Innovative Gene Editing
Empowering your journey from discovery to commercialization
Building a successful gene editing therapy program faces numerous challenges at every step, from discovery through clinical stages and, ultimately, commercialization. From identifying precise genetic targets and optimizing gene editing tools, to navigating preclinical and clinical trials, you need access to cutting-edge tools, high-quality materials, and expert support.
Aldevron's comprehensive solutions empower drug developers to turn innovative ideas into life-changing treatments. Together, we pave the way for a future where genetic disorders can be effectively treated and lead to life-changing cures.
Tailored solutions for gene editing discovery
Identifying appropriate genetic targets for editing is a complex and time-consuming process requiring a deep understanding of intricate genetic and molecular pathways. It is crucial to ensure these targets are specific to the disease and do not affect other essential genes.
Whether your gene editing therapeutic delivery approach is in vivo or ex vivo, Aldevron has a comprehensive suite of high-quality products and services to support gene editing discovery designed to progress with your program as you move towards clinical applications.
Design and develop
Custom manufacture of client-specific Cas enzymes and other alternatives to CRISPR-Cas9.
Engineered and wild-type nucleases in stock at research grade to cGMP quality grades with DMF access supporting 15 commercial cell and gene therapy programs.
Custom manufacturing of mRNA-encoded CRISPR nucleases.
Guide RNA (gRNA) from IDT for discovery with a path to cGMP gRNAs.
Ribonucleoproteins (RNP)* complexing and characterization.
Custom manufactured donor templates including traditional plasmids and high-performance Nanoplasmid™ vector system.
Deliver, validate, screen
LNP-encapsulated mRNA encoding, CRISPR nucleases.
Viral vector transgene and packaging constructs.
RNP complexes of CRISPR nuclease and gRNA.
Comprehensive testing and analytics on each component, including our proprietary 21CFR Part 210 and 211 RNP quality assays.
By leveraging Aldevron's expertise and high-quality materials, researchers can achieve precise and effective gene editing outcomes, ensuring success at every stage of development.
Rigorous support to advance your therapeutic journey
Before moving to human trials, your gene editing therapy must undergo rigorous preclinical testing to evaluate safety, efficacy, and potential side effects. This phase includes toxicology studies and pharmacokinetics/pharmacodynamics assessments. Meeting that challenge will help ensure the production of sufficient materials for preclinical studies.
Whether you are moving forward with Aldevron-manufactured discovery tools and components or transferring in your own tools, Aldevron supports preclinical research by providing high-quality materials at the appropriate scale, with comprehensive analytical testing services.
Optimize and validate
25+ years of robust technology transfer/process optimization or process development capabilities to move your discovery to clinic efficiently.
Right-sized scale to grow with your needs as you progress from discovery to clinical trials.
Analyze
Comprehensive testing and analytics on each component, including our proprietary 21CFR Part 210 and 211 RNP* quality assays.
Adhere to regulatory standards
Early-phase regulatory support, including Investigational New Drug (IND) filing and comprehensive guidance, ensures that your products meet all necessary regulatory requirements.
Partnering with Aldevron for your gene editing solutions ensures a seamless transition from preclinical to clinical stages, providing the expertise and support needed to advance your projects with confidence.
Your Partner in Clinical and Commercial Gene Editing
Navigate clinical trials and commercial production with confidence
Clinical trials are not only necessary to ensure safety and efficacy but also require navigating the regulatory landscape and maintaining high manufacturing standards. Once approved, therapies must be scaled up for commercial production while ensuring consistent quality and safety. Quality control is paramount in this process, as it ensures that every batch of gene editing components meets stringent standards for purity, potency, and consistency.
Aldevron supports this process with cGMP-compliant production rigorous quality control, and adherence to clinical trial standards to minimize the risk of variability and ensure scalability at your request. Having supported 1,100+ clinical trials and 15 commercial programs, Aldevron helps navigate complex regulations and offers scalable manufacturing solutions to meet commercial demands.
Our commitment to quality control ensures that as therapies transition from clinical trials to commercial production, they maintain the highest levels of quality and safety.
Scale-up and phase-appropriate quality
25+ years of robust technology transfer/process optimization and cGMP-compliant manufacturing of custom components at scale for clinical and commercial programs.
In-stock cGMP manufactured catalog nucleases to streamline the transition to clinical/commercial applications.
Guide RNA (gRNA) produced at IDT’s Therapeutic Oligonucleotide Manufacturing Facility, paired with an Aldevron cGMP nuclease, creates GMP RNP* further supported by our proprietary RNP quality assay.
Execute clinical trials
Comprehensive quality control measures ensure product consistency and safety, essential for successful clinical trials.
Regulatory approval and commercialization
Extensive experience working with regulatory agencies over 25+ years supporting thousands of clinical trials.
Supporting 15 commercial programs with reliable commercial-scale manufacturing.
By partnering with Aldevron, researchers and pharmaceutical companies can confidently scale up their gene therapy projects with phase-appropriate quality and comprehensive support, ensuring successful clinical and commercial outcomes.
*Aldevron provides RNPs only to customers who are duly licensed, including to make and have made RNPs, for their intended use.
Gene Editing Products & Services
Aldevron offers a comprehensive suite of high-quality products and services to support every stage of your gene editing program, from discovery to cGMP.