adenovirus, lentivirus, etc.). Partner with us to empower your project with the trusted, capable support you seek to successfully advance.
Comprehensive Drug Development
Beyond development and validation of assay, identity, and purity methods, our scientists can help characterize the chemical and physical properties of your materials and determine the identity of unexpected impurities due to the process or chemical nature of your drug product.
Our team offers the experience and technical skills to support the bioanalytical testing for your GLP and clinical studies. We develop and validate bioanalytical methods and perform sample testing to provide you with more options to complete needed DMPK studies.
Our services encompass the entire lifecycle of analytical methods to support your GMP manufacturing needs. Then, we transfer your method to the manufacturing facility and GMP testing laboratory for validation and release or stability testing. As an integrated central testing laboratory, you can add or change manufacturers while maintaining confidence in the consistency and comparability of the analytical results, thereby saving time and money investigating anomalous results due to different methods or testing sites.
Delivery of nucleic acids to the cytosol or nucleus of target cells is usually the largest challenge for a given drug. We have experience developing formulations for diverse oligonucleotides, such as lipid conjugates and RNA-based drugs in lipid nanoparticles or other novel nanoparticles. We work with you to create a custom formulation development plan for your drug substance and intended route of administration that best achieves your target stability profile.
Our process development experience for nucleic acid-based molecules covers novel processes beyond the common platform processes of solid phase synthesis of oligonucleotides or in vitro transcription of RNA therapeutics. We support development of processes including chemical conjugations, enzymatic synthesis/modification of nucleic acids, and downstream purification or buffer exchanges. Our services also include development of lyophilization processes.
Our GMP manufacturing capabilities include downstream purification and buffer exchange as well as sterile fill/finish processes. We maintain clean room certification and have a successful track record of media fills. All filled materials undergo rigorous release testing and examination by our dedicated QC and QA teams before shipment to clinical sites.
Obtaining Orphan Drug Designations in the US and the EU for Rare Disease Treatments
Orphan designation programs at the FDA and the EMA offer incentives for sponsors to stimulate drug development for rare diseases, which otherwise would not be profitable due to small patient populations. This presentation will focus on what the orphan designation and RPD programs are, the incentives for obtaining such designations, and the required data needed to position your drug for regulatory success.
Integrated Laboratory Services
When your team needs additional CDMO support, our scientists are ready. Our state-of-the-art facilities and highly trained experts can add power to your project.